Comparative Efficacy and Safety of Multiple Routes of Direct CNS Administration of Adeno-Associated Virus Gene Transfer Vector Serotype rh.10 Expressing the Human Arylsulfatase A cDNA to Nonhuman Primates

Metachromatic leukodystrophy (MLD), a fatal disorder caused by deficiency of the lysosomal enzyme arylsulfatase A (ARSA), is associated with an accumulation of sulfatides, causing widespread demyelination in both central and peripheral nervous systems. On the basis of prior studies demonstrating tha...

Cijeli opis

Spremljeno u:
Bibliografski detalji
Izdano u:Hum Gene Ther Clin Dev
Glavni autori: Rosenberg, Jonathan B., Sondhi, Dolan, Rubin, David G., Monette, Sébastien, Chen, Alvin, Cram, Sara, De, Bishnu P., Kaminsky, Stephen M., Sevin, Caroline, Aubourg, Patrick, Crystal, Ronald G.
Format: Artigo
Jezik:Inglês
Izdano: Mary Ann Liebert, Inc. 2014
Teme:
Research Articles
Online pristup:https://ncbi.nlm.nih.gov/pmc/articles/PMC4227442/
https://ncbi.nlm.nih.gov/pubmed/25144894
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/humc.2013.239
Oznake: Dodaj oznaku
Bez oznaka, Budi prvi tko označuje ovaj zapis!

Similar Items