Development of Intrathecal AAV9 Gene Therapy for Giant Axonal Neuropathy

An NIH-sponsored phase I clinical trial is underway to test a potential treatment for giant axonal neuropathy (GAN) using viral-mediated GAN gene replacement (https://clinicaltrials.gov/ct2/show/NCT02362438). This trial marks the first instance of intrathecal (IT) adeno-associated viral (AAV) gene t...

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Detalles Bibliográficos
Autores principales: Bailey, Rachel M., Armao, Diane, Nagabhushan Kalburgi, Sahana, Gray, Steven J.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2018
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5948230/
https://www.ncbi.nlm.nih.gov/pubmed/29766026
http://dx.doi.org/10.1016/j.omtm.2018.02.005

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5948230/
https://www.ncbi.nlm.nih.gov/pubmed/29766026
http://dx.doi.org/10.1016/j.omtm.2018.02.005

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