Year |
Citation |
Score |
2024 |
Mylvara AV, Gibson AL, Gu T, Davidson CD, Incao AA, Melnyk K, Pierre-Jacques D, Cologna SM, Venditti CP, Porter FD, Pavan WJ. Optimization of systemic AAV9 gene therapy in Niemann-Pick disease type C1 mice. Biorxiv : the Preprint Server For Biology. PMID 38895471 DOI: 10.1101/2024.06.07.597901 |
0.395 |
|
2022 |
Yerger J, Cougnoux AC, Abbott CB, Luke R, Clark TS, Cawley NX, Porter FD, Davidson CD. Phenotype assessment for neurodegenerative murine models with ataxia and application to Niemann-Pick disease, type C1. Biology Open. 11. PMID 35452076 DOI: 10.1242/bio.059052 |
0.404 |
|
2021 |
Davidson CD, Gibson AL, Gu T, Baxter LL, Deverman BE, Beadle K, Incao AA, Rodriguez-Gil JL, Fujiwara H, Jiang X, Chandler RJ, Ory DS, Gradinaru V, Venditti CP, Pavan WJ. Improved systemic AAV gene therapy with a neurotrophic capsid in Niemann-Pick disease type C1 mice. Life Science Alliance. 4. PMID 34407999 DOI: 10.26508/lsa.202101040 |
0.408 |
|
2021 |
Rodriguez-Gil JL, Baxter LL, Watkins-Chow DE, Johnson NL, Davidson CD, Carlson SR, Incao AA, Wallom KL, Farhat NY, Platt FM, Dale RK, Porter FD, Pavan WJ. Transcriptome of HPβCD-treated Niemann-pick disease type C1 cells highlights GPNMB as a biomarker for therapeutics. Human Molecular Genetics. PMID 34296265 DOI: 10.1093/hmg/ddab194 |
0.414 |
|
2019 |
Cougnoux A, Fellmeth M, Gu T, Davidson CD, Gibson AL, Pavan WJ, Porter FD. Maternal immune activation modifies the course of Niemann-pick disease, type C1 in a gender specific manner. Molecular Genetics and Metabolism. PMID 31668555 DOI: 10.1016/j.ymgme.2019.10.004 |
0.33 |
|
2019 |
Davidson J, Molitor E, Moores S, Gale SE, Subramanian K, Jiang X, Sidhu R, Kell P, Zhang J, Fujiwara H, Davidson C, Helquist P, Melancon BJ, Grigalunas M, Liu G, et al. 2-Hydroxypropyl-β-cyclodextrin is the active component in a triple combination formulation for treatment of Niemann-Pick C1 disease. Biochimica Et Biophysica Acta. Molecular and Cell Biology of Lipids. PMID 31051283 DOI: 10.1016/J.Bbalip.2019.04.011 |
0.704 |
|
2019 |
Pergande MR, Nguyen TTA, Haney-Ball C, Davidson CD, Cologna SM. Quantitative, label-free proteomics in the symptomatic Niemann-Pick, type C1 mouse model using standard flow liquid chromatography and thermal focusing electrospray ionization. Proteomics. e1800432. PMID 30888112 DOI: 10.1002/Pmic.201800432 |
0.393 |
|
2019 |
Davidson C, Gibson A, Gu T, Ali N, Chandler R, Venditti C, Walkley SU, Pavan W. Improved disease amelioration with combination therapy for Niemann-Pick disease type C1 Molecular Genetics and Metabolism. 126: S46. DOI: 10.1016/J.Ymgme.2018.12.100 |
0.668 |
|
2018 |
Davidson C, Gibson A, Gu T, Chandler R, Deverman B, Gradinaru V, Venditti C, Pavan W. Gene therapy for the treatment of Niemann-Pick disease type C1: Comparison of AAV9 to a novel serotype, AAV-PHP.B Molecular Genetics and Metabolism. 123: S36-S37. DOI: 10.1016/J.Ymgme.2017.12.075 |
0.438 |
|
2017 |
Ory DS, Ottinger EA, Farhat NY, King KA, Jiang X, Weissfeld L, Berry-Kravis E, Davidson CD, Bianconi S, Keener LA, Rao R, Soldatos A, Sidhu R, Walters KA, Xu X, et al. Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological disease progression in Niemann-Pick disease, type C1: a non-randomised, open-label, phase 1-2 trial. Lancet (London, England). PMID 28803710 DOI: 10.1016/S0140-6736(17)31465-4 |
0.648 |
|
2017 |
Yang DS, Stavrides P, Kumar A, Jiang Y, Mohan PS, Ohno M, Dobrenis K, Davidson CD, Saito M, Pawlik M, Huo C, Walkley SU, Nixon RA. Cyclodextrin has conflicting actions on autophagy flux in vivo in brains of normal and Alzheimer model mice. Human Molecular Genetics. PMID 28062666 DOI: 10.1093/Hmg/Ddx001 |
0.624 |
|
2016 |
Walkley SU, Davidson CD, Jacoby J, Marella PD, Ottinger EA, Austin CP, Porter FD, Vite CH, Ory DS. Fostering collaborative research for rare genetic disease: the example of niemann-pick type C disease. Orphanet Journal of Rare Diseases. 11: 161. PMID 27903269 DOI: 10.1186/S13023-016-0540-X |
0.613 |
|
2016 |
Chandler RJ, Williams IM, Gibson AL, Davidson CD, Incao AA, Hubbard BT, Porter FD, Pavan WJ, Venditti CP. Systemic AAV9 gene therapy improves the lifespan of mice with Niemann-Pick disease, type C1. Human Molecular Genetics. PMID 27798114 DOI: 10.1093/Hmg/Ddw367 |
0.427 |
|
2016 |
Davidson CD, Fishman YI, Puskás I, Szemán J, Sohajda T, McCauliff LA, Sikora J, Storch J, Vanier MT, Szente L, Walkley SU, Dobrenis K. Efficacy and ototoxicity of different cyclodextrins in Niemann-Pick C disease. Annals of Clinical and Translational Neurology. 3: 366-80. PMID 27231706 DOI: 10.1002/Acn3.306 |
0.673 |
|
2016 |
Davidson C, Fishman Y, Puskas I, Szeman J, Sohajda T, Sikora J, Vanier MT, Szente L, Walkley SU, Dobrenis K. Different cyclodextrins for the treatment of Niemann-Pick disease type C Molecular Genetics and Metabolism. 117: S38. DOI: 10.1016/J.Ymgme.2015.12.232 |
0.665 |
|
2015 |
Praggastis M, Tortelli B, Zhang J, Fujiwara H, Sidhu R, Chacko A, Chen Z, Chung C, Lieberman AP, Sikora J, Davidson C, Walkley SU, Pipalia NH, Maxfield FR, Schaffer JE, et al. A Murine Niemann-Pick C1 I1061T Knock-In Model Recapitulates the Pathological Features of the Most Prevalent Human Disease Allele. The Journal of Neuroscience : the Official Journal of the Society For Neuroscience. 35: 8091-106. PMID 26019327 DOI: 10.1523/Jneurosci.4173-14.2015 |
0.679 |
|
2015 |
Herbert AS, Davidson C, Kuehne AI, Bakken R, Braigen SZ, Gunn KE, Whelan SP, Brummelkamp TR, Twenhafel NA, Chandran K, Walkley SU, Dye JM. Niemann-pick C1 is essential for ebolavirus replication and pathogenesis in vivo. Mbio. 6: e00565-15. PMID 26015498 DOI: 10.1128/Mbio.00565-15 |
0.632 |
|
2015 |
Vite CH, Bagel JH, Swain GP, Prociuk M, Sikora TU, Stein VM, O'Donnell P, Ruane T, Ward S, Crooks A, Li S, Mauldin E, Stellar S, De Meulder M, Kao ML, ... ... Davidson C, et al. Intracisternal cyclodextrin prevents cerebellar dysfunction and Purkinje cell death in feline Niemann-Pick type C1 disease. Science Translational Medicine. 7: 276ra26. PMID 25717099 DOI: 10.1126/Scitranslmed.3010101 |
0.697 |
|
2015 |
Vite CH, Bagel JH, Swain GP, Prociuk M, Sikora TU, O'Donnell P, Ruane T, Ward S, Crooks A, Li S, Mauldin E, Stein V, Ory DS, Kao ML, Davidson C, et al. Intracisternal cyclodextrin ameliorates neurological dysfunction, increases survival time, and stops Purkinje cell death in feline Niemann–Pick type C1 disease Molecular Genetics and Metabolism. 114: S122. DOI: 10.1016/J.Ymgme.2014.12.280 |
0.347 |
|
2014 |
Davidson C, Jacoby J, Marella P, Recke S, Hadley B, Gallo-Hadley L, Vite C, Walkley SU, Ory DS. SOAR-NPC: A unique collaboration designed to rapidly advance promising therapies to the clinic Molecular Genetics and Metabolism. 111: S34. DOI: 10.1016/J.Ymgme.2013.12.063 |
0.54 |
|
2013 |
Fan M, Sidhu R, Fujiwara H, Tortelli B, Zhang J, Davidson C, Walkley SU, Bagel JH, Vite C, Yanjanin NM, Porter FD, Schaffer JE, Ory DS. Identification of Niemann-Pick C1 disease biomarkers through sphingolipid profiling. Journal of Lipid Research. 54: 2800-14. PMID 23881911 DOI: 10.1194/Jlr.M040618 |
0.673 |
|
2013 |
Pontikis CC, Davidson CD, Walkley SU, Platt FM, Begley DJ. Cyclodextrin alleviates neuronal storage of cholesterol in Niemann-Pick C disease without evidence of detectable blood-brain barrier permeability. Journal of Inherited Metabolic Disease. 36: 491-8. PMID 23412751 DOI: 10.1007/S10545-012-9583-X |
0.61 |
|
2013 |
Xu M, Liu K, Swaroop M, Porter FD, Sidhu R, Firnkes S, Ory DS, Marugan JJ, Xiao J, Southall N, Pavan WJ, Davidson C, Walkley SU, Remaley AT, Baxa U, et al. δ-Tocopherol reduces lipid accumulation in Niemann-Pick type C1 and Wolman cholesterol storage disorders (Journal of Biological Chemistry (2012) 287, (39349-39360)) Journal of Biological Chemistry. 288: 296. DOI: 10.1074/Jbc.A112.357707 |
0.624 |
|
2012 |
Xu M, Liu K, Swaroop M, Porter FD, Sidhu R, Firnkes S, Finkes S, Ory DS, Marugan JJ, Xiao J, Southall N, Pavan WJ, Davidson C, Walkley SU, Remaley AT, et al. δ-Tocopherol reduces lipid accumulation in Niemann-Pick type C1 and Wolman cholesterol storage disorders. The Journal of Biological Chemistry. 287: 39349-60. PMID 23035117 DOI: 10.1074/Jbc.M112.357707 |
0.717 |
|
2012 |
Cluzeau CV, Watkins-Chow DE, Fu R, Borate B, Yanjanin N, Dail MK, Davidson CD, Walkley SU, Ory DS, Wassif CA, Pavan WJ, Porter FD. Microarray expression analysis and identification of serum biomarkers for Niemann-Pick disease, type C1. Human Molecular Genetics. 21: 3632-46. PMID 22619379 DOI: 10.1093/Hmg/Dds193 |
0.64 |
|
2011 |
Strømme P, Dobrenis K, Sillitoe RV, Gulinello M, Ali NF, Davidson C, Micsenyi MC, Stephney G, Ellevog L, Klungland A, Walkley SU. X-linked Angelman-like syndrome caused by Slc9a6 knockout in mice exhibits evidence of endosomal-lysosomal dysfunction. Brain : a Journal of Neurology. 134: 3369-83. PMID 21964919 DOI: 10.1093/Brain/Awr250 |
0.737 |
|
2011 |
Zhou S, Davidson C, McGlynn R, Stephney G, Dobrenis K, Vanier MT, Walkley SU. Endosomal/lysosomal processing of gangliosides affects neuronal cholesterol sequestration in Niemann-Pick disease type C. The American Journal of Pathology. 179: 890-902. PMID 21708114 DOI: 10.1016/J.Ajpath.2011.04.017 |
0.694 |
|
2011 |
Ulatowski L, Parker R, Davidson C, Yanjanin N, Kelley TJ, Corey D, Atkinson J, Porter F, Arai H, Walkley SU, Manor D. Altered vitamin E status in Niemann-Pick type C disease. Journal of Lipid Research. 52: 1400-10. PMID 21550990 DOI: 10.1194/Jlr.M015560 |
0.641 |
|
2010 |
Walkley SU, Sikora J, Micsenyi M, Davidson C, Dobrenis K. Lysosomal compromise and brain dysfunction: examining the role of neuroaxonal dystrophy. Biochemical Society Transactions. 38: 1436-41. PMID 21118103 DOI: 10.1042/Bst0381436 |
0.732 |
|
2010 |
Davidson CD, Walkley SU. Niemann-Pick Type C Disease—Pathophysiology and Future Perspectives for Treatment Us Neurology. 6: 88. DOI: 10.17925/Usn.2010.06.01.88 |
0.569 |
|
2010 |
Walkley S, Davidson C, Ali N, Vanier M. 141. Cyclodextrin treatment not only delays but also reduces established intraneuronal storage in Niemann–Pick type C disease Molecular Genetics and Metabolism. 99: S37. DOI: 10.1016/J.Ymgme.2009.10.158 |
0.667 |
|
2009 |
Davidson CD, Ali NF, Micsenyi MC, Stephney G, Renault S, Dobrenis K, Ory DS, Vanier MT, Walkley SU. Chronic cyclodextrin treatment of murine Niemann-Pick C disease ameliorates neuronal cholesterol and glycosphingolipid storage and disease progression. Plos One. 4: e6951. PMID 19750228 DOI: 10.1371/Journal.Pone.0006951 |
0.767 |
|
2009 |
Davidson C, Walkley SU, Ali N, Stephney G. 37. Chronic cyclodextrin administration ameliorates clinical symptoms and storage accumulation in Niemann-Pick Type C1 mice Molecular Genetics and Metabolism. 96: S20. DOI: 10.1016/J.Ymgme.2008.11.038 |
0.608 |
|
Show low-probability matches. |