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

The postnatal injection of AAV9-FOXG1 rescues corpus callosum agenesis and other brain deficits in the mouse model of FOXG1 syndrome

出生后注射 AAV9-FOXG1 可挽救 FOXG1 综合征小鼠模型中的胼胝体发育不全和其他脑功能障碍

Shin Jeon, Jaein Park, Shibi Likhite, Ji Hwan Moon, Dongjun Shin, Liwen Li, Kathrin C Meyer, Jae W Lee, Soo-Kyung Lee

Novel MECP2 gene therapy is effective in a multicenter study using two mouse models of Rett syndrome and is safe in non-human primates

新型 MECP2 基因疗法在一项使用两种雷特综合征小鼠模型的多中心研究中表现出疗效,且在非人类灵长类动物中安全

Samantha Powers, Shibi Likhite, Kamal K Gadalla, Carlos J Miranda, Amy J Huffenberger, Cassandra Dennys, Kevin D Foust, Pablo Morales, Christopher R Pierson, Federica Rinaldi, Stephanie Perry, Brad Bolon, Nicolas Wein, Stuart Cobb, Brian K Kaspar, Kathrin C Meyer

Early postnatal administration of an AAV9 gene therapy is safe and efficacious in CLN3 disease

出生后早期进行 AAV9 基因治疗对 CLN3 疾病安全有效

Tyler B Johnson, Jon J Brudvig, Shibi Likhite, Melissa A Pratt, Katherine A White, Jacob T Cain, Clarissa D Booth, Derek J Timm, Samantha S Davis, Brandon Meyerink, Ricardo Pineda, Cassandra Dennys-Rivers, Brian K Kaspar, Kathrin Meyer, Jill M Weimer

Prevalence of Neutralizing Antibodies against Adeno-Associated Virus Serotypes 1, 2, and 9 in Non-Injected Latin American Patients with Heart Failure-ANVIAS Study

拉丁美洲非注射性心力衰竭患者中腺相关病毒血清型 1、2 和 9 中和抗体的流行率 - ANVIAS 研究

Julieth A Sierra-Delgado, Shibi Likhite, Paula K Bautista, Sergio A Gómez-Ochoa, Luis E Echeverría, Elizabeth Guío, Clara Vargas, Norma C Serrano, Kathrin C Meyer, Melvin Y Rincon

AAV-based gene therapy ameliorated CNS-specific GPI defect in mouse models

基于 AAV 的基因治疗改善了小鼠模型中 CNS 特异性 GPI 缺陷

Yoshiko Murakami, Saori Umeshita, Kae Imanishi, Yoshichika Yoshioka, Akinori Ninomiya, Takehiko Sunabori, Shibi Likhite, Masato Koike, Kathrin C Meyer, Taroh Kinoshita

In Vitro Modeling as a Tool for Testing Therapeutics for Spinal Muscular Atrophy and IGHMBP2-Related Disorders

体外建模作为脊髓性肌萎缩症和 IGHMBP2 相关疾病治疗方法的测试工具

Julieth Andrea Sierra-Delgado, Shrestha Sinha-Ray, Abuzar Kaleem, Meysam Ganjibakhsh, Mohini Parvate, Samantha Powers, Xiaojin Zhang, Shibi Likhite, Kathrin Meyer

In-depth comparison of Anc80L65 and AAV9 retinal targeting and characterization of cross-reactivity to multiple AAV serotypes in humans

深入比较 Anc80L65 和 AAV9 视网膜靶向性以及对人类多种 AAV 血清型的交叉反应性

Maura K Schwartz, Shibi Likhite, Tatyana A Vetter, Megan C Baird, Vicki McGovern, Andrea Sierra Delgado, Tom Mendel, Arthur Burghes, Kathrin C Meyer

Neurotoxic Astrocytes Directly Converted from Sporadic and Familial ALS Patient Fibroblasts Reveal Signature Diversities and miR-146a Theragnostic Potential in Specific Subtypes

直接由散发性和家族性 ALS 患者成纤维细胞转化而来的神经毒性星形胶质细胞揭示出特征多样性以及 miR-146a 在特定亚型中的诊断潜力

Cátia Gomes, Catarina Sequeira, Shibi Likhite, Cassandra N Dennys, Stephen J Kolb, Pamela J Shaw, Ana R Vaz, Brian K Kaspar, Kathrin Meyer, Dora Brites

Optimization and validation of CAR transduction into human primary NK cells using CRISPR and AAV

使用 CRISPR 和 AAV 对 CAR 转导至人类原代 NK 细胞进行优化和验证

Meisam Naeimi Kararoudi, Shibi Likhite, Ezgi Elmas, Kenta Yamamoto, Maura Schwartz, Kinnari Sorathia, Marcelo de Souza Fernandes Pereira, Yasemin Sezgin, Raymond D Devine, Justin M Lyberger, Gregory K Behbehani, Nitin Chakravarti, Branden S Moriarity, Kathrin Meyer, Dean A Lee

Sex-split analysis of pathology and motor-behavioral outcomes in a mouse model of CLN8-Batten disease reveals an increased disease burden and trajectory in female Cln8mnd mice

对 CLN8-Batten 病小鼠模型中的病理学和运动行为结果进行性别分割分析,结果显示雌性 Cln8mnd 小鼠的疾病负担和发展轨迹增加

Andrew D Holmes, Katherine A White, Melissa A Pratt, Tyler B Johnson, Shibi Likhite, Kathrin Meyer, Jill M Weimer