Long‐term functional correction of cystathionine β‐synthase deficiency in mice by adeno‐associated viral gene therapy
Author:
Affiliation:
1. Cancer Biology Program Fox Chase Cancer Center Philadelphia Pennsylvania USA
2. Department of Genetic Medicine Weill Cornell Medical College New York New York USA
Funder
National Cancer Institute
National Institute of Diabetes and Digestive and Kidney Diseases
Publisher
Wiley
Subject
Genetics (clinical),Genetics
Link
https://onlinelibrary.wiley.com/doi/pdf/10.1002/jimd.12437
Reference29 articles.
1. Hypermethioninemias of genetic and non-genetic origin: A review
2. Homocystinuria patient and caregiver survey: experiences of diagnosis and patient satisfaction
3. Expression of mutant human cystathionine β-synthase rescues neonatal lethality but not homocystinuria in a mouse model
4. Cystathionine Beta-Synthase Deficiency Causes Fat Loss in Mice
5. Mouse models of cystathionine β‐synthase deficiency reveal significant threshold effects of hyperhomocysteinemia
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