Brian K. Kaspar - Publications

Ohio State University Ohio State University, Columbus, Columbus, OH 
Spinal Muscular Atrophy, ALS, CNS Gene Delivery, Gene Therapy

112 high-probability publications. We are testing a new system for linking publications to authors. You can help! If you notice any inaccuracies, please sign in and mark papers as correct or incorrect matches. If you identify any major omissions or other inaccuracies in the publication list, please let us know.

Year Citation  Score
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  0.36
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  0.32
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  0.36
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  0.44
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  0.52
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  0.56
2019 Kaspar B, Thomsen G, Hsieh C, Do J, Solano S, Chu B, Barkho B, Fugere M, Kaufmann P, Foust K, Kaspar A, L'Italien J, Sproule D, Feltner D, Chung W, et al. O.24Biodistribution of onasemnogene abeparvovec (AVXS-101) DNA, mRNA, and SMN protein in human tissue Neuromuscular Disorders. 29. DOI: 10.1016/J.Nmd.2019.06.307  0.56
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  0.92
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  0.92
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  0.32
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  0.56
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  0.56
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  0.92
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  0.92
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  0.92
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  0.92
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  0.92
2016 Boczonadi, Meyer K, Kaspar B, Bartsakoulia M, Bansagi B, Bruni F, Boycott K, Mueller J, Horvath R. Mutations In Glycyl-Trna-Synthetase Impair Mitochondrial Function In Neurons Mitochondrion. DOI: 10.1016/J.Mito.2016.08.010  0.56
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  0.92
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  0.92
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  0.92
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  0.92
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  0.92
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  0.92
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  0.92
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  0.92
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  0.92
2015 Kaspar B. Gene delivery for treating neurological disorders Neuromuscular Disorders. 25. DOI: 10.1016/J.Nmd.2015.06.445  0.56
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  0.92
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  0.92
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  0.92
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  0.92
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  0.36
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  0.92
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  0.92
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  0.92
2014 Lowes LP, Alfano LN, Berry KM, Wolock S, Rumpf RW, Ray W, Meyer A, Jackson J, Firmalan A, Flanigan KM, Kaspar B, Mendell JR. G.O.16 Neuromuscular Disorders. 24: 919-920. DOI: 10.1016/J.Nmd.2014.06.415  0.56
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  0.56
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  0.92
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  0.92
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  0.92
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  0.92
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  0.92
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  0.36
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  0.56
2013 Martin P, Wang C, Camboni M, Miranda C, Yoon JH, Xu R, Zygmunt D, Kaspar B. Active and passive immunization strategies based on the SDPM1 peptide demonstrate pre-clinical efficacy in a mouse model for Alzheimer's disease Alzheimers & Dementia. 9. DOI: 10.1016/J.Jalz.2013.04.168  0.56
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  0.92
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  0.92
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  0.92
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  0.92
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  0.92
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  0.92
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  0.92
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  0.56
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  0.92
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  0.92
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  0.92
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  0.92
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  0.92
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  0.92
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  0.92
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  0.92
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  0.92
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  0.92
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  0.92
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  0.92
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  0.92
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  0.92
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  0.92
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  0.92
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  0.92
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  0.92
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  0.92
2010 Kaspar BK. Gene therapy: Direct viral delivery Encyclopedia of Neuroscience. 633-639. DOI: 10.1016/B978-008045046-9.00012-7  0.92
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  0.92
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  0.92
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  0.92
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  0.92
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  0.92
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  0.92
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  0.92
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  0.92
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  0.92
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  0.92
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  0.92
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  0.92
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  0.92
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  0.92
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  0.92
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  0.56
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  0.92
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  0.92
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  0.92
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  0.92
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  0.92
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  0.92
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  0.92
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  0.92
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  0.92
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  0.64
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  0.56
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  0.72
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  0.92
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  0.64
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  0.72
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  0.92
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  0.92
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  0.92
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  0.92
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  0.92
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  0.92
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  0.92
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