Established and Emerging Nucleic Acid Therapies for Familial Hypercholesterolemia.
Circulation
; 150(9): 724-735, 2024 Aug 27.
Article
en En
| MEDLINE
| ID: mdl-39186530
ABSTRACT
Familial hypercholesterolemia (FH) is a genetic disease that leads to elevated low-density lipoprotein cholesterol levels and risk of coronary heart disease. Current therapeutic options for FH remain relatively limited and only partially effective in both lowering low-density lipoprotein cholesterol and modifying coronary heart disease risk. The unique characteristics of nucleic acid therapies to target the underlying cause of the disease can offer solutions unachievable with conventional medications. DNA- and RNA-based therapeutics have the potential to transform the care of patients with FH. Recent advances are overcoming obstacles to clinical translation of nucleic acid-based medications, including greater stability of the formulations as well as site-specific delivery, making gene-based therapy for FH an alternative approach for treatment of FH.
Palabras clave
Texto completo:
1
Colección:
01-internacional
Base de datos:
MEDLINE
Asunto principal:
Terapia Genética
/
Hiperlipoproteinemia Tipo II
Límite:
Animals
/
Humans
Idioma:
En
Revista:
Circulation
Año:
2024
Tipo del documento:
Article