Safety of Direct Intraparenchymal AAVrh.10-Mediated Central Nervous System Gene Therapy for Metachromatic Leukodystrophy

Metachromatic leukodystrophy, a fatal pediatric neurodegenerative lysosomal storage disease caused by mutations in the arylsulfatase A (ARSA) gene, is characterized by intracellular accumulation of sulfatides in the lysosomes of cells of the central nervous system (CNS). In previous studies, we have...

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Bibliografski detalji
Izdano u:Hum Gene Ther
Glavni autori: Rosenberg, Jonathan B., Chen, Alvin, De, Bishnu P., Dyke, Jonathan P., Ballon, Douglas J., Monette, Sebastien, Ricart Arbona, Rodolfo J., Kaminsky, Stephen M., Crystal, Ronald G., Sondhi, Dolan
Format: Artigo
Jezik:Inglês
Izdano: Mary Ann Liebert, Inc., publishers 2021
Teme:
Research Articles
Online pristup:https://ncbi.nlm.nih.gov/pmc/articles/PMC8236561/
https://ncbi.nlm.nih.gov/pubmed/33380277
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2020.269
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