Muscle-directed AAV gene therapy rescues the maple syrup urine disease phenotype in a mouse model-Reference-Cited by-同舟云学术

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Muscle-directed AAV gene therapy rescues the maple syrup urine disease phenotype in a mouse model

Published:2021-09 Issue:1-2 Volume:134 Page:139-146
ISSN:1096-7192
Container-title:Molecular Genetics and Metabolism
language:en
Short-container-title:Molecular Genetics and Metabolism

Author:

Greig Jenny A.,Jennis Matthew,Dandekar Aditya,Chorazeczewski Joanna K.,Smith Melanie K.,Ashley Scott N.,Yan Hanying,Wilson James M.

Publisher

Elsevier BV

Subject

Endocrinology,Genetics,Molecular Biology,Biochemistry,Endocrinology, Diabetes and Metabolism

Reference27 articles.

1. Developing a public health-tracking system for follow-up of newborn screening metabolic conditions: a four-state pilot project structure and initial findings;Hinton;Genet. Med.,2014

2. Inborn errors of metabolism identified via newborn screening: ten-year incidence data and costs of nutritional interventions for research agenda planning;Therrell;Mol. Genet. Metab.,2014

3. Relationship of causative genetic mutations in maple syrup urine disease with their clinical expression;Nellis;Mol. Genet. Metab.,2003

4. Fourteen new mutations of BCKDHA, BCKDHB and DBT genes associated with maple syrup urine disease (MSUD) in Malaysian population;Ali;Mol. Genet. Metab. Rep.,2018

5. Identification of twelve novel mutations in patients with classic and variant forms of maple syrup urine disease;Henneke;Hum. Mutat.,2003

Cited by 10 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Lipid Nanoparticle mRNA Therapy Improves Survival and Reduces Serum Branched-Chain Amino Acids in Mouse Models of Maple Syrup Urine Disease;Human Gene Therapy;2024-08-05

2. Liver gene transfer for metabolite detoxification in inherited metabolic diseases;FEBS Letters;2024-06-17

3. Genotypic and phenotypic spectrum of maple syrup urine disease in Zhejiang of China;QJM: An International Journal of Medicine;2024-06-05

4. Computational structural genomics and clinical evidence suggest BCKDK gain‐of‐function may cause a potentially asymptomatic maple syrup urine disease phenotype;JIMD Reports;2024-04-08

5. Exploration of a Predictive Model for Keloid and Potential Therapeutic Drugs Based on Immune Infiltration and Cuproptosis-Related Genes;Journal of Burn Care & Research;2024-02-09

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