Enhanced muscle uptake of chemically optimized miR-23b antisense oligonucleotides as lead compounds for myotonic dystrophy type 1
增强肌肉对化学优化的miR-23b反义寡核苷酸的吸收,使其成为治疗1型强直性肌营养不良症的先导化合物
期刊:American Journal of Human Genetics
影响因子:8.1
doi:10.1016/j.ajhg.2026.01.016
González-Martínez, Irene; Cerro-Herreros, Estefanía; Carrascosa-Sàez, Marc; García-Rey, Andrea; Piqueras-Losilla, Diego; Colom-Rodrigo, Anna; Moreno, Nerea; Chakraborty, Mouli; Huguet-Lachon, Aline; González-Barriga, Anchel; Naldaiz-Gastesi, Neia; Dehesa, Martxel; Díaz-Maqueda, Ana; Barquero, Nuria; Varela, Miguel A; López de Munain, Adolfo; Eritja, Ramon; Gourdon, Geneviève; López-Castel, Arturo; Pérez-Alonso, Manuel; Llamusi, Beatriz; Artero, Rubén