AAV-mediated gene therapy for metabolic diseases: dosage and reapplication studies in the molybdenum cofactor deficiency model

In a mouse model for molybdenum cofactor deficiency as an example for an inherited metabolic disease we have determined the dosage of recombinant AAV necessary to rescue the lethal deficiency phenotype. We demonstrated long-term expression of different expression cassettes delivered in a chimeric AA...

Descripción completa

Detalles Bibliográficos
Autores principales: Hahnewald, Rita, Wegner, Waja, Reiss, Jochen
Formato: Texto
Lenguaje:English
Publicado: BioMed Central 2009
Materias:
Short Paper
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2702341/
https://www.ncbi.nlm.nih.gov/pubmed/19538746
http://dx.doi.org/10.1186/1479-0556-7-9

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2702341/
https://www.ncbi.nlm.nih.gov/pubmed/19538746
http://dx.doi.org/10.1186/1479-0556-7-9

Ejemplares similares