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

Implications of the FDA's new plausible mechanism framework for the development of a personalized in vivo prime editing platform

FDA新的合理机制框架对开发个性化体内先导基因编辑平台的影响

Feierman, Emily R; Whittaker, Madelynn N; Quigley, Aidan; Brooks, Dominique L; McVeigh, Paige; Nan, Angela X; Hsu, Alvin; Said, Hooda; Soliman, Ousamah Younoss; Giovenco, Ryan; Davidson, Beverly L; Alameh, Mohamad-Gabriel; Liu, David R; Wang, Xiao; Musunuru, Kiran; Ahrens-Nicklas, Rebecca C

Co-Opting MBNL-Dependent Alternative Splicing Cassette Exons to Control Gene Therapy in Myotonic Dystrophy.

利用 MBNL 依赖性选择性剪接盒外显子来控制强直性肌营养不良症的基因治疗。

Carrell Samuel T, Carrell Ellie M, Giovenco Ryan, Davidson Beverly L

Temporal single-cell atlas of full-length Huntington's disease mouse model defines stage-specific signatures of corticostriatal dysfunction

全长亨廷顿病小鼠模型的单细胞时间图谱定义了皮质纹状体功能障碍的阶段特异性特征。

Robbins, Ashley B; Ranum, Paul T; Huerta-Ocampo, Icnelia; Kuckyr, Michael; Davidson, Beverly L

Author Correction: AAV-based delivery of RNAi targeting ataxin-2 improves survival and pathology in TDP-43 mice

作者更正:基于腺相关病毒(AAV)的RNAi靶向ataxin-2可改善TDP-43小鼠的存活率和病理状况。

Amado, Defne A; Robbins, Ashley B; Whiteman, Katherine R; Smith, Alicia R; Chillon, Guillem; Chen, Yonghong; Fuller, Joshua A; Patty, Nicholas A; Izda, Aleksandar; Cheng, Congsheng; Nelson, Shareen; Dichter, Abigail I; Mazzoni, Esteban O; Monteys, Alex Mas; Davidson, Beverly L

AAV-based delivery of RNAi targeting ataxin-2 improves survival and pathology in TDP-43 mice.

基于 AAV 的 RNAi 递送靶向 ataxin-2 可改善 TDP-43 小鼠的生存率和病理状况

Amado Defne A, Robbins Ashley B, Whiteman Katherine R, Smith Alicia R, Chillon Guillem, Chen Yonghong, Fuller Joshua A, Patty Nicholas A, Izda Aleksandar, Cheng Congsheng, Nelson Shareen, Dichter Abigail I, Mazzoni Esteban O, Monteys Alex Mas, Davidson Beverly L

The future of gene therapy: Safer vectors, sharper focus: High-profile failures demand deep root cause analysis - but the transformative potential of AAV remains within reach if the field is willing to learn and evolve

基因疗法的未来:更安全的载体,更精准的靶点:备受瞩目的失败案例需要进行深入的根本原因分析——但如果该领域愿意学习和发展,AAV的变革潜力仍然触手可及。

Chamberlain, Jeffrey S; Davidson, Beverly L; George, Lindsey A; Byrne, Barry J; Barrett, David

Current trends in gene therapy to treat inherited disorders of the brain

目前基因疗法治疗遗传性脑部疾病的发展趋势

Matuszek, Zaneta; Brown, Brandon L; Yrigollen, Carolyn M; Keiser, Megan S; Davidson, Beverly L

CASP8 intronic expansion identified by poly-glycine-arginine pathology increases Alzheimer's disease risk

通过聚甘氨酸-精氨酸病理学鉴定的CASP8内含子扩增会增加阿尔茨海默病风险

Nguyen, Lien; Ajredini, Ramadan; Guo, Shu; Romano, Lisa E L; Tomas, Rodrigo F; Bell, Logan R; Ranum, Paul T; Zu, Tao; Bañez Coronel, Monica; Kelley, Chase P; Redding-Ochoa, Javier; Nizamis, Evangelos; Melloni, Alexandra; Connors, Theresa R; Gaona, Angelica; Thangaraju, Kiruphagaran; Pletnikova, Olga; Clark, H Brent; Davidson, Beverly L; Yachnis, Anthony T; Golde, Todd E; Lou, XiangYang; Wang, Eric T; Renton, Alan E; Goate, Alison; Valdmanis, Paul N; Prokop, Stefan; Troncoso, Juan C; Hyman, Bradley T; Ranum, Laura P W

CRISPR-mediated transcriptional activation as a mutation-independent therapeutic strategy for SYNGAP1-related intellectual disability

CRISPR介导的转录激活作为一种不依赖于突变的SYNGAP1相关智力障碍治疗策略

Sichlinger, Laura; Reilly, Molly B; Arora, Sakshi; Zhang, Shuo; Marotta, Nicolas; Rodríguez-Acevedo, Kiara L; Hooks, Marisol; Czarnecki, Kyle S; Winter, Julia J; Waxman, Elisa A; Dungan, Lea V; Hong, Ingie; Araki, Yoichi; Johnson, Richard; Huganir, Richard L; Pavani, Giulia; French, Deborah L; Davidson, Beverly L; Prosser, Benjamin L; Heller, Elizabeth A

Scouring the human Hsp70 network uncovers diverse chaperone safeguards buffering TDP-43 toxicity.

对人类 Hsp70 网络的深入研究揭示了多种分子伴侣保护机制,可以缓冲 TDP-43 的毒性

Barbieri Edward M, Linsenmeier Miriam, Whiteman Katherine R, Cheng Yan, Braganza Sylvanne, Copley Katie E, Miranda-Castrodad Paola, Lewis Brennen, Villafañe Kevin, Amado Defne A, Davidson Beverly L, Shorter James