Showing results (21-30 of 28) with videos related to
Sort By:
Pageof 3
You have reached the last page of results.This site can display upto 28 results.
Molecular Therapy : the Journal of the American Society of Gene Therapy|July 23, 2023
Novel MECP2 gene therapy is effective in a multicenter study using two mouse models of Rett syndrome and is safe in non-human primatesSamantha Powers, Shibi Likhite, Kamal K Gadalla, et al.Cell Reports Methods|July 5, 2022
Optimization and validation of CAR transduction into human primary NK cells using CRISPR and AAVMeisam Naeimi Kararoudi, Shibi Likhite, Ezgi Elmas, et al.Molecular Therapy. Nucleic Acids|September 10, 2018
Translating SOD1 Gene Silencing toward the Clinic: A Highly Efficacious, Off-Target-free, and Biomarker-Supported Strategy for fALSTommaso Iannitti, Joseph M Scarrott, Shibi Likhite, et al.Frontiers in Genetics|April 10, 2023
Early postnatal administration of an AAV9 gene therapy is safe and efficacious in CLN3 diseaseTyler B Johnson, Jon J Brudvig, Shibi Likhite, et al.Nature Biotechnology|August 12, 2011
Astrocytes from familial and sporadic ALS patients are toxic to motor neuronsAmanda M Haidet-Phillips, Mark E Hester, Carlos J Miranda, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|July 24, 2019
Gene Therapy Corrects Brain and Behavioral Pathologies in CLN6-Batten DiseaseJacob T Cain, Shibi Likhite, Katherine A White, et al.Nature Communications|July 4, 2025
Targeting microRNA-dependent control of X chromosome inactivation improves the Rett Syndrome phenotypeSong Lou, Rachisan DJiake Tihagam, Urszula N Wasko, et al.The New England Journal of Medicine|November 2, 2017
Single-Dose Gene-Replacement Therapy for Spinal Muscular AtrophyJerry R Mendell, Samiah Al-Zaidy, Richard Shell, et al.Pageof 3