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

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...

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Bibliographic Details
Main Authors: Gayoung Jang, Ha Rim Shin, Hyo-Sang Do, Jiyeon Kweon, Soojin Hwang, Soyoung Kim, Sun Hee Heo, Yongsub Kim, Beom Hee Lee
Format: Article
Language:English
Published: Elsevier 2023-03-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
MT: RNA/DNA Editing
Lesch-Nyhan syndrome
LNS
HPRT1, CRISPR-Cas
gene correction
base editing
Online Access:http://www.sciencedirect.com/science/article/pii/S216225312300029X

Internet

http://www.sciencedirect.com/science/article/pii/S216225312300029X

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