Adeno-Associated Virus Serotype 8 Gene Transfer Rescues a Neonatal Lethal Murine Model of Propionic Acidemia-Reference-Cited by-同舟云学术

Adeno-Associated Virus Serotype 8 Gene Transfer Rescues a Neonatal Lethal Murine Model of Propionic Acidemia

Published:2011-04 Issue:4 Volume:22 Page:477-481
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Chandler Randy J.¹²,Chandrasekaran Suma¹,Carrillo-Carrasco Nuria¹,Senac Julien S.¹,Hofherr Sean E.³,Barry Michael A.⁴,Venditti Charles P.¹

Affiliation:

1. Organic Acid Research Section, Genetics and Molecular Biology Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD 20892.

2. Institute for Biomedical Sciences, The George Washington University, Washington, DC 20037.

3. Department of Laboratory Medicine & Pathology, Mayo Clinic, Rochester, MN 55905.

4. Division of Infectious Diseases, Department of Internal Medicine, Department of Immunology, Translational Immunovirology Program, and Molecular Medicine Program, Mayo Clinic, Rochester, MN 55905.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

http://www.liebertpub.com/doi/pdf/10.1089/hum.2010.164

Reference21 articles.

1. Elevation of 2-methylcitric acid I and II levels in serum, urine, and cerebrospinal fluid of patients with cobalamin deficiency

2. 3-Hydroxypropionate: Significance of -Oxidation of Propionate in Patients with Propionic Acidemia and Methylmalonic Acidemia

3. Isolation and Identification of Methylcitrate, a Major Metabolic Product of Propionate in Patients with Propionic Acidemia

4. Propionic Acidemia: A Rare Cause of Cardiomyopathy

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

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