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

• Egile Nagusiak: Gayoung Jang (Egilea), Ha Rim Shin (Egilea), Hyo-Sang Do (Egilea), Jiyeon Kweon (Egilea), Soojin Hwang (Egilea), Soyoung Kim (Egilea), Sun Hee Heo (Egilea), Yongsub Kim (Egilea), Beom Hee Lee (Egilea)
• Formatua: Liburua
• Argitaratua: Elsevier, 2023-03-01T00:00:00Z.
• Gaiak: article
• Sarrera elektronikoa: 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...