Gene Therapy for Parkinson’s Disease: Preclinical Evaluation of Optimally Configured TH:CH1 Fusion for Maximal Dopamine Synthesis

A recent phase I-II, open-label trial of ProSavin, a lentiviral vector delivering the key enzymes in the dopamine biosynthetic pathway to non-dopaminergic striatal neurons, demonstrated safety and improved motor function in parkinsonian patients. However, the magnitude of the effect suggested that o...

Descripción completa

Detalles Bibliográficos
Autores principales: Badin, Romina Aron, Binley, Katie, Van Camp, Nadja, Jan, Caroline, Gourlay, Jeanne, Robert, Camille, Gipchtein, Pauline, Fayard, Audrey, Stewart, Hannah, Ralph, G. Scott, Lad, Yatish, Kelleher, Michelle, Loader, Julie, Hosomi, Koichi, Palfi, Stéphane, Mitrophanous, Kyriacos A., Hantraye, Philippe
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2019
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6685641/
https://www.ncbi.nlm.nih.gov/pubmed/31406701
http://dx.doi.org/10.1016/j.omtm.2019.07.002

Ejemplares similares

Ejemplares similares