Therapeutic gene correction for Lesch-Nyhan syndrome using CRISPR-mediated base and prime editing

Lesch-Nyhan syndrome (LNS) is inherited as an X-linked recessive genetic disorder caused by mutations in hypoxanthine-guanine phosphoribosyl transferase 1 (HPRT1). Patients with LNS show various clinical phenotypes, including hyperuricemia, gout, devastating behavioral abnormality, intellectual disa...

Descripción completa

Detalles Bibliográficos
Autores principales: Jang, Gayoung, Shin, Ha Rim, Do, Hyo-Sang, Kweon, Jiyeon, Hwang, Soojin, Kim, Soyoung, Heo, Sun Hee, Kim, Yongsub, Lee, Beom Hee
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2023
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9996127/
https://www.ncbi.nlm.nih.gov/pubmed/36910714
http://dx.doi.org/10.1016/j.omtn.2023.02.009

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9996127/
https://www.ncbi.nlm.nih.gov/pubmed/36910714
http://dx.doi.org/10.1016/j.omtn.2023.02.009

Ejemplares similares