Adeno-associated virus-mediated gene therapy in a patient with Canavan disease using dual routes of administration and immune modulation

Gene replacement therapy is a rational therapeutic strategy and clinical intervention for neurodegenerative disorders like Canavan disease, a leukodystrophy caused by biallelic mutations in the aspartoacylase (ASPA) gene. We aimed to investigate whether simultaneous intravenous (i.v.) and intracereb...

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Detalles Bibliográficos
Autores principales: Corti, Manuela, Byrne, Barry J., Gessler, Dominic J., Thompson, Grace, Norman, Samantha, Lammers, Jenna, Coleman, Kirsten E., Liberati, Cristina, Elder, Melissa E., Escolar, Maria L., Tuna, Ibrahim S., Mesaros, Clementina, Kleiner, Gary I., Barbouth, Deborah S., Gray-Edwards, Heather L., Clement, Nathalie, Cleaver, Brian D., Gao, Guangping
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2023
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10432950/
https://www.ncbi.nlm.nih.gov/pubmed/37601414
http://dx.doi.org/10.1016/j.omtm.2023.06.001

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10432950/
https://www.ncbi.nlm.nih.gov/pubmed/37601414
http://dx.doi.org/10.1016/j.omtm.2023.06.001

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