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

Dual AAV gene therapy achieves recovery of hearing and auditory processing in a DFNB16 mouse model

双重AAV基因疗法在DFNB16小鼠模型中实现了听力和听觉处理的恢复

Iranfar, Sepideh; Bagur, Sophie; Felgerolle, Chloé; Cornille, Maxence; Benamer, Najate; Ribeuz, Hélène Le; Giorgi, Marie; Jamali, Sara; Saoudi, Amel; Lecomte, Marie-José; Michel, Vincent; Bathellier, Brice; Safieddine, Saaid

Ineffective behavioral rescue despite partial brain Dp427 restoration by AAV9-U7-mediated exon 51 skipping in mdx52 mice.

尽管通过 AAV9-U7 介导的外显子 51 跳跃在 mdx52 小鼠中部分恢复了脑 Dp427,但行为恢复效果不佳。

Vacca Ophélie, Saoudi Amel, Doisy Mathilde, Phongsavanh Xaysongkhame, Le Coz Olivier, Nagy Cathy, Kuzniar Julia, Vaillend Cyrille, Goyenvalle Aurélie

Regional Expression of Dystrophin Gene Transcripts and Proteins in the Mouse Brain.

小鼠脑内肌营养不良蛋白基因转录本和蛋白质的区域表达。

Tetorou Konstantina, Aghaeipour Artadokht, Ma Shunyi, Gileadi Talia, Saoudi Amel, Perdomo Quinteiro Pablo, Aragón Jorge, van Putten Maaike, Spitali Pietro, Montanez Cecilia, Vaillend Cyrille, Morgan Jennifer E, Montanaro Federica, Muntoni Francesco

Valproic Acid Improves Antisense-Mediated Exon-Skipping Efficacy in mdx Mice.

丙戊酸可提高 mdx 小鼠中反义介导的外显子跳跃效率

Phongsavanh Micky, Bizot Flavien, Saoudi Amel, Gastaldi Cecile, Le Coz Olivier, Tensorer Thomas, Brisebard Elise, Garcia Luis, Goyenvalle Aurélie

Impact of distinct dystrophin gene mutations on behavioral phenotypes of Duchenne muscular dystrophy

不同肌营养不良蛋白基因突变对杜氏肌营养不良症行为表型的影响

Saoudi, Amel; Mitsogiannis, Manuela D; Zarrouki, Faouzi; Fergus, Claire; Stojek, Erwina; Talavera, Silvia; Moore-Frederick, Dervla; Kelly, Vincent P; Goyenvalle, Aurélie; Montanaro, Federica; Muntoni, Francesco; Prenderville, Jack A; Sokolowska, Ewa; Vaillend, Cyrille

Investigating the Impact of Delivery Routes for Exon Skipping Therapies in the CNS of DMD Mouse Models

研究外显子跳跃疗法递送途径对DMD小鼠模型中枢神经系统的影响

Saoudi, Amel; Fergus, Claire; Gileadi, Talia; Montanaro, Federica; Morgan, Jennifer E; Kelly, Vincent P; Tensorer, Thomas; Garcia, Luis; Vaillend, Cyrille; Muntoni, Francesco; Goyenvalle, Aurélie