A novel adeno-associated virus capsid with enhanced neurotropism corrects a lysosomal transmembrane enzyme deficiency

Recombinant adeno-associated viruses (AAVs) are popular in vivo gene transfer vehicles. However, vector doses needed to achieve therapeutic effect are high and some target tissues in the central nervous system remain difficult to transduce. Gene therapy trials using AAV for the treatment of neurolog...

Szczegółowa specyfikacja

Zapisane w:
Opis bibliograficzny
Wydane w:Brain
Główni autorzy: Tordo, Julie, O’Leary, Claire, Antunes, André S L M, Palomar, Nuria, Aldrin-Kirk, Patrick, Basche, Mark, Bennett, Antonette, D’Souza, Zelpha, Gleitz, Hélène, Godwin, Annie, Holley, Rebecca J, Parker, Helen, Liao, Ai Yin, Rouse, Paul, Youshani, Amir Saam, Dridi, Larbi, Martins, Carla, Levade, Thierry, Stacey, Kevin B, Davis, Daniel M, Dyer, Adam, Clément, Nathalie, Björklund, Tomas, Ali, Robin R, Agbandje-McKenna, Mavis, Rahim, Ahad A, Pshezhetsky, Alexey, Waddington, Simon N, Linden, R Michael, Bigger, Brian W, Henckaerts, Els
Format: Artigo
Język:Inglês
Wydane: Oxford University Press 2018
Hasła przedmiotowe:
Original Articles
Dostęp online:https://ncbi.nlm.nih.gov/pmc/articles/PMC6037107/
https://ncbi.nlm.nih.gov/pubmed/29788236
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/brain/awy126
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