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

Published in:	Molecular Therapy: Nucleic Acids
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
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