| Year |
Citation |
Score |
| 2023 |
Powers S, Likhite S, Gadalla KK, Miranda CJ, Huffenberger AJ, Dennys C, Foust KD, Morales P, Pierson CR, Rinaldi F, Perry S, Bolon B, Wein N, Cobb S, Kaspar BK, et al. Novel MeCP2 Gene Therapy is Effective in a Multicenter Study using Two Mouse Models of Rett Syndrome and is Safe in Non-human Primates. Molecular Therapy : the Journal of the American Society of Gene Therapy. PMID 37481701 DOI: 10.1016/j.ymthe.2023.07.013 |
0.757 |
|
| 2023 |
Johnson TB, Brudvig JJ, Likhite S, Pratt MA, White KA, Cain JT, Booth CD, Timm DJ, Davis SS, Meyerink B, Pineda R, Dennys-Rivers C, Kaspar BK, Meyer K, Weimer JM. Early postnatal administration of an AAV9 gene therapy is safe and efficacious in CLN3 disease. Frontiers in Genetics. 14: 1118649. PMID 37035740 DOI: 10.3389/fgene.2023.1118649 |
0.607 |
|
| 2022 |
Foust KD, Wang X, McGovern VL, Braun L, Bevan AK, Haidet AM, Le TT, Morales PR, Rich MM, Burghes AHM, Kaspar BK. Retraction Note: Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN. Nature Biotechnology. PMID 36203015 DOI: 10.1038/s41587-022-01497-7 |
0.752 |
|
| 2022 |
Gomes C, Sequeira C, Likhite S, Dennys CN, Kolb SJ, Shaw PJ, Vaz AR, Kaspar BK, Meyer K, Brites D. Neurotoxic Astrocytes Directly Converted from Sporadic and Familial ALS Patient Fibroblasts Reveal Signature Diversities and miR-146a Theragnostic Potential in Specific Subtypes. Cells. 11. PMID 35406750 DOI: 10.3390/cells11071186 |
0.528 |
|
| 2022 |
Liao XH, Avalos P, Shelest O, Ofan R, Shilo M, Bresee C, Likhite S, Vit JP, Heuer H, Kaspar B, Meyer K, Dumitrescu AM, Refetoff S, Svendsen CN, Vatine GD. AAV9-MCT8 delivery at juvenile stage ameliorates neurological and behavioral deficits in a mouse model of MCT8-deficiency. Thyroid : Official Journal of the American Thyroid Association. PMID 35350867 DOI: 10.1089/thy.2022.0034 |
0.579 |
|
| 2021 |
Iyer CC, Chugh D, Bobbili PJ, Iii AJB, Crum AE, Yi AF, Kaspar BK, Meyer KC, Burghes AHM, Arnold WD. Follistatin-induced muscle hypertrophy in aged mice improves neuromuscular junction innervation and function. Neurobiology of Aging. 104: 32-41. PMID 33964607 DOI: 10.1016/j.neurobiolaging.2021.03.005 |
0.571 |
|
| 2021 |
Chugh D, Iyer CC, Bobbili P, Blatnik AJ, Kaspar BK, Meyer K, Burghes AH, Clark BC, Arnold WD. Voluntary wheel running with and without follistatin overexpression improves NMJ transmission but not motor unit loss in late life of C57BL/6J mice. Neurobiology of Aging. PMID 33678425 DOI: 10.1016/j.neurobiolaging.2021.01.012 |
0.576 |
|
| 2021 |
White KA, Nelvagal HR, Poole TA, Lu B, Johnson TB, Davis S, Pratt MA, Brudvig J, Assis AB, Likhite S, Meyer K, Kaspar BK, Cooper JD, Wang S, Weimer JM. Intracranial delivery of AAV9 gene therapy partially prevents retinal degeneration and visual deficits in CLN6-Batten disease mice. Molecular Therapy. Methods & Clinical Development. 20: 497-507. PMID 33665223 DOI: 10.1016/j.omtm.2020.12.014 |
0.622 |
|
| 2020 |
Blatnik AJ, McGovern VL, Le TT, Iyer CC, Kaspar BK, Burghes AHM. Conditional deletion of SMN in cell culture identifies functional SMN alleles. Human Molecular Genetics. PMID 33075805 DOI: 10.1093/hmg/ddaa229 |
0.556 |
|
| 2020 |
von Wittgenstein J, Zheng F, Wittmann MT, Balta EA, Ferrazzi F, Schäffner I, Häberle BM, Valero-Aracama MJ, Koehl M, Miranda CJ, Kaspar BK, Ekici AB, Reis A, Abrous DN, Alzheimer C, et al. Sox11 is an Activity-Regulated Gene with Dentate-Gyrus-Specific Expression Upon General Neural Activation. Cerebral Cortex (New York, N.Y. : 1991). PMID 32080705 DOI: 10.1093/Cercor/Bhz338 |
0.633 |
|
| 2019 |
Bravo-Hernandez M, Tadokoro T, Navarro MR, Platoshyn O, Kobayashi Y, Marsala S, Miyanohara A, Juhas S, Juhasova J, Skalnikova H, Tomori Z, Vanicky I, Studenovska H, Proks V, Chen P, ... Kaspar BK, et al. Spinal subpial delivery of AAV9 enables widespread gene silencing and blocks motoneuron degeneration in ALS. Nature Medicine. PMID 31873312 DOI: 10.1038/S41591-019-0674-1 |
0.674 |
|
| 2019 |
Al-Zaidy SA, Kolb SJ, Lowes L, Alfano LN, Shell R, Church KR, Nagendran S, Sproule DM, Feltner DE, Wells C, Ogrinc F, Menier M, L'Italien J, Arnold WD, Kissel JT, Kaspar BK, et al. AVXS-101 (Onasemnogene Abeparvovec) for SMA1: Comparative Study with a Prospective Natural History Cohort. Journal of Neuromuscular Diseases. PMID 31381526 DOI: 10.3233/Jnd-190403 |
0.612 |
|
| 2019 |
Cain JT, Likhite S, White KA, Timm DJ, Davis SS, Johnson TB, Dennys-Rivers CN, Rinaldi F, Motti D, Corcoran S, Morales P, Pierson C, Hughes SM, Lee SY, Kaspar BK, et al. Gene Therapy Corrects Brain and Behavioral Pathologies in CLN6-Batten Disease. Molecular Therapy : the Journal of the American Society of Gene Therapy. PMID 31331814 DOI: 10.1016/J.Ymthe.2019.06.015 |
0.678 |
|
| 2019 |
Mendonça LS, Nóbrega C, Tavino S, Brinkhaus M, Matos C, Tomé S, Moreira R, Henriques D, Kaspar BK, de Almeida LP. Ibuprofen enhances synaptic function and neural progenitors proliferation markers and improves neuropathology and motor coordination in Machado-Joseph disease models. Human Molecular Genetics. PMID 31127937 DOI: 10.1093/Hmg/Ddz097 |
0.663 |
|
| 2019 |
Noroozian Z, Xhima K, Huang Y, Kaspar BK, Kügler S, Hynynen K, Aubert I. MRI-Guided Focused Ultrasound for Targeted Delivery of rAAV to the Brain. Methods in Molecular Biology (Clifton, N.J.). 1950: 177-197. PMID 30783974 DOI: 10.1007/978-1-4939-9139-6_10 |
0.771 |
|
| 2019 |
Day JW, Chiriboga CA, Crawford TO, Darras BT, Finkel RS, Connolly AM, Iannaccone ST, Kuntz NL, Pena LD, Schultz M, Shieh PB, Smith EC, Farrar M, Feltner DE, Ogrinc FG, ... ... Kaspar BK, et al. 066 Avxs-101 gene-replacement therapy (GRT) for spinal muscular atrophy type 1 (SMA1): pivotal phase 3 study (STR1VE) update Journal of Neurology, Neurosurgery & Psychiatry. 90: A22.1-A22. DOI: 10.1136/Jnnp-2019-Anzan.58 |
0.616 |
|
| 2019 |
Day JW, Chiriboga CA, Crawford TO, Darras BT, Finkel RS, Connolly AM, Iannaccone ST, Kuntz NL, Peña LD, Schultz M, Shieh PB, Smith EC, Kausar I, Feltner DE, Ogrinc FG, ... ... Kaspar BK, et al. 250 AVXS-101 phase 3 study in spinal muscular atrophy type 1 Journal of Neurology, Neurosurgery, and Psychiatry. 90. DOI: 10.1136/Jnnp-2019-Abn-2.202 |
0.337 |
|
| 2019 |
Finkel R, Day J, Darras B, Kuntz N, Connolly A, Crawford T, Butterfield R, Shieh P, Tennekoon G, Iannaccone S, Ogrinc F, Kavanagh S, Kernbauer E, Whittle J, L'Italien J, ... Kaspar B, et al. O.40Intrathecal administration of onasemnogene abeparvovec gene-replacement therapy (GRT) for spinal muscular atrophy type 2 (SMA2): phase 1/2a study (STRONG) Neuromuscular Disorders. 29. DOI: 10.1016/J.Nmd.2019.06.594 |
0.323 |
|
| 2019 |
Finkel R, Day J, Darras B, Kuntz N, Connolly A, Crawford T, Butterfield R, Shieh P, Tennekoon G, Iannaccone S, Meriggioli M, Ogrinc F, Kavanagh S, Kernbauer E, Whittle J, ... ... Kaspar B, et al. Intrathecal administration of AVXS-101 gene-replacement therapy (GRT) for spinal muscular atrophy type 2 (SMA2): Phase 1/2A study (strong) Journal of the Neurological Sciences. 405: 57. DOI: 10.1016/J.Jns.2019.10.322 |
0.335 |
|
| 2018 |
Iannitti T, Scarrott JM, Likhite S, Coldicott IRP, Lewis KE, Heath PR, Higginbottom A, Myszczynska MA, Milo M, Hautbergue GM, Meyer K, Kaspar BK, Ferraiuolo L, Shaw PJ, Azzouz M. Translating SOD1 Gene Silencing toward the Clinic: A Highly Efficacious, Off-Target-free, and Biomarker-Supported Strategy for fALS. Molecular Therapy. Nucleic Acids. 12: 75-88. PMID 30195799 DOI: 10.1016/J.Omtn.2018.04.015 |
0.666 |
|
| 2018 |
Awad H, Bratasz A, Nuovo G, Burry R, Meng X, Kelani H, Brown M, Ramadan ME, Williams J, Bouhliqah L, Popovich PG, Guan Z, Mcallister C, Corcoran SE, Kaspar B, et al. MiR-155 deletion reduces ischemia-induced paralysis in an aortic aneurysm repair mouse model: Utility of immunohistochemistry and histopathology in understanding etiology of spinal cord paralysis. Annals of Diagnostic Pathology. 36: 12-20. PMID 29966831 DOI: 10.1016/J.Anndiagpath.2018.06.002 |
0.607 |
|
| 2018 |
Boczonadi V, Meyer K, Gonczarowska-Jorge H, Griffin H, Roos A, Bartsakoulia M, Bansagi B, Ricci G, Palinkas F, Zahedi RP, Bruni F, Kaspar B, Lochmüller H, Boycott KM, Müller JS, et al. Mutations in glycyl-tRNA-synthetase impair mitochondrial metabolism in neurons. Human Molecular Genetics. PMID 29648643 DOI: 10.1093/Hmg/Ddy127 |
0.592 |
|
| 2017 |
Mendell JR, Al-Zaidy S, Shell R, Arnold WD, Rodino-Klapac LR, Prior TW, Lowes L, Alfano L, Berry K, Church K, Kissel JT, Nagendran S, L'Italien J, Sproule DM, Wells C, ... ... Kaspar BK, et al. Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy. The New England Journal of Medicine. 377: 1713-1722. PMID 29091557 DOI: 10.1056/Nejmoa1706198 |
0.78 |
|
| 2017 |
Heilman PL, Song S, Miranda CJ, Meyer K, Srivastava AK, Knapp A, Wier CG, Kaspar BK, Kolb SJ. HSPB1 mutations causing hereditary neuropathy in humans disrupt non-cell autonomous protection of motor neurons. Experimental Neurology. PMID 28797631 DOI: 10.1016/J.Expneurol.2017.08.002 |
0.673 |
|
| 2017 |
Gombash SE, Cowley CJ, Fitzgerald JA, Lepak CA, Neides MG, Hook K, Todd LJ, Wang GD, Mueller C, Kaspar BK, Bielefeld EC, Fischer AJ, Wood JD, Foust KD. Systemic gene delivery transduces the enteric nervous system of guinea pigs and cynomolgus macaques. Gene Therapy. PMID 28771235 DOI: 10.1038/Gt.2017.72 |
0.816 |
|
| 2017 |
Hautbergue GM, Castelli LM, Ferraiuolo L, Sanchez-Martinez A, Cooper-Knock J, Higginbottom A, Lin YH, Bauer CS, Dodd JE, Myszczynska MA, Alam SM, Garneret P, Chandran JS, Karyka E, Stopford MJ, ... Kaspar BK, et al. SRSF1-dependent nuclear export inhibition of C9ORF72 repeat transcripts prevents neurodegeneration and associated motor deficits. Nature Communications. 8: 16063. PMID 28677678 DOI: 10.1038/Ncomms16063 |
0.648 |
|
| 2017 |
Brulet R, Matsuda T, Zhang L, Miranda C, Giacca M, Kaspar BK, Nakashima K, Hsieh J. NEUROD1 Instructs Neuronal Conversion in Non-Reactive Astrocytes. Stem Cell Reports. PMID 28506534 DOI: 10.1016/J.Stemcr.2017.04.013 |
0.749 |
|
| 2017 |
Mendell JR, Sahenk Z, Al-Zaidy S, Rodino-Klapac LR, Lowes LP, Alfano LN, Berry K, Miller N, Yalvac M, Dvorchik I, Moore-Clingenpeel M, Flanigan KM, Church K, Shontz K, Curry C, ... Kaspar BK, et al. Follistatin Gene Therapy for Sporadic Inclusion Body Myositis Improves Functional Outcomes. Molecular Therapy : the Journal of the American Society of Gene Therapy. PMID 28279643 DOI: 10.1016/J.Ymthe.2017.02.015 |
0.603 |
|
| 2017 |
Frakes AE, Braun L, Ferraiuolo L, Guttridge DC, Kaspar BK. Additive amelioration of ALS by co-targeting independent pathogenic mechanisms. Annals of Clinical and Translational Neurology. 4: 76-86. PMID 28168207 DOI: 10.1002/Acn3.375 |
0.81 |
|
| 2017 |
Lowes L, Al-Zaidy S, Shell R, Arnold W, Rodino-Klapac L, Prior T, Alfano L, Berry K, Church K, Kissel J, Nagendran S, L'Italien J, Sproule D, Wells C, Burghes A, ... ... Kaspar B, et al. P.374 - AVXS-101 phase 1 gene therapy clinical trial in SMA Type 1: patients treated early with the proposed therapeutic dose were able to sit unassisted at a younger age Neuromuscular Disorders. 27. DOI: 10.1016/J.Nmd.2017.06.414 |
0.31 |
|
| 2017 |
Mendell J, Al-Zaidy S, Shell R, Arnold W, Rodino-Klapac L, Prior T, Lowes L, Alfano L, Berry K, Church K, Kissel J, Nagendran S, L'Italien J, Sproule D, Wells C, ... ... Kaspar B, et al. P.372 - AVXS-101 phase 1 gene therapy clinical trial in SMA Type 1: end-of-Study event free survival and achievement of developmental milestones Neuromuscular Disorders. 27. DOI: 10.1016/J.Nmd.2017.06.412 |
0.304 |
|
| 2017 |
Shell R, Al-Zaidy S, Arnold WD, Rodino-Klapac L, Prior TW, Lowes L, Alfano L, Berry K, Church K, Kissel JT, Nagendran S, L'Italien J, Sproule DM, Burghes AH, Foust K, ... ... Kaspar BK, et al. AVXS-101 Phase 1 gene therapy clinical trial in SMA Type 1: Interim data demonstrates improvements in supportive care use European Journal of Paediatric Neurology. 21: e14. DOI: 10.1016/J.Ejpn.2017.04.1217 |
0.738 |
|
| 2017 |
Mendell JR, Al-Zaidy S, Shell R, Arnold WD, Rodino-Klapac L, Prior TW, Lowes L, Alfano L, Berry K, Church K, Kissel JT, Nagendran S, L'Italien J, Sproule DM, Cardenas J, ... ... Kaspar BK, et al. AVXS-101 Phase 1 gene therapy clinical trial in SMA Type 1: Event free survival and achievement of developmental milestones European Journal of Paediatric Neurology. 21: e13-e14. DOI: 10.1016/J.Ejpn.2017.04.1216 |
0.743 |
|
| 2016 |
Rinaldi F, Motti D, Ferraiuolo L, Kaspar BK. High Content Analysis in Amyotrophic Lateral Sclerosis. Molecular and Cellular Neurosciences. PMID 27965018 DOI: 10.1016/J.Mcn.2016.12.001 |
0.647 |
|
| 2016 |
Ferraiuolo L, Meyer K, Sherwood TW, Vick J, Likhite S, Frakes A, Miranda CJ, Braun L, Heath PR, Pineda R, Beattie CE, Shaw PJ, Askwith CC, McTigue D, Kaspar BK. Oligodendrocytes contribute to motor neuron death in ALS via SOD1-dependent mechanism. Proceedings of the National Academy of Sciences of the United States of America. PMID 27688759 DOI: 10.1073/Pnas.1607496113 |
0.809 |
|
| 2016 |
Miyanohara A, Kamizato K, Juhas S, Juhasova J, Navarro M, Marsala S, Lukacova N, Hruska-Plochan M, Curtis E, Gabel B, Ciacci J, Ahrens ET, Kaspar BK, Cleveland D, Marsala M. Potent spinal parenchymal AAV9-mediated gene delivery by subpial injection in adult rats and pigs. Molecular Therapy. Methods & Clinical Development. 3: 16046. PMID 27462649 DOI: 10.1038/Mtm.2016.46 |
0.688 |
|
| 2016 |
Iwayama H, Liao XH, Braun L, Bárez-López S, Kaspar B, Weiss R, Dumitrescu AM, Guadaño-Ferraz A, Refetoff S. Adeno associated virus 9-based gene therapy delivers a functional monocarboxylate transporter 8 (MCT8) which improves thyroid hormone availability to brain of Mct8 deficient mice. Thyroid : Official Journal of the American Thyroid Association. PMID 27432638 DOI: 10.1089/Thy.2016.0060 |
0.615 |
|
| 2016 |
Webster CP, Smith EF, Bauer CS, Moller A, Hautbergue GM, Ferraiuolo L, Myszczynska MA, Higginbottom A, Walsh MJ, Whitworth AJ, Kaspar BK, Meyer K, Shaw PJ, Grierson AJ, De Vos KJ. The C9orf72 protein interacts with Rab1a and the ULK1 complex to regulate initiation of autophagy. The Embo Journal. PMID 27334615 DOI: 10.15252/Embj.201694401 |
0.606 |
|
| 2016 |
Ahmed SS, Schattgen SA, Frakes AE, Sikoglu EM, Su Q, Li J, Hampton TG, Denninger AR, Kirschner DA, Kaspar B, Matalon R, Gao G. rAAV gene therapy in a Canavan's disease mouse model reveals immune impairments and an extended pathology beyond the central nervous system. Molecular Therapy : the Journal of the American Society of Gene Therapy. PMID 27039844 DOI: 10.1038/Mt.2016.68 |
0.795 |
|
| 2016 |
Song S, Miranda CJ, Braun L, Meyer K, Frakes AE, Ferraiuolo L, Likhite S, Bevan AK, Foust KD, McConnell MJ, Walker CM, Kaspar BK. Major histocompatibility complex class I molecules protect motor neurons from astrocyte-induced toxicity in amyotrophic lateral sclerosis. Nature Medicine. PMID 26928464 DOI: 10.1038/Nm.4052 |
0.806 |
|
| 2016 |
Meyer K, Kaspar BK. Glia - Neuron Interactions in Neurological Diseases: Testing Non-cell Autonomy in a Dish. Brain Research. PMID 26778174 DOI: 10.1016/J.Brainres.2015.12.051 |
0.653 |
|
| 2016 |
Gu JM, Wang DJ, Peterson JM, Shintaku J, Liyanarachchi S, Coppola V, Frakes AE, Kaspar BK, Cornelison DD, Guttridge DC. An NF-κB - EphrinA5-Dependent Communication between NG2(+) Interstitial Cells and Myoblasts Promotes Muscle Growth in Neonates. Developmental Cell. PMID 26777211 DOI: 10.1016/J.Devcel.2015.12.018 |
0.774 |
|
| 2016 |
Gombash SE, Cowley CJ, Fitzgerald JA, Hook K, Armstrong E, Kaspar BK, Fischer AJ, Todd L, Bielefeld EC, Foust KD. 612. Systemic AAV Injection in Guinea Pigs and Non-Human Primates Targets the Enteric Nervous System Molecular Therapy. 24: S242-S243. DOI: 10.1016/S1525-0016(16)33420-7 |
0.784 |
|
| 2016 |
Mendell JR, Sahenk Z, Hogan M, Al-Zaidy S, Flanigan K, Rodino-Klapac LR, McColly M, Church K, Lewis S, Lowes L, Alfano L, Berry K, Miller N, Dvorchik I, Moore-Clingenpeel M, ... Kaspar BK, et al. 497. Follistatin Gene Therapy Improves Six Minute Walk Distance in Sporadic Inclusion Body Myositis (sIBM) Molecular Therapy. 24: S197-S198. DOI: 10.1016/S1525-0016(16)33306-8 |
0.624 |
|
| 2016 |
Mendell JR, Al-Zaidy S, Shell R, Arnold WD, Rodino-Klapac L, Kissel JT, Prior TW, Miranda C, Lowes L, Alfano L, Berry K, Petek C, Church K, Braun L, Corcoran S, ... ... Kaspar BK, et al. 480. Gene Therapy for Spinal Muscular Atrophy Type 1 Shows Potential to Improve Survival and Motor Functional Outcomes Molecular Therapy. 24: S190. DOI: 10.1016/S1525-0016(16)33289-0 |
0.774 |
|
| 2015 |
Al-Zaidy SA, Sahenk Z, Rodino-Klapac LR, Kaspar B, Mendell JR. Follistatin Gene Therapy Improves Ambulation in Becker Muscular Dystrophy. Journal of Neuromuscular Diseases. 2: 185-192. PMID 27858738 DOI: 10.3233/Jnd-150083 |
0.62 |
|
| 2015 |
Sun S, Sun Y, Ling SC, Ferraiuolo L, McAlonis-Downes M, Zou Y, Drenner K, Wang Y, Ditsworth D, Tokunaga S, Kopelevich A, Kaspar BK, Lagier-Tourenne C, Cleveland DW. Translational profiling identifies a cascade of damage initiated in motor neurons and spreading to glia in mutant SOD1-mediated ALS. Proceedings of the National Academy of Sciences of the United States of America. PMID 26621731 DOI: 10.1073/Pnas.1520639112 |
0.66 |
|
| 2015 |
Nizzardo M, Simone C, Rizzo F, Salani S, Dametti S, Rinchetti P, Del Bo R, Foust K, Kaspar BK, Bresolin N, Comi GP, Corti S. Gene therapy rescues disease phenotype in a spinal muscular atrophy with respiratory distress type 1 (SMARD1) mouse model. Science Advances. 1: e1500078. PMID 26601156 DOI: 10.1126/Sciadv.1500078 |
0.814 |
|
| 2015 |
Israelson A, Ditsworth D, Sun S, Song S, Liang J, Hruska-Plochan M, McAlonis-Downes M, Abu-Hamad S, Zoltsman G, Shani T, Maldonado M, Bui A, Navarro M, Zhou H, Marsala M, Kaspar BK, et al. Macrophage migration inhibitory factor as a chaperone inhibiting accumulation of misfolded SOD1. Neuron. 86: 218-32. PMID 25801706 DOI: 10.1016/J.Neuron.2015.02.034 |
0.649 |
|
| 2015 |
Weber-Adrian D, Thévenot E, O'Reilly MA, Oakden W, Akens MK, Ellens N, Markham-Coultes K, Burgess A, Finkelstein J, Yee AJ, Whyne CM, Foust KD, Kaspar BK, Stanisz GJ, Chopra R, et al. Gene delivery to the spinal cord using MRI-guided focused ultrasound. Gene Therapy. 22: 568-77. PMID 25781651 DOI: 10.1038/Gt.2015.25 |
0.802 |
|
| 2015 |
Cheever TR, Berkley D, Braun S, Brown RH, Byrne BJ, Chamberlain JS, Cwik V, Duan D, Federoff HJ, High KA, Kaspar BK, Klinger KW, Larkindale J, Lincecum J, Mavilio F, et al. Perspectives on best practices for gene therapy programs. Human Gene Therapy. 26: 127-33. PMID 25654329 DOI: 10.1089/Hum.2014.147 |
0.608 |
|
| 2015 |
Hesp ZC, Goldstein EA, Miranda CJ, Kaspar BK, McTigue DM. Chronic oligodendrogenesis and remyelination after spinal cord injury in mice and rats. The Journal of Neuroscience : the Official Journal of the Society For Neuroscience. 35: 1274-90. PMID 25609641 DOI: 10.1523/Jneurosci.2568-14.2015 |
0.579 |
|
| 2015 |
Mendonça LS, Nóbrega C, Hirai H, Kaspar BK, Pereira de Almeida L. Transplantation of cerebellar neural stem cells improves motor coordination and neuropathology in Machado-Joseph disease mice. Brain : a Journal of Neurology. 138: 320-35. PMID 25527827 DOI: 10.1093/Brain/Awu352 |
0.649 |
|
| 2015 |
Duque SI, Arnold WD, Odermatt P, Li X, Porensky PN, Schmelzer L, Meyer K, Kolb SJ, Schümperli D, Kaspar BK, Burghes AH. A large animal model of spinal muscular atrophy and correction of phenotype. Annals of Neurology. 77: 399-414. PMID 25516063 DOI: 10.1002/Ana.24332 |
0.677 |
|
| 2015 |
Meyer K, Ferraiuolo L, Schmelzer L, Braun L, McGovern V, Likhite S, Michels O, Govoni A, Fitzgerald J, Morales P, Foust KD, Mendell JR, Burghes AH, Kaspar BK. Improving single injection CSF delivery of AAV9-mediated gene therapy for SMA: a dose-response study in mice and nonhuman primates. Molecular Therapy : the Journal of the American Society of Gene Therapy. 23: 477-87. PMID 25358252 DOI: 10.1038/Mt.2014.210 |
0.804 |
|
| 2015 |
Mendell JR, Sahenk Z, Malik V, Gomez AM, Flanigan KM, Lowes LP, Alfano LN, Berry K, Meadows E, Lewis S, Braun L, Shontz K, Rouhana M, Clark KR, Rosales XQ, ... Kaspar BK, et al. A phase 1/2a follistatin gene therapy trial for becker muscular dystrophy. Molecular Therapy : the Journal of the American Society of Gene Therapy. 23: 192-201. PMID 25322757 DOI: 10.1038/Mt.2014.200 |
0.614 |
|
| 2015 |
Kaspar B. Gene delivery for treating neurological disorders Neuromuscular Disorders. 25. DOI: 10.1016/J.Nmd.2015.06.445 |
0.315 |
|
| 2014 |
Thomsen GM, Gowing G, Latter J, Chen M, Vit JP, Staggenborg K, Avalos P, Alkaslasi M, Ferraiuolo L, Likhite S, Kaspar BK, Svendsen CN. Delayed disease onset and extended survival in the SOD1G93A rat model of amyotrophic lateral sclerosis after suppression of mutant SOD1 in the motor cortex. The Journal of Neuroscience : the Official Journal of the Society For Neuroscience. 34: 15587-600. PMID 25411487 DOI: 10.1523/Jneurosci.2037-14.2014 |
0.681 |
|
| 2014 |
Gombash Lampe SE, Kaspar BK, Foust KD. Intravenous injections in neonatal mice. Journal of Visualized Experiments : Jove. e52037. PMID 25407048 DOI: 10.3791/52037 |
0.758 |
|
| 2014 |
Meyer K, Kaspar BK. Making sense of pain: are pluripotent stem cell-derived sensory neurons a new tool for studying pain mechanisms? Molecular Therapy : the Journal of the American Society of Gene Therapy. 22: 1403-5. PMID 25082088 DOI: 10.1038/Mt.2014.123 |
0.631 |
|
| 2014 |
Frakes AE, Ferraiuolo L, Haidet-Phillips AM, Schmelzer L, Braun L, Miranda CJ, Ladner KJ, Bevan AK, Foust KD, Godbout JP, Popovich PG, Guttridge DC, Kaspar BK. Microglia induce motor neuron death via the classical NF-κB pathway in amyotrophic lateral sclerosis. Neuron. 81: 1009-23. PMID 24607225 DOI: 10.1016/J.Neuron.2014.01.013 |
0.797 |
|
| 2014 |
Arnold WD, Porensky PN, McGovern VL, Iyer CC, Duque S, Li X, Meyer K, Schmelzer L, Kaspar BK, Kolb SJ, Kissel JT, Burghes AH. Electrophysiological Biomarkers in Spinal Muscular Atrophy: Preclinical Proof of Concept. Annals of Clinical and Translational Neurology. 1: 34-44. PMID 24511555 DOI: 10.1002/Acn3.23 |
0.658 |
|
| 2014 |
Meyer K, Ferraiuolo L, Miranda CJ, Likhite S, McElroy S, Renusch S, Ditsworth D, Lagier-Tourenne C, Smith RA, Ravits J, Burghes AH, Shaw PJ, Cleveland DW, Kolb SJ, Kaspar BK. Direct conversion of patient fibroblasts demonstrates non-cell autonomous toxicity of astrocytes to motor neurons in familial and sporadic ALS. Proceedings of the National Academy of Sciences of the United States of America. 111: 829-32. PMID 24379375 DOI: 10.1073/Pnas.1314085111 |
0.676 |
|
| 2014 |
Sahenk Z, Galloway G, Clark KR, Malik V, Rodino-Klapac LR, Kaspar BK, Chen L, Braganza C, Montgomery C, Mendell JR. AAV1.NT-3 gene therapy for charcot-marie-tooth neuropathy. Molecular Therapy : the Journal of the American Society of Gene Therapy. 22: 511-21. PMID 24162799 DOI: 10.1038/Mt.2013.250 |
0.636 |
|
| 2014 |
Camboni M, Wang CM, Miranda C, Yoon JH, Xu R, Zygmunt D, Kaspar BK, Martin PT. Active and passive immunization strategies based on the SDPM1 peptide demonstrate pre-clinical efficacy in the APPswePSEN1dE9 mouse model for Alzheimer's disease. Neurobiology of Disease. 62: 31-43. PMID 24021662 DOI: 10.1016/J.Nbd.2013.09.001 |
0.582 |
|
| 2014 |
Mendell J, Sahenk Z, Rodino-Klapac L, Clark K, Lewis K, Shontz K, Al-Zaidy S, Alfano L, Lowes, L, Berry K, Malik V, Shilling C, Rosales X, Walker C, ... ... Kaspar B, et al. G.O.25 Neuromuscular Disorders. 24: 923. DOI: 10.1016/J.Nmd.2014.06.424 |
0.599 |
|
| 2014 |
Burghes A, Arnold D, Duque S, McGovern V, Ruhno C, Iyer C, Meyer K, Kaspar B. G.O.15 Neuromuscular Disorders. 24: 919. DOI: 10.1016/J.Nmd.2014.06.414 |
0.447 |
|
| 2013 |
Foust KD, Salazar DL, Likhite S, Ferraiuolo L, Ditsworth D, Ilieva H, Meyer K, Schmelzer L, Braun L, Cleveland DW, Kaspar BK. Therapeutic AAV9-mediated suppression of mutant SOD1 slows disease progression and extends survival in models of inherited ALS. Molecular Therapy : the Journal of the American Society of Gene Therapy. 21: 2148-59. PMID 24008656 DOI: 10.1038/Mt.2013.211 |
0.81 |
|
| 2013 |
Garg SK, Lioy DT, Cheval H, McGann JC, Bissonnette JM, Murtha MJ, Foust KD, Kaspar BK, Bird A, Mandel G. Systemic delivery of MeCP2 rescues behavioral and cellular deficits in female mouse models of Rett syndrome. The Journal of Neuroscience : the Official Journal of the Society For Neuroscience. 33: 13612-20. PMID 23966684 DOI: 10.1523/Jneurosci.1854-13.2013 |
0.752 |
|
| 2013 |
Rodino-Klapac LR, Janssen PM, Shontz KM, Canan B, Montgomery CL, Griffin D, Heller K, Schmelzer L, Handy C, Clark KR, Sahenk Z, Mendell JR, Kaspar BK. Micro-dystrophin and follistatin co-delivery restores muscle function in aged DMD model. Human Molecular Genetics. 22: 4929-37. PMID 23863459 DOI: 10.1093/Hmg/Ddt342 |
0.623 |
|
| 2013 |
Ferraiuolo L, Frakes A, Kaspar BK. Neural stem cells as a therapeutic approach for amyotrophic lateral sclerosis. Molecular Therapy : the Journal of the American Society of Gene Therapy. 21: 503-5. PMID 23449106 DOI: 10.1038/Mt.2013.24 |
0.828 |
|
| 2013 |
Meyer K, Miranda CJ, Kaspar BK. Transplantation of gene-corrected motor neurons as a therapeutic strategy for spinal muscular atrophy. Molecular Therapy : the Journal of the American Society of Gene Therapy. 21: 502-3. PMID 23449105 DOI: 10.1038/Mt.2013.23 |
0.652 |
|
| 2013 |
Duque S, Porensky P, William DA, Odermatt P, Nlend RN, Bevan AK, Foust K, Braun L, Schmelzer L, Schumperli D, Kaspar BK, Burghes AHM. O.8 Intrathecal delivery of AAV9 vectors to model and rescue a large animal model of SMA Neuromuscular Disorders. 23: 797. DOI: 10.1016/J.Nmd.2013.06.558 |
0.432 |
|
| 2013 |
Burghes A, McGovern V, Porensky P, Arnold D, Laporte A, Foust K, Mitrpant C, Le T, Wilton S, Kaspar B. O.7 Spinal muscular atrophy: How it works and therapeutic targets Neuromuscular Disorders. 23: 797. DOI: 10.1016/J.Nmd.2013.06.557 |
0.417 |
|
| 2013 |
Lowes L, Alfano L, Ray W, Wolock S, Flanigan K, Mendell J, Kaspar B. P.6.2 Development of a proof-of-concept device using the Microsoft Kinect to assess movement in infants with spinal muscular atrophy Neuromuscular Disorders. 23: 770. DOI: 10.1016/J.Nmd.2013.06.473 |
0.571 |
|
| 2012 |
Ferraiuolo L, Kaspar BK. Gene delivery improvement for treating the lysosomal storage disorder metachromatic leukodystrophy. Human Gene Therapy. 23: 793-5. PMID 22900577 DOI: 10.1089/Hum.2012.2516 |
0.597 |
|
| 2012 |
Thévenot E, Jordão JF, O'Reilly MA, Markham K, Weng YQ, Foust KD, Kaspar BK, Hynynen K, Aubert I. Targeted delivery of self-complementary adeno-associated virus serotype 9 to the brain, using magnetic resonance imaging-guided focused ultrasound. Human Gene Therapy. 23: 1144-55. PMID 22838844 DOI: 10.1089/Hum.2012.013 |
0.799 |
|
| 2012 |
Mendell JR, Rodino-Klapac L, Sahenk Z, Malik V, Kaspar BK, Walker CM, Clark KR. Gene therapy for muscular dystrophy: lessons learned and path forward. Neuroscience Letters. 527: 90-9. PMID 22609847 DOI: 10.1016/J.Neulet.2012.04.078 |
0.634 |
|
| 2012 |
Miranda CJ, Braun L, Jiang Y, Hester ME, Zhang L, Riolo M, Wang H, Rao M, Altura RA, Kaspar BK. Aging brain microenvironment decreases hippocampal neurogenesis through Wnt-mediated survivin signaling. Aging Cell. 11: 542-52. PMID 22404871 DOI: 10.1111/J.1474-9726.2012.00816.X |
0.768 |
|
| 2012 |
Lowes LP, Alfano L, Viollet L, Rosales XQ, Sahenk Z, Kaspar BK, Clark KR, Flanigan KM, Mendell JR, McDermott MP. Knee extensor strength exhibits potential to predict function in sporadic inclusion-body myositis. Muscle & Nerve. 45: 163-8. PMID 22246869 DOI: 10.1002/Mus.22321 |
0.568 |
|
| 2012 |
Samaranch L, Salegio EA, San Sebastian W, Kells AP, Foust KD, Bringas JR, Lamarre C, Forsayeth J, Kaspar BK, Bankiewicz KS. Adeno-associated virus serotype 9 transduction in the central nervous system of nonhuman primates. Human Gene Therapy. 23: 382-9. PMID 22201473 DOI: 10.1089/Hum.2011.200 |
0.775 |
|
| 2012 |
Porensky PN, Mitrpant C, McGovern VL, Bevan AK, Foust KD, Kaspar BK, Wilton SD, Burghes AH. A single administration of morpholino antisense oligomer rescues spinal muscular atrophy in mouse. Human Molecular Genetics. 21: 1625-38. PMID 22186025 DOI: 10.1093/Hmg/Ddr600 |
0.782 |
|
| 2012 |
Kota J, Shilling C, Montgomery C, Lewis S, Bevan A, Shontz K, Kaminoh Y, Rosales X, Viollet L, Flanigan K, Clark R, Kaspar B, Sahenk Z, Mendell J. Adeno-Associated Viral (AAV)-Mediated Follistatin (FS) Gene Transfer Toxicology Studies in Preparation for Phase I Clinical Trial (SC02.004) Neurology. 78: SC02.004-SC02.004. DOI: 10.1212/Wnl.78.1_Meetingabstracts.Sc02.004 |
0.621 |
|
| 2012 |
Burghes A, McGovern V, Porensky P, Duque S, Bevan A, Foust K, Odermatt P, Nlend R, Massoni-Laporte A, Mitrpant C, Wilton S, Schümperli D, Kaspar B. T.O.5 Spinal muscular atrophy SMA: SMNs spatial requirement and therapies Neuromuscular Disorders. 22: 907-908. DOI: 10.1016/J.Nmd.2012.06.342 |
0.447 |
|
| 2011 |
Lin Y, Roman K, Foust KD, Kaspar BK, Bailey MT, Stephens RL. Glutamate Transporter GLT-1 Upregulation Attenuates Visceral Nociception and Hyperalgesia via Spinal Mechanisms Not Related to Anti-Inflammatory or Probiotic Effects. Pain Research and Treatment. 2011: 507029. PMID 22220274 DOI: 10.1155/2011/507029 |
0.735 |
|
| 2011 |
Bockstael O, Foust KD, Kaspar B, Tenenbaum L. Recombinant AAV delivery to the central nervous system. Methods in Molecular Biology (Clifton, N.J.). 807: 159-77. PMID 22034030 DOI: 10.1007/978-1-61779-370-7_7 |
0.785 |
|
| 2011 |
White RE, Rao M, Gensel JC, McTigue DM, Kaspar BK, Jakeman LB. Transforming growth factor α transforms astrocytes to a growth-supportive phenotype after spinal cord injury. The Journal of Neuroscience : the Official Journal of the Society For Neuroscience. 31: 15173-87. PMID 22016551 DOI: 10.1523/Jneurosci.3441-11.2011 |
0.594 |
|
| 2011 |
Beastrom N, Lu H, Macke A, Canan BD, Johnson EK, Penton CM, Kaspar BK, Rodino-Klapac LR, Zhou L, Janssen PM, Montanaro F. mdx(⁵cv) mice manifest more severe muscle dysfunction and diaphragm force deficits than do mdx Mice. The American Journal of Pathology. 179: 2464-74. PMID 21893021 DOI: 10.1016/J.Ajpath.2011.07.009 |
0.617 |
|
| 2011 |
Fidler JA, Treleaven CM, Frakes A, Tamsett TJ, McCrate M, Cheng SH, Shihabuddin LS, Kaspar BK, Dodge JC. Disease progression in a mouse model of amyotrophic lateral sclerosis: the influence of chronic stress and corticosterone. Faseb Journal : Official Publication of the Federation of American Societies For Experimental Biology. 25: 4369-77. PMID 21876068 DOI: 10.1096/Fj.11-190819 |
0.795 |
|
| 2011 |
Haidet-Phillips AM, Hester ME, Miranda CJ, Meyer K, Braun L, Frakes A, Song S, Likhite S, Murtha MJ, Foust KD, Rao M, Eagle A, Kammesheidt A, Christensen A, Mendell JR, ... Kaspar BK, et al. Astrocytes from familial and sporadic ALS patients are toxic to motor neurons. Nature Biotechnology. 29: 824-8. PMID 21832997 DOI: 10.1038/Nbt.1957 |
0.809 |
|
| 2011 |
Bevan AK, Duque S, Foust KD, Morales PR, Braun L, Schmelzer L, Chan CM, McCrate M, Chicoine LG, Coley BD, Porensky PN, Kolb SJ, Mendell JR, Burghes AH, Kaspar BK. Systemic gene delivery in large species for targeting spinal cord, brain, and peripheral tissues for pediatric disorders. Molecular Therapy : the Journal of the American Society of Gene Therapy. 19: 1971-80. PMID 21811247 DOI: 10.1038/Mt.2011.157 |
0.805 |
|
| 2011 |
Hester ME, Murtha MJ, Song S, Rao M, Miranda CJ, Meyer K, Tian J, Boulting G, Schaffer DV, Zhu MX, Pfaff SL, Gage FH, Kaspar BK. Rapid and efficient generation of functional motor neurons from human pluripotent stem cells using gene delivered transcription factor codes. Molecular Therapy : the Journal of the American Society of Gene Therapy. 19: 1905-12. PMID 21772256 DOI: 10.1038/Mt.2011.135 |
0.812 |
|
| 2011 |
Lioy DT, Garg SK, Monaghan CE, Raber J, Foust KD, Kaspar BK, Hirrlinger PG, Kirchhoff F, Bissonnette JM, Ballas N, Mandel G. A role for glia in the progression of Rett's syndrome. Nature. 475: 497-500. PMID 21716289 DOI: 10.1038/Nature10214 |
0.775 |
|
| 2011 |
Peterson JM, Kline W, Canan BD, Ricca DJ, Kaspar B, Delfín DA, DiRienzo K, Clemens PR, Robbins PD, Baldwin AS, Flood P, Kaumaya P, Freitas M, Kornegay JN, Mendell JR, et al. Peptide-based inhibition of NF-κB rescues diaphragm muscle contractile dysfunction in a murine model of Duchenne muscular dystrophy. Molecular Medicine (Cambridge, Mass.). 17: 508-15. PMID 21267511 DOI: 10.2119/Molmed.2010.00263 |
0.585 |
|
| 2011 |
Jukkola PI, Rogers JT, Kaspar BK, Weeber EJ, Nishijima I. Secretin deficiency causes impairment in survival of neural progenitor cells in mice. Human Molecular Genetics. 20: 1000-7. PMID 21159798 DOI: 10.1093/Hmg/Ddq545 |
0.627 |
|
| 2011 |
Dodge J, Kaspar B, Shihabuddin L, Cheng S. P4.56 Alleviation of a cacostatic stress response slows disease progression in a mouse model of familial ALS Neuromuscular Disorders. 21: 721. DOI: 10.1016/J.Nmd.2011.06.1021 |
0.58 |
|
| 2011 |
Foust KD, Kaspar BK. Delivery strategies for RNAi to the nervous system Neuromethods. 58: 59-76. DOI: 10.1007/978-1-61779-114-7_4 |
0.731 |
|
| 2010 |
Dodge JC, Treleaven CM, Fidler JA, Hester M, Haidet A, Handy C, Rao M, Eagle A, Matthews JC, Taksir TV, Cheng SH, Shihabuddin LS, Kaspar BK. AAV4-mediated expression of IGF-1 and VEGF within cellular components of the ventricular system improves survival outcome in familial ALS mice. Molecular Therapy : the Journal of the American Society of Gene Therapy. 18: 2075-84. PMID 20859261 DOI: 10.1038/Mt.2010.206 |
0.821 |
|
| 2010 |
Petruska JC, Kitay B, Boyce VS, Kaspar BK, Pearse DD, Gage FH, Mendell LM. Intramuscular AAV delivery of NT-3 alters synaptic transmission to motoneurons in adult rats. The European Journal of Neuroscience. 32: 997-1005. PMID 20849530 DOI: 10.1111/J.1460-9568.2010.07392.X |
0.651 |
|
| 2010 |
Bevan AK, Hutchinson KR, Foust KD, Braun L, McGovern VL, Schmelzer L, Ward JG, Petruska JC, Lucchesi PA, Burghes AH, Kaspar BK. Early heart failure in the SMNDelta7 model of spinal muscular atrophy and correction by postnatal scAAV9-SMN delivery. Human Molecular Genetics. 19: 3895-905. PMID 20639395 DOI: 10.1093/Hmg/Ddq300 |
0.791 |
|
| 2010 |
Sahenk Z, Galloway G, Edwards C, Malik V, Kaspar BK, Eagle A, Yetter B, Forgie A, Tsao D, Lin JC. TrkB and TrkC agonist antibodies improve function, electrophysiologic and pathologic features in Trembler J mice. Experimental Neurology. 224: 495-506. PMID 20553714 DOI: 10.1016/J.Expneurol.2010.05.013 |
0.597 |
|
| 2010 |
Haidet AM, Mendell JR, Kaspar BK. Could gene therapy be the future for muscular dystrophy? Therapy. 7: 287-290. PMID 20543898 DOI: 10.2217/Thy.10.14 |
0.646 |
|
| 2010 |
An MC, Lin W, Yang J, Dominguez B, Padgett D, Sugiura Y, Aryal P, Gould TW, Oppenheim RW, Hester ME, Kaspar BK, Ko CP, Lee KF. Acetylcholine negatively regulates development of the neuromuscular junction through distinct cellular mechanisms. Proceedings of the National Academy of Sciences of the United States of America. 107: 10702-7. PMID 20498043 DOI: 10.1073/Pnas.1004956107 |
0.756 |
|
| 2010 |
Foust KD, Wang X, McGovern VL, Braun L, Bevan AK, Haidet AM, Le TT, Morales PR, Rich MM, Burghes AH, Kaspar BK. Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN. Nature Biotechnology. 28: 271-4. PMID 20190738 DOI: 10.1038/Nbt.1610 |
0.808 |
|
| 2010 |
Kaspar BK. Gene therapy: Direct viral delivery Encyclopedia of Neuroscience. 633-639. DOI: 10.1016/B978-008045046-9.00012-7 |
0.577 |
|
| 2009 |
Kota J, Handy CR, Haidet AM, Montgomery CL, Eagle A, Rodino-Klapac LR, Tucker D, Shilling CJ, Therlfall WR, Walker CM, Weisbrode SE, Janssen PM, Clark KR, Sahenk Z, Mendell JR, Kaspar BK, et al. Follistatin gene delivery enhances muscle growth and strength in nonhuman primates. Science Translational Medicine. 1: 6ra15. PMID 20368179 DOI: 10.1126/Scitranslmed.3000112 |
0.623 |
|
| 2009 |
Foust KD, Kaspar BK. Over the barrier and through the blood: to CNS delivery we go. Cell Cycle (Georgetown, Tex.). 8: 4017-8. PMID 19949299 DOI: 10.4161/Cc.8.24.10245 |
0.803 |
|
| 2009 |
Hester ME, Foust KD, Kaspar RW, Kaspar BK. AAV as a gene transfer vector for the treatment of neurological disorders: novel treatment thoughts for ALS. Current Gene Therapy. 9: 428-33. PMID 19860657 DOI: 10.2174/156652309789753383 |
0.839 |
|
| 2009 |
Hester ME, Song S, Miranda CJ, Eagle A, Schwartz PH, Kaspar BK. Two factor reprogramming of human neural stem cells into pluripotency. Plos One. 4: e7044. PMID 19763260 DOI: 10.1371/Journal.Pone.0007044 |
0.762 |
|
| 2009 |
Kaspar RW, Wills CE, Kaspar BK. Gene therapy and informed consent decision making: nursing research directions. Biological Research For Nursing. 11: 98-107. PMID 19398415 DOI: 10.1177/1099800409333169 |
0.606 |
|
| 2009 |
Excoffon KJ, Koerber JT, Dickey DD, Murtha M, Keshavjee S, Kaspar BK, Zabner J, Schaffer DV. Directed evolution of adeno-associated virus to an infectious respiratory virus. Proceedings of the National Academy of Sciences of the United States of America. 106: 3865-70. PMID 19237554 DOI: 10.1073/Pnas.0813365106 |
0.679 |
|
| 2009 |
Rodino-Klapac LR, Haidet AM, Kota J, Handy C, Kaspar BK, Mendell JR. Inhibition of myostatin with emphasis on follistatin as a therapy for muscle disease. Muscle & Nerve. 39: 283-96. PMID 19208403 DOI: 10.1002/Mus.21244 |
0.621 |
|
| 2009 |
Foust KD, Nurre E, Montgomery CL, Hernandez A, Chan CM, Kaspar BK. Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes. Nature Biotechnology. 27: 59-65. PMID 19098898 DOI: 10.1038/Nbt.1515 |
0.81 |
|
| 2008 |
Kaspar BK. Mesenchymal stem cells as trojan horses for GDNF delivery in ALS. Molecular Therapy : the Journal of the American Society of Gene Therapy. 16: 1905-6. PMID 19023270 DOI: 10.1038/Mt.2008.216 |
0.674 |
|
| 2008 |
Kim ML, Chandrasekharan K, Glass M, Shi S, Stahl MC, Kaspar B, Stanley P, Martin PT. O-fucosylation of muscle agrin determines its ability to cluster acetylcholine receptors. Molecular and Cellular Neurosciences. 39: 452-64. PMID 18775496 DOI: 10.1016/J.Mcn.2008.07.026 |
0.585 |
|
| 2008 |
Dodge JC, Haidet AM, Yang W, Passini MA, Hester M, Clarke J, Roskelley EM, Treleaven CM, Rizo L, Martin H, Kim SH, Kaspar R, Taksir TV, Griffiths DA, Cheng SH, ... Kaspar BK, et al. Delivery of AAV-IGF-1 to the CNS extends survival in ALS mice through modification of aberrant glial cell activity. Molecular Therapy : the Journal of the American Society of Gene Therapy. 16: 1056-64. PMID 18388910 DOI: 10.1038/Mt.2008.60 |
0.807 |
|
| 2008 |
Haidet AM, Rizo L, Handy C, Umapathi P, Eagle A, Shilling C, Boue D, Martin PT, Sahenk Z, Mendell JR, Kaspar BK. Long-term enhancement of skeletal muscle mass and strength by single gene administration of myostatin inhibitors. Proceedings of the National Academy of Sciences of the United States of America. 105: 4318-22. PMID 18334646 DOI: 10.1073/Pnas.0709144105 |
0.603 |
|
| 2008 |
McCrate ME, Kaspar BK. Physical activity and neuroprotection in amyotrophic lateral sclerosis. Neuromolecular Medicine. 10: 108-17. PMID 18286388 DOI: 10.1007/S12017-008-8030-5 |
0.631 |
|
| 2008 |
Miller TM, Smith RA, Kordasiewicz H, Kaspar BK. Gene-targeted therapies for the central nervous system. Archives of Neurology. 65: 447-51. PMID 18268183 DOI: 10.1001/Archneur.65.4.Nnr70007 |
0.63 |
|
| 2008 |
Popkie A, Buescher JL, Miranda CJ, Kaspar BK, Phiel CJ, Doble BW. O1-03-02: Analyzing the endogenous roles of GSK-3 isoforms in APP processing in GSK-3 deficient cells Alzheimer's & Dementia. 4: T112-T112. DOI: 10.1016/J.Jalz.2008.05.230 |
0.581 |
|
| 2007 |
Rodino-Klapac LR, Chicoine LG, Kaspar BK, Mendell JR. Gene therapy for duchenne muscular dystrophy: expectations and challenges. Archives of Neurology. 64: 1236-41. PMID 17846262 DOI: 10.1001/Archneur.64.9.1236 |
0.643 |
|
| 2007 |
Dodge J, Treleaven C, Yang W, Clarke J, Martin H, Handy C, Hester M, Taksir T, Griffiths D, Cheng S, Kaspar B, Shihabuddin L. G.P.11.07 AAV mediated gene transfer of IGF-1 and VEGF to the ventricular system provides significant therapeutic benefit in a mouse model of amyotrophic lateral sclerosis Neuromuscular Disorders. 17: 824-825. DOI: 10.1016/J.Nmd.2007.06.214 |
0.334 |
|
| 2006 |
Suhonen J, Ray J, Blömer U, Gage FH, Kaspar B. Ex vivo and in vivo gene delivery to the brain. Current Protocols in Human Genetics / Editorial Board, Jonathan L. Haines ... [Et Al.]. Unit 13.3. PMID 18428389 DOI: 10.1002/0471142905.Hg1303S51 |
0.672 |
|
| 2006 |
Koerber JT, Maheshri N, Kaspar BK, Schaffer DV. Construction of diverse adeno-associated viral libraries for directed evolution of enhanced gene delivery vehicles. Nature Protocols. 1: 701-6. PMID 17406299 DOI: 10.1038/Nprot.2006.93 |
0.684 |
|
| 2006 |
Miller TM, Kim SH, Yamanaka K, Hester M, Umapathi P, Arnson H, Rizo L, Mendell JR, Gage FH, Cleveland DW, Kaspar BK. Gene transfer demonstrates that muscle is not a primary target for non-cell-autonomous toxicity in familial amyotrophic lateral sclerosis. Proceedings of the National Academy of Sciences of the United States of America. 103: 19546-51. PMID 17164329 DOI: 10.1073/Pnas.0609411103 |
0.817 |
|
| 2006 |
Maheshri N, Koerber JT, Kaspar BK, Schaffer DV. Directed evolution of adeno-associated virus yields enhanced gene delivery vectors. Nature Biotechnology. 24: 198-204. PMID 16429148 DOI: 10.1038/Nbt1182 |
0.695 |
|
| 2006 |
Borrell V, Kaspar BK, Gage FH, Callaway EM. In vivo evidence for radial migration of neurons by long-distance somal translocation in the developing ferret visual cortex. Cerebral Cortex (New York, N.Y. : 1991). 16: 1571-83. PMID 16357334 DOI: 10.1093/Cercor/Bhj094 |
0.633 |
|
| 2006 |
Haenggeli C, Lepore AC, Perez N, Kaspar BK, Rothstein JD. 245. Delivery of IGF-1 in the Spinal Cord of a Mouse Model of ALS after Intraparenchymal Injection of an Adeno-Associated Vector Molecular Therapy. 13: S94. DOI: 10.1016/J.Ymthe.2006.08.272 |
0.697 |
|
| 2006 |
Dodge JC, Passini MA, Clarke J, Grissett L, Kim SH, Wen R, Taksir TV, Griffiths DA, Cheng SH, Kaspar BK, Shihabuddin LS. 1062. Intracerebellar Injection of AAV-IGF-1 Improves Motor Function and Extends Survival in a Mouse Model of Amyotrophic Lateral Sclerosis Molecular Therapy. 13: S407. DOI: 10.1016/J.Ymthe.2006.08.1160 |
0.698 |
|
| 2006 |
Rizo L, Shilling C, Haidet A, Umapathi Umapathi P, Sahenk Z, Mendell JR, Kaspar BK. 914. Recombinant AAV Gene Delivery of Follistatin for Muscle Enhancement in Models of Muscular Dystrophy Molecular Therapy. 13: S352. DOI: 10.1016/J.Ymthe.2006.08.1004 |
0.631 |
|
| 2005 |
Lee GK, Maheshri N, Kaspar B, Schaffer DV. PEG conjugation moderately protects adeno-associated viral vectors against antibody neutralization. Biotechnology and Bioengineering. 92: 24-34. PMID 15937953 DOI: 10.1002/Bit.20562 |
0.664 |
|
| 2005 |
Kaspar BK, Frost LM, Christian L, Umapathi P, Gage FH. Synergy of insulin-like growth factor-1 and exercise in amyotrophic lateral sclerosis. Annals of Neurology. 57: 649-55. PMID 15852403 DOI: 10.1002/Ana.20451 |
0.625 |
|
| 2005 |
Miller TM, Kaspar BK, Kops GJ, Yamanaka K, Christian LJ, Gage FH, Cleveland DW. Virus-delivered small RNA silencing sustains strength in amyotrophic lateral sclerosis. Annals of Neurology. 57: 773-6. PMID 15852369 DOI: 10.1002/Ana.20453 |
0.675 |
|
| 2005 |
Kaspar BK, Roth DM, Lai NC, Drumm JD, Erickson DA, McKirnan MD, Hammond HK. Myocardial gene transfer and long-term expression following intracoronary delivery of adeno-associated virus. The Journal of Gene Medicine. 7: 316-24. PMID 15515115 DOI: 10.1002/Jgm.665 |
0.578 |
|
| 2005 |
Koerber JT, Maheshri N, Kaspar B, Schaffer DV. 1085. Directed Evolution of AAV2 Leads to Improved Viral Function Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.07.632 |
0.553 |
|
| 2005 |
Haenggeli C, Rothstein JD, Kaspar BK. 653. Injection of AAV Directly into the Spinal Cord Increases Levels of Gene Expression Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.07.193 |
0.476 |
|
| 2005 |
Haenggeli C, Kaspar B, Rothstein J. 652. Adeno-Associated Vector Delivery of Gutamate Transporters in an ALS Mouse Model Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.07.192 |
0.444 |
|
| 2005 |
Kaspar BK, Christian L, Frost L, Rothstein JD, Gage FH. 650. AAV-IGF1 and Exercise for Synergistic Therapies in ALS Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.07.190 |
0.433 |
|
| 2004 |
Hsieh J, Aimone JB, Kaspar BK, Kuwabara T, Nakashima K, Gage FH. IGF-I instructs multipotent adult neural progenitor cells to become oligodendrocytes. The Journal of Cell Biology. 164: 111-22. PMID 14709544 DOI: 10.1083/Jcb.200308101 |
0.781 |
|
| 2004 |
Maheshri N, Kaspar B, Schaffer DV. 20. Directed Evolution of AAV2 Yields Novel Capsid Variants Molecular Therapy. 9. DOI: 10.1016/J.Ymthe.2004.05.101 |
0.578 |
|
| 2004 |
Kaspar BK, Vich J, Christian L, Rothsten JD, Gage FH. 44. AAV Retrograde Transport Potential and Therapeutic Approaches for ALS|[ast]| Molecular Therapy. 9. DOI: 10.1016/J.Ymthe.2004.05.066 |
0.413 |
|
| 2003 |
Kaspar BK, Lladó J, Sherkat N, Rothstein JD, Gage FH. Retrograde viral delivery of IGF-1 prolongs survival in a mouse ALS model. Science (New York, N.Y.). 301: 839-42. PMID 12907804 DOI: 10.1126/Science.1086137 |
0.71 |
|
| 2003 |
South SM, Kohno T, Kaspar BK, Hegarty D, Vissel B, Drake CT, Ohata M, Jenab S, Sailer AW, Malkmus S, Masuyama T, Horner P, Bogulavsky J, Gage FH, Yaksh TL, et al. A conditional deletion of the NR1 subunit of the NMDA receptor in adult spinal cord dorsal horn reduces NMDA currents and injury-induced pain. The Journal of Neuroscience : the Official Journal of the Society For Neuroscience. 23: 5031-40. PMID 12832526 DOI: 10.1523/Jneurosci.23-12-05031.2003 |
0.714 |
|
| 2003 |
Lai K, Kaspar BK, Gage FH, Schaffer DV. Sonic hedgehog regulates adult neural progenitor proliferation in vitro and in vivo. Nature Neuroscience. 6: 21-7. PMID 12469128 DOI: 10.1038/Nn983 |
0.692 |
|
| 2002 |
Lie DC, Dziewczapolski G, Willhoite AR, Kaspar BK, Shults CW, Gage FH. The adult substantia nigra contains progenitor cells with neurogenic potential. The Journal of Neuroscience : the Official Journal of the Society For Neuroscience. 22: 6639-49. PMID 12151543 DOI: 10.1523/Jneurosci.22-15-06639.2002 |
0.794 |
|
| 2002 |
Kaspar BK, Vissel B, Bengoechea T, Crone S, Randolph-Moore L, Muller R, Brandon EP, Schaffer D, Verma IM, Lee KF, Heinemann SF, Gage FH. Adeno-associated virus effectively mediates conditional gene modification in the brain. Proceedings of the National Academy of Sciences of the United States of America. 99: 2320-5. PMID 11842206 DOI: 10.1073/Pnas.042678699 |
0.812 |
|
| 2002 |
Kaspar BK, Erickson D, Schaffer D, Hinh L, Gage FH, Peterson DA. Targeted retrograde gene delivery for neuronal protection. Molecular Therapy : the Journal of the American Society of Gene Therapy. 5: 50-6. PMID 11786045 DOI: 10.1006/mthe.2001.0520 |
0.774 |
|
| 1997 |
Snyder RO, Spratt SK, Lagarde C, Bohl D, Kaspar B, Sloan B, Cohen LK, Danos O. Efficient and stable adeno-associated virus-mediated transduction in the skeletal muscle of adult immunocompetent mice Human Gene Therapy. 8: 1891-1900. PMID 9382955 DOI: 10.1089/Hum.1997.8.16-1891 |
0.622 |
|
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