In silico analysis of potential off-target sites to gene editing for Mucopolysaccharidosis type I using the CRISPR/Cas9 system: Implications for population-specific treatments

Mucopolysaccharidosis type I (MPS I) is caused by alpha-L-iduronidase deficiency encoded by the IDUA gene. Therapy with CRISPR/Cas9 is being developed for treatment, however a detailed investigation of off-target effects must be performed. This study aims to evaluate possible off-targets for a sgRNA...

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Detalles Bibliográficos
Autores principales: Carneiro, Paola, de Freitas, Martiela Vaz, Matte, Ursula
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2022
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8786118/
https://www.ncbi.nlm.nih.gov/pubmed/35073349
http://dx.doi.org/10.1371/journal.pone.0262299

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8786118/
https://www.ncbi.nlm.nih.gov/pubmed/35073349
http://dx.doi.org/10.1371/journal.pone.0262299

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