Alpha-1-Antitrypsin Promoter Improves the Efficacy of an Adeno-Associated Virus Vector for the Treatment of Mitochondrial Neurogastrointestinal Encephalomyopathy-Reference-Cited by-同舟云学术

Alpha-1-Antitrypsin Promoter Improves the Efficacy of an Adeno-Associated Virus Vector for the Treatment of Mitochondrial Neurogastrointestinal Encephalomyopathy

Published:2019-08 Issue:8 Volume:30 Page:985-998
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Cabrera-Pérez Raquel¹²,Vila-Julià Ferran¹²,Hirano Michio³,Mingozzi Federico⁴⁵,Torres-Torronteras Javier¹²,Martí Ramon¹²

Affiliation:

1. Research Group on Neuromuscular and Mitochondrial Diseases, Vall d'Hebron Research Institute, Universitat Autònoma de Barcelona, Barcelona, Catalonia, Spain; Paris, France.

2. Biomedical Network Research Centre on Rare Diseases, Instituto de Salud Carlos III, Madrid, Spain; Paris, France.

3. Department of Neurology, H. Houston Merritt Neuromuscular Research Center, Columbia University Medical Center, New York, New York; Paris, France.

4. Genethon and INSERM U951, Evry, France; Paris, France.

5. University Pierre and Marie Curie, Paris, France.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

https://www.liebertpub.com/doi/pdf/10.1089/hum.2018.217

Reference54 articles.

1. Thymidine Phosphorylase Gene Mutations in MNGIE, a Human Mitochondrial Disorder