CRISPR targeting of SNPs associated with Age-related Macular Degeneration in ARPE-19 cells: a potential model for manipulating the complement system
Lentiviral vector gene therapy and CFTR modulators show comparable effectiveness in cystic fibrosis rat airway models
Systemic Delivery of Full-Length Dystrophin in DMD Mice
Biodistribution of Lentiviral Transduced Adipose-Derived Stem Cells for “Ex-vivo” Regional Gene Therapy for Bone Repair
HIV-1 sequences in lentiviral vector genomes can be substantially reduced without compromising transduction efficiency