Efficient Delivery of FMR1 across the Blood Brain Barrier Using AAVphp Construct in Adult FMR1 KO Mice Suggests the Feasibility of Gene Therapy for Fragile X Syndrome

Background Fragile X syndrome (FXS) is the most common inherited cause of intellectual disability and autism. Gene therapy may offer an efficient method to ameliorate the symptoms of this disorder. Methods An AAVphp.eb-hSyn-mFMR1IOS7 vector and an empty control were injected into the tail vein of ad...

Descripción completa

Detalles Bibliográficos
Autores principales: Chadman, Kathryn K., Adayev, Tatyana, Udayan, Aishwarya, Ahmed, Rida, Dai, Chun-Ling, Goodman, Jeffrey H., Meeker, Harry, Dolzhanskaya, Natalia, Velinov, Milen
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9957373/
https://www.ncbi.nlm.nih.gov/pubmed/36833432
http://dx.doi.org/10.3390/genes14020505

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9957373/
https://www.ncbi.nlm.nih.gov/pubmed/36833432
http://dx.doi.org/10.3390/genes14020505

Ejemplares similares