Wird geladen...

Long-term Rescue of a Lethal Murine Model of Methylmalonic Acidemia Using Adeno associated Viral Gene Therapy

Methylmalonic acidemia (MMA) is an organic acidemia caused by deficient activity of the mitochondrial enzyme methylmalonyl-CoA mutase (MUT). This disorder is associated with lethal metabolic instability and carries a poor prognosis for long-term survival. A murine model of MMA that replicates a seve...

Ausführliche Beschreibung

Gespeichert in:
Bibliographische Detailangaben
Hauptverfasser: Chandler, Randy J, Venditti, Charles P
Format: Artigo
Sprache:Inglês
Veröffentlicht: Nature Publishing Group 2009
Schlagworte:
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC2839224/
https://ncbi.nlm.nih.gov/pubmed/19861951
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2009.247
Tags: Tag hinzufügen
Keine Tags, Fügen Sie den ersten Tag hinzu!