In vivo base editing of PCSK9 with VERVE-102 for hypercholesterolemia (Registro nro. 22825)
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| Campo de control interno | 02909nam a2200301 4500 |
| 001 - Número de control | |
| control field | ESSALUD |
| 005 - Fecha y hora de la última transacción | |
| Campo de control | 20260817160341.0 |
| 007 - Tipo material - Descripcion fisica - info general | |
| Tipo material - Descripcion fisica - info general | ta |
| 008 - Elementos de Longitud Fija - Información General | |
| Elementos de Longitud Fija - Información | t pe ||||| |||| 00| 0 spa d |
| 040 ## - Origen de la Catalogación | |
| Origen de la Catalogación | BMG |
| 041 ## - Idioma | |
| Idioma | eng |
| 100 ## - Autor | |
| Autor | Vafai, Scott B. |
| Rol del Autor | Autor |
| 9 (RLIN) | 54105 |
| 245 ## - Titulo | |
| Titulo | In vivo base editing of PCSK9 with VERVE-102 for hypercholesterolemia |
| 300 ## - Páginas | |
| Paginación | páginas: 648-659 |
| 520 ## - Resumen | |
| Resumen | Background: Persons carrying loss-of-function variants of proprotein convertase subtilisin–kexin type 9 (PCSK9) have reduced levels of low-density lipoprotein (LDL) cholesterol and fewer atherosclerotic cardiovascular disease events than persons without such variants. VERVE-102 is an investigational base-editing therapy designed to durably inactivate PCSK9 in the liver. Methods: In this phase 1, open-label, single-ascending-dose study, we administered one intravenous infusion of VERVE-102 at one of six doses (ranging from 0.3 to 1.0 mg of total RNA per kilogram of body weight [mg per kilogram]) to adults with heterozygous familial hypercholesterolemia or premature coronary artery disease. VERVE-102 consists of a messenger RNA encoding an adenine base-editor protein and a guide RNA targeting PCSK9, which are encapsulated in a lipid nanoparticle incorporating N-acetylgalactosamine. The objectives were to assess safety and changes in blood PCSK9 protein and LDL cholesterol levels. Results: A total of 35 participants across the six dose cohorts received VERVE-102 and had at least 28 days of follow-up. No dose-limiting toxic effects occurred. Mild-to-moderate infusion-related reactions and transient elevations in alanine aminotransferase levels were observed. Aspiration pneumonitis occurred in a participant with gastroesophageal reflux disease. Dose-dependent mean reductions in the PCSK9 level ranged from 51% at the 0.3-mg-per-kilogram dose to 88% at the 1.0-mg-per-kilogram dose. Corresponding reductions in the LDL cholesterol level ranged from 9% at the 0.3-mg-per-kilogram dose to 62% at the 1.0-mg-per-kilogram dose, with an absolute reduction of 78 mg per deciliter at the highest dose. Reductions appeared to be durable throughout follow-up, which was at least 1 year in 15 participants. Conclusions: One dose of VERVE-102 led to dose-dependent, substantial, and sustained reductions in PCSK9 and LDL cholesterol levels. |
| 650 ## - Temas - Descriptores | |
| Temas - Descriptores | CARDIOLOGÍA GENERAL |
| 9 (RLIN) | 53638 |
| 650 ## - Temas - Descriptores | |
| Temas - Descriptores | EDICIÓN GENÉTICA |
| 9 (RLIN) | 54106 |
| 650 ## - Temas - Descriptores | |
| Temas - Descriptores | GENÉTICA GENERAL |
| 9 (RLIN) | 54107 |
| 650 ## - Temas - Descriptores | |
| Temas - Descriptores | LÍPIDOS |
| 9 (RLIN) | 51326 |
| 700 ## - Autor Personal | |
| Autor Personal | Täubel, Jörg |
| 9 (RLIN) | 54108 |
| 700 ## - Autor Personal | |
| Autor Personal | Ashdown, Thomas |
| 9 (RLIN) | 54109 |
| 700 ## - Autor Personal | |
| Autor Personal | Patel, Riyaz S. |
| 9 (RLIN) | 54110 |
| 700 ## - Autor Personal | |
| Autor Personal | Diamondali, Sadaf |
| 9 (RLIN) | 54111 |
| 700 ## - Autor Personal | |
| Autor Personal | Cegla, Jaimini |
| 9 (RLIN) | 54112 |
| 700 ## - Autor Personal | |
| Autor Personal | Soran, Handrean |
| 9 (RLIN) | 54113 |
| 773 0# - Revista (Relacion con el numero) | |
| Host Biblionumber | 22717 |
| Host Itemnumber | 22647 |
| Ciudad, Editorial | Massachusetts NEJM Group |
| Codigo barras item/ejemplar | NEJM010 |
| Titulo de la Revista | The New England Journal of Medicine |
| Número de control de registro | ESSALUD |
| ISSN | 0028-4793 |
| 942 ## - Elementos de Koha | |
| Tipo de Documento | Artículos |
| Fecha procesamiento | 2026-08-17 |
| Catalogador | SQB |
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