Disease correction by combined neonatal intracranial AAV and systemic lentiviral gene therapy in Sanfilippo Syndrome type B mice

Mucopolysaccharidosis type IIIB (MPS IIIB) or Sanfilippo Syndrome type B is a lysosomal storage disease resulting from the deficiency of N-acetyl glucosaminidase (NAGLU) activity. We previously showed that intracranial adeno-associated virus (AAV) -based gene therapy results in partial improvements...

Descripción completa

Guardado en:
Detalles Bibliográficos
Autores principales: Heldermon, Coy D., Qin, Elizabeth Y., Ohlemiller, Kevin K., Herzog, Erik D., Brown, Jillian R., Vogler, Carol, Hou, Wei, Orrock, John L., Crawford, Brett E., Sands, Mark S.
Formato: Artigo
Lenguaje:Inglês
Publicado: 2013
Materias:
Article
Acceso en línea:https://ncbi.nlm.nih.gov/pmc/articles/PMC3701029/
https://ncbi.nlm.nih.gov/pubmed/23535899
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/gt.2013.14
Etiquetas: Agregar Etiqueta
Sin Etiquetas, Sea el primero en etiquetar este registro!

Ejemplares similares