Expression of a large coding sequence: Gene therapy vectors for Ataxia Telangiectasia

Ataxia telangiectasia is a monogenetic disorder caused by mutations in the ATM gene. Its encoded protein kinase ATM plays a fundamental role in DNA repair of double strand breaks (DSBs). Impaired function of this kinase leads to a multisystemic disorder including immunodeficiency, progressive cerebe...

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Detalles Bibliográficos
Autores principales: Hirch, Tanja, Brander, Nadine, Schenk, Franziska, Pöllmann, Simon J., Reichenbach, Janine, Schubert, Ralf, Modlich, Ute
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10632516/
https://www.ncbi.nlm.nih.gov/pubmed/37938627
http://dx.doi.org/10.1038/s41598-023-46332-4

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10632516/
https://www.ncbi.nlm.nih.gov/pubmed/37938627
http://dx.doi.org/10.1038/s41598-023-46332-4

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