VERVE-101, a CRISPR base-editing therapy designed to permanently inactivate hepatic PCSK9 and reduce LDL-cholesterol.

Hooper, Amanda J; Tang, Xuan L; Burnett, John R

Hooper, Amanda J; Tang, Xuan L; Burnett, John R.

Afiliación

Hooper AJ; Department of Clinical Biochemistry, PathWest Laboratory Medicine WA, Royal Perth Hospital & Fiona Stanley Hospital Network, Perth, Western Australia, Australia.
Tang XL; School of Medicine, University of Western Australia, Perth, Western Australia, Australia.
Burnett JR; Department of Clinical Biochemistry, PathWest Laboratory Medicine WA, Royal Perth Hospital & Fiona Stanley Hospital Network, Perth, Western Australia, Australia.

Expert Opin Investig Drugs ; 33(8): 753-756, 2024 Aug.

Article en En | MEDLINE | ID: mdl-38878270

Asunto(s)

LDL-Colesterol; Edición Génica; Proproteína Convertasa 9; Proproteína Convertasa 9/genética; Humanos; LDL-Colesterol/sangre; Animales; Edición Génica/métodos; Terapia Genética/métodos; Sistemas CRISPR-Cas; Hígado

Palabras clave

Base editing; CRISPR; LDL; LDL-cholesterol; PCSK9; PCSK9 inhibitor; VERVE-101; VERVE-102

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Texto completo: 1 Base de datos: MEDLINE Asunto principal: Proproteína Convertasa 9 / Edición Génica / LDL-Colesterol Idioma: En Revista: Expert Opin Investig Drugs / Expert opin. investig. drugs / Expert opinion on investigational drugs Asunto de la revista: TERAPIA POR MEDICAMENTOS Año: 2024 Tipo del documento: Article

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