Fatal outcomes following onasemnogene abeparvovec in advanced-stage spinal muscular atrophy
CRISPR targeting of SNPs associated with Age-related Macular Degeneration in ARPE-19 cells: a potential model for manipulating the complement system
Low Efficiency of Homology-Independent Targeted Integration for CRISPR/Cas9 Correction in the Vicinity of SLC26A4 c.919–2A>G Variant
Lentiviral vector gene therapy and CFTR modulators show comparable effectiveness in cystic fibrosis rat airway models
Safety, Efficacy, and Immunogenicity of a Novel IgG Degrading Enzyme (KJ103): Results from Two Randomised, Blinded, Phase 1 Clinical Trials
Characterization of brain transduction capability of a BBB-penetrant, peptide-displaying AAV vector in mice, rats and non-human primates
Manu on Lenti G2B Gene Therapy for β-Thalassemia
Efficacy of an AAV vector encoding a thermostable form of glucocerebrosidase in alleviating symptoms in a Gaucher disease mouse model
hInGeTox: A human-based in vitro platform to evaluate lentivirus contribution to genotoxicity
Activated Factor X delivered by Adeno-Associated virus significantly inhibited bleeding and alleviated hemophilia A/B arthropathy in hemophilia mice