Cargando…
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...
| Autores principales: | , , |
|---|---|
| Formato: | Texto |
| Lenguaje: | English |
| Publicado: |
BioMed Central
2009
|
| Materias: | |
| 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 |