日期:
2020 年 — 2026 年
2020
2021
2022
2023
2024
2025
2026
影响因子:

CRISPR activation to repair electrocardiogram abnormalities caused by a FLNC truncating variant in mice

利用 CRISPR 激活修复小鼠中由 FLNC 截短变体引起的心电图异常

Cañas-Alvaro, Rodrigo; Lalaguna, Laura; Rubio, Blanca; Ausiello, Antonella; López-Olañeta, Marina; Serrano-Blanco, Rocío F; Ochoa, Juan Pablo; de la Pompa, Jose Luis; Chavez, Alejandro; García-Pavía, Pablo; Lara-Pezzi, Enrique

Overexpression of Wild-Type TMEM43 Improves Cardiac Function in Arrhythmogenic Right Ventricular Cardiomyopathy Type 5.

野生型 TMEM43 的过表达可改善致心律失常性右室心肌病 5 型的心脏功能

Lalaguna Laura, Arévalo-Núñez de Arenas María, López-Olañeta Marina, Villalba-Orero María, Jiménez-Riobóo Rafael J, Gómez-Gaviro María Victoria, Isern Joan, Muñoz-Cánoves Pura, Byrne Barry J, Ochoa Juan Pablo, García-Pavía Pablo, Lara-Pezzi Enrique

Current clinical applications of AAV-mediated gene therapy

目前AAV介导的基因治疗的临床应用

Byrne, Barry J; Flanigan, Kevin M; Matesanz, Susan E; Finkel, Richard S; Waldrop, Megan A; D'Ambrosio, Eleonora S; Johnson, Nicholas E; Smith, Barbara K; Bönnemann, Carsten; Carrig, Sean; Rossano, Joseph W; Greenberg, Barry; Lalaguna, Laura; Lara-Pezzi, Enrique; Subramony, Sub; Corti, Manuela; Mercado-Rodriguez, Claudia; Leon-Astudillo, Carmen; Ahrens-Nicklas, Rebecca; Bharucha-Goebel, Diana; Gao, Guangping; Gessler, Dominic J; Hwu, Wuh-Liang; Chien, Yin-Hsiu; Lee, Ni-Chung; Boye, Sanford L; Boye, Shannon E; George, Lindsey A

Biallelic Loss of Function Variants in Myocardial Zonula Adherens Protein Gene (MYZAP) Cause a Severe Recessive Form of Dilated Cardiomyopathy

心肌黏着带蛋白基因 (MYZAP) 的双等位基因功能缺失变异会导致严重的隐性遗传性扩张型心肌病

Ochoa, Juan Pablo; Lalaguna, Laura; Mirelis, Jesús G; Dominguez, Fernando; Gonzalez-Lopez, Esther; Salas, Clara; Roustan, Gaston; McGurk, Kathryn A; Zheng, Sean L; Barton, Paul J R; Ware, James S; Gómez-Gaviro, María Victoria; Lara-Pezzi, Enrique; Garcia-Pavia, Pablo