Un fármaco de edición genética reduce el colesterol malo un 62 por ciento en un ensayo

28 de mayo de 2026

Reportado por IA

Una terapia experimental llamada VERVE-102 redujo el colesterol LDL hasta en un 62 por ciento tras una sola dosis en un estudio preliminar de seguridad. Los resultados provienen de un ensayo de fase I en el que participaron 35 pacientes con colesterol alto o enfermedad cardiovascular temprana. Los datos se publicaron esta semana en el New England Journal of Medicine.

Los investigadores informaron que el tratamiento parecía seguro, sin efectos secundarios graves, incluso con la dosis más alta. Los participantes experimentaron solo un aumento leve y temporal de las enzimas hepáticas. El fármaco utiliza ARNm encapsulado en nanopartículas para editar el gen PCSK9 en las células hepáticas, lo que reduce de forma permanente la producción de una proteína que aumenta los niveles de LDL.

UT Southwestern researchers identify HELZ2 protein that controls the liver’s release of cholesterol-carrying particles

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Scientists at UT Southwestern Medical Center report they have identified a protein, HELZ2, that acts as a key regulator of how many cholesterol-carrying particles the liver releases into the bloodstream by affecting the gene APOB. The study was published in the American Heart Association journal Circulation and could inform future research into heart disease and fatty liver disease.

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An experimental oral pill called enlicitide lowered LDL cholesterol by about 60% in a large phase three clinical trial, according to results published in The New England Journal of Medicine. The trial, led by Dr. Ann Marie Navar at UT Southwestern Medical Center and sponsored by Merck, involved 2,909 participants mostly already on statins. If approved, the daily pill could improve access to effective cholesterol treatment.

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Un fármaco de edición genética reduce el colesterol malo un 62 por ciento en un ensayo

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