Gene Therapy for Methylmalonic Acidemia: Past, Present, and Future-Reference-Cited by-同舟云学术

Gene Therapy for Methylmalonic Acidemia: Past, Present, and Future

Published:2019-10-01 Issue:10 Volume:30 Page:1236-1244
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Chandler Randy J.¹,Venditti Charles P.¹

Affiliation:

1. Medical Genomics and Metabolic Genetics Branch, National Human Genome Research Institute, National Institutes of Health, Department of Health and Human Services, Bethesda, Maryland.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

https://www.liebertpub.com/doi/pdf/10.1089/hum.2019.113

Reference52 articles.

1. Targeting proximal tubule mitochondrial dysfunction attenuates the renal disease of methylmalonic acidemia

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

3. Methylmalonic acidemia/propionic acidemia – the biochemical presentation and comparing the outcome between liver transplantation versus non-liver transplantation groups

4. Liver Transplantation for Propionic Acidemia and Methylmalonic Acidemia: Perioperative Management and Clinical Outcomes

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