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

AAVrh74.tMCK.NT-3 Surrogate Gene Therapy in a Mouse Model of CMT2A

在 CMT2A 小鼠模型中应用 AAVrh74.tMCK.NT-3 替代基因疗法

Ozes, Burcak; Tong, Lingying; Moss, Kyle; Myers, Morgan; Ndengabaganizi, Israel; Sahenk, Zarife

AAV1.NT3 gene therapy mitigates the severity of autoimmune encephalomyelitis in the mouse model for multiple sclerosis

AAV1.NT3基因疗法可减轻多发性硬化症小鼠模型中自身免疫性脑脊髓炎的严重程度

Lingying Tong ,Burcak Ozes ,Kyle Moss ,Morgan Myers ,Zayed Attia ,Tatyana A Vetter ,Bruce D Trapp ,Zarife Sahenk

Validity and Reliability of Clinical and Patient-Reported Outcomes in Multisystem Proteinopathy 1

多系统蛋白病1中临床和患者报告结局的有效性和可靠性

Alfano, Lindsay N; Iammarino, Megan A; Reash, Natalie F; Lowes, Linda P; Pietruszewski, Lindsay; Adderley, Kathleen; Humphrey, Lauren; Knight, Audrey B; Steiner, Christopher L; Smith, Melissa A; Sahenk, Zarife; Connolly, Anne M; Almomen, Momen; D'Ambrosio, Eleonora S; Peck, Nathan; Peck, Allison

AAV1.tMCK.NT-3 gene therapy improves phenotype in Sh3tc2(-/-) mouse model of Charcot-Marie-Tooth Type 4C

AAV1.tMCK.NT-3基因疗法改善了夏科-马里-图斯病4C型Sh3tc2(-/-)小鼠模型的表型

Ozes, Burcak; Tong, Lingying; Moss, Kyle; Myers, Morgan; Morrison, Lilye; Attia, Zayed; Sahenk, Zarife

AAV1.NT-3 gene therapy in the SOD1KO mouse model of accelerated sarcopenia

AAV1.NT-3 基因治疗加速性肌肉减少症 SOD1KO 小鼠模型

Lingying Tong, Burcak Ozes, Kyle Moss, Morgan Myers, Alicia Ridgley, Zarife Sahenk

Expression of SRP-9001 dystrophin and stabilization of motor function up to 2 years post-treatment with delandistrogene moxeparvovec gene therapy in individuals with Duchenne muscular dystrophy

在接受 delandistrogene moxeparvovec 基因治疗的杜氏肌营养不良症患者中,SRP-9001 肌营养不良蛋白的表达和运动功能的稳定可持续长达 2 年。

Mendell, Jerry R; Shieh, Perry B; McDonald, Craig M; Sahenk, Zarife; Lehman, Kelly J; Lowes, Linda P; Reash, Natalie F; Iammarino, Megan A; Alfano, Lindsay N; Sabo, Brenna; Woods, Jeremy D; Skura, Christy L; Mao, Howard C; Staudt, Loretta A; Griffin, Danielle A; Lewis, Sarah; Wang, Shufang; Potter, Rachael A; Singh, Teji; Rodino-Klapac, Louise R

Clinical Classification of Variants in the Valosin-Containing Protein Gene Associated With Multisystem Proteinopathy

与多系统蛋白病相关的含缬氨酸蛋白基因变异的临床分类

Schiava, Marianela; Ikenaga, Chiseko; Topf, Ana; Caballero-Ávila, Marta; Chou, Tsui-Fen; Li, Shan; Wang, Feng; Daw, Jil; Stojkovic, Tanya; Villar-Quiles, Rocio; Nishino, Ichizo; Inoue, Michio; Nishimori, Yukako; Saito, Yoshihiko; Katsuno, Masahisa; Noda, Seiya; Ito, Chihiro; Otsuka, Mieko; Nahir, Sruthi; Manousakis, Georgios; Walk, David; Quinn, Colin; Alfano, Lindsay; Sahenk, Zarife; Tasca, Giorgio; Monforte, Mauro; Sabatelli, Mario; Bisogni, Giulia; Oldfors, Anders; Rydeliu, Anna; Pal, Endre; Paradas, Carmen; Velez, Beatriz; De Bleecker, Jan L; Farugia, Maria Elena; Longman, Cheryl; Harms, Matthew B; Ralston, Stuart; Zanoteli, Edmar; Macedo Serafim da Silva, Andre; Sotoca, Javier; Juntas-Morales, Raul; Bevilacqua, Jorge; Balart, Mireya; Talbot, Stuart; Straub, Volker; Guglieri, Michela; Marini-Bettolo, Chiara; Diaz-Manera, Jordi; Weihl, Conrad Chris

AAV1.NT-3 gene therapy prevents age-related sarcopenia

AAV1.NT-3基因治疗可预防与年龄相关的肌肉减少症

Burcak Ozes, Lingying Tong, Morgan Myers, Kyle Moss, Alicia Ridgley, Zarife Sahenk

Systemic delivery of AAVrh74.tMCK.hCAPN3 rescues the phenotype in a mouse model for LGMD2A/R1

系统性递送 AAVrh74.tMCK.hCAPN3 可挽救 LGMD2A/R1 小鼠模型的表型

Sahenk, Zarife; Ozes, Burcak; Murrey, Darren; Myers, Morgan; Moss, Kyle; Yalvac, Mehmet E; Ridgley, Alicia; Chen, Lei; Mendell, Jerry R

AAV1.NT-3 gene therapy in a CMT2D model: phenotypic improvements in Gars(P278KY/+) mice

在 CMT2D 模型中应用 AAV1.NT-3 基因疗法:Gars(P278KY/+) 小鼠表型改善

Ozes, Burcak; Moss, Kyle; Myers, Morgan; Ridgley, Alicia; Chen, Lei; Murrey, Darren; Sahenk, Zarife