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

AAV9 gene therapy optimization for SMARD1/CMT2S: safety and long-term efficacy comparison of two vectors in a SMARD1 preclinical model

针对SMARD1/CMT2S的AAV9基因治疗优化:在SMARD1临床前模型中比较两种载体的安全性和长期疗效

Pagliari, Elisa; Anastasia, Alessia; Bellandi, Floriana; Garbellini, Manuela; Ongaro, Jessica; Taiana, Michela; Comi, Giacomo P; Ottoboni, Linda; Sierra-Delgado, Julieth Andrea; Likhite, Shibi; Meyer, Kathrin C; Nizzardo, Monica; Corti, Stefania P

Targeting microRNA-dependent control of X chromosome inactivation improves the Rett Syndrome phenotype

靶向microRNA依赖的X染色体失活调控可改善雷特综合征表型

Lou, Song; DJiake Tihagam, Rachisan; Wasko, Urszula N; Equbal, Zaffar; Venkatesan, Sanjay; Braczyk, Klaudia; Przanowski, Piotr; Il Koo, Bon; Saltani, Ilyas; Singh, Arjun Tushir; Likhite, Shibi; Powers, Samantha; Souza, George M P R; Maxwell, Robert A; Yu, Jun; Zhu, Lihua J; Beenhakker, Mark; Abbott, Stephen B G; Lu, Zhipeng; Green, Michael R; Meyer, Kathrin C; Tushir-Singh, Jogender; Bhatnagar, Sanchita

The common HAQ STING allele prevents clinical penetrance of COPA syndrome.

常见的 HAQ STING 等位基因可阻止 COPA 综合征的临床表现

Simchoni Noa, Koide Shogo, Likhite Maryel, Kuchitsu Yoshihiko, Kadirvel Senkottuvelan, Law Christopher S, Elicker Brett M, Kurra Santosh, Wong Margaret Mei-Kay, Yuan Bo, Grossi Alice, Laxer Ronald M, Volpi Stefano, Dissanayake Dilan, Taguchi Tomohiko, Beck David B, Vogel Tiphanie P, Shum Anthony K

Adapting the WHO BeSD COVID-19 Survey to Examine Behavioral and Social Drivers of Vaccine Uptake Among Transgender, Intersex, and Disability Communities in India

调整世卫组织 BeSD COVID-19 调查,以研究印度跨性别者、双性人和残疾人群体疫苗接种的行为和社会驱动因素

Lavalekar, Eesha; D'souza, Sharin; Raghuram, Harikeerthan; Dongare, Namdeo; Khan, Mohammed A; Likhite, Chaitanya; Mahajan, Gauri; Chowdhury, Pabitra; Shaikh, Aqsa; Bandewar, Sunita Sheel; Singh, Satendra; Bhan, Anant

DDEL-14. Optimizing dose interval and formulation of neural stem cell-delivered Oncolytic adenovirus therapy for high-grade glioma

DDEL-14. 优化用于治疗高级别胶质瘤的神经干细胞溶瘤腺病毒疗法的给药间隔和制剂

McCleary, Nicola; Duncan, Eilidh M; Stewart, Fiona; Francis, Jill J; Zewde, Yared Z; Russell, Sarah G; Quigley, Rachel; Strivens, Edward; See, Rhiann Sue; Kwan, Heather; Parker, Ashleigh F; Szoeke, Cassandra; Gawryluk, Jodie; Taneri, P E; Likhite, Nathalie; Adossi, Dedenyo; Oot, Lesley; Dain, Anne Sophie Le; Tharaney, Manisha; Nanama, Simeon; Contreras, Gisselle; Flores, Linda; Lara, Jackie; McDonald, Marisa; Mooney, Rachael; Lesniak, Maciej; Portnow, Jana; Aboody, Karen

Gene replacement therapy for Piga GPI-anchor deficiency in the developing nervous system

针对发育中神经系统Piga GPI锚定缺陷的基因替代疗法

Watts, Jennifer L; Likhite, Shibi; Murakami, Yoshiko; Kinoshita, Taroh; Meyer, Kathrin; Stottmann, Rolf W

Shared and distinct mechanisms of UBA1 inactivation across different diseases

不同疾病中 UBA1 失活的共同机制和不同机制

Jason C Collins, Samuel J Magaziner, Maya English, Bakar Hassan, Xiang Chen, Nicholas Balanda, Meghan Anderson, Athena Lam, Sebastian Fernandez-Pol, Bernice Kwong, Peter L Greenberg, Benjamin Terrier, Mary E Likhite, Olivier Kosmider, Yan Wang, Nadine L Samara, Kylie J Walters, David B Beck #, Achim

Combination AAV therapy with galectin-1 and SOD1 downregulation demonstrates superior therapeutic effect in a severe ALS mouse model

AAV 联合 galectin-1 和 SOD1 下调疗法在重度 ALS 小鼠模型中表现出优异的治疗效果

Megan C Baird, Shibi B Likhite, Tatyana A Vetter, Joseph R Caporale, Holly B Girard, Florence S Roussel, Abigail E Howard, Maura K Schwartz, Addison R Reed, Abuzar Kaleem, Xiaojin Zhang, Kathrin C Meyer

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