CNS-restricted Transduction and CRISPR/Cas9-mediated Gene Deletion with an Engineered AAV Vector

Gene therapy using recombinant adeno-associated viral (AAV) vectors is emerging as a promising approach to treat central nervous system disorders such as Spinal muscular atrophy, Batten, Parkinson and Alzheimer disease amongst others. A critical remaining challenge for central nervous system-targete...

Descripción completa

Detalles Bibliográficos
Autores principales: Murlidharan, Giridhar, Sakamoto, Kensuke, Rao, Lavanya, Corriher, Travis, Wang, Dan, Gao, Guangping, Sullivan, Patrick, Asokan, Aravind
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2016
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5330941/
https://www.ncbi.nlm.nih.gov/pubmed/27434683
http://dx.doi.org/10.1038/mtna.2016.49

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5330941/
https://www.ncbi.nlm.nih.gov/pubmed/27434683
http://dx.doi.org/10.1038/mtna.2016.49

Ejemplares similares