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

• Main Authors: Gayoung Jang (Author), Ha Rim Shin (Author), Hyo-Sang Do (Author), Jiyeon Kweon (Author), Soojin Hwang (Author), Soyoung Kim (Author), Sun Hee Heo (Author), Yongsub Kim (Author), Beom Hee Lee (Author)
• Format: Book
• Published: Elsevier, 2023-03-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...