Gene therapy in a murine model of methylmalonic acidemia using rAAV9-mediated gene delivery

Robust cardiomyocyte-specific gene expression following systemic injection of AAV: in vivo gene delivery follows a Poisson distribution

(2010) K-M R Prasad et al.   GENE THERAPY

Liver-Directed Recombinant Adeno-Associated Viral Gene Delivery Rescues a Lethal Mouse Model of Methylmalonic Acidemia and Provides Long-Term Phenotypic Correction

(2010) Nuria Carrillo-Carrasco et al.   HUMAN GENE THERAPY

Combined Paracrine and Endocrine AAV9 mediated Expression of Hepatocyte Growth Factor for the Treatment of Renal Fibrosis

(2010) Stephanie Schievenbusch et al.   MOLECULAR THERAPY

Long-term outcome in methylmalonic aciduria: A series of 30 French patients

(2009) M.A. Cosson et al.   MOLECULAR GENETICS AND METABOLISM

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

(2009) Randy J Chandler et al.   MOLECULAR THERAPY

Mitochondrial dysfunction in mut methylmalonic acidemia

(2008) Randy J. Chandler et al.   FASEB JOURNAL

A Single Intravenous Injection of Adeno-associated Virus Serotype-9 Leads to Whole Body Skeletal Muscle Transduction in Dogs

(2008) Yongping Yue et al.   MOLECULAR THERAPY

Analysis of AAV Serotypes 1–9 Mediated Gene Expression and Tropism in Mice After Systemic Injection

(2008) Carmela Zincarelli et al.   MOLECULAR THERAPY

Combined liver–kidney transplant for the management of methylmalonic aciduria: A case report and review of the literature

(2007) Peter J. Mc Guire et al.   MOLECULAR GENETICS AND METABOLISM