Modeling Glutaric Aciduria Type I in human neuroblastoma cells recapitulates neuronal damage that can be rescued by gene replacement-Reference-Cited by-同舟云学术

Modeling Glutaric Aciduria Type I in human neuroblastoma cells recapitulates neuronal damage that can be rescued by gene replacement

Published:2023-11-20 Issue:1-2 Volume:31 Page:12-18
ISSN:0969-7128
Container-title:Gene Therapy
language:en
Short-container-title:Gene Ther

Author:

Mateu-Bosch A.,Segur-Bailach E.,García-Villoria J.,Gea-Sorlí S.,Ruiz I.,del Rey J.,Camps J.,Guitart-Mampel M.,Garrabou G.,Tort F.,Ribes A.,Fillat C.^ORCID

Publisher

Springer Science and Business Media LLC

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

https://www.nature.com/articles/s41434-023-00428-8.pdf

Reference33 articles.

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3. Yuan Y, Dimitrov B, Boy N, Gleich F, Zielonka M, Kölker S. Phenotypic prediction in glutaric aciduria type 1 combining in silico and in vitro modeling with real-world data. J Inherit Metab Dis. 2023;46:391–405.

4. Kölker S, Sauer SW, Hoffmann GF, Müller I, Morath MA, Okun JG. Pathogenesis of CNS involvement in disorders of amino and organic acid metabolism. J Inherit Metab Dis. 2008;31:194–204.

5. Kölker S, Garbade SF, Greenberg CR, Leonard JV, Saudubray JM, Ribes A, et al. Natural history, outcome, and treatment efficacy in children and adults with glutaryl-CoA dehydrogenase deficiency. Pediatr Res. 2006;59:840–7.

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