Systemic delivery of AAV-GCDH ameliorates HLD-induced phenotype in a glutaric aciduria type I mouse model-Reference-Cited by-同舟云学术

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Systemic delivery of AAV-GCDH ameliorates HLD-induced phenotype in a glutaric aciduria type I mouse model

Published:2024-09 Issue:3 Volume:32 Page:101276
ISSN:2329-0501
Container-title:Molecular Therapy - Methods & Clinical Development
language:en
Short-container-title:Molecular Therapy - Methods & Clinical Development

Author:

Mateu-Bosch Anna,Segur-Bailach Eulàlia,Muñoz-Moreno Emma,Barallobre María José,Arbonés Maria Lourdes,Gea-Sorlí Sabrina,Tort Frederic,Ribes Antonia,García-Villoria Judit,Fillat Cristina^ORCID

Funder

Generalitat de Catalunya

Espana Ministerio de Ciencia e Innovacion

Instituto de Salud Carlos III

Fundación Ramon Areces

Publisher

Elsevier BV

Reference44 articles.

1. Glutaric aciduria: inherited deficiency of glutaryl-CoA dehydrogenase activity;Goodman;Biochem. Med.,1975

2. Recommendations for diagnosing and managing individuals with glutaric aciduria type 1: Third revision;Boy;J. Inherit. Metab. Dis.,2023

3. Exploring genotype-phenotype correlations in glutaric aciduria type 1;Schuurmans;J. Inherit. Metab. Dis.,2023

4. Glutaryl-CoA dehydrogenase deficiency in Spain: evidence of two groups of patients, genetically, and biochemically distinct;Busquets;Pediatr. Res.,2000

5. Unravelling the complex MRI pattern in glutaric aciduria type I using statistical models-a cohort study in 180 patients;Garbade;J. Inherit. Metab. Dis.,2014

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