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

• Główni autorzy: Gayoung Jang (Autor), Ha Rim Shin (Autor), Hyo-Sang Do (Autor), Jiyeon Kweon (Autor), Soojin Hwang (Autor), Soyoung Kim (Autor), Sun Hee Heo (Autor), Yongsub Kim (Autor), Beom Hee Lee (Autor)
• Format: Książka
• Wydane: Elsevier, 2023-03-01T00:00:00Z.
• Hasła przedmiotowe: article
• Dostęp online: 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...