• The present disclosure provides adeno-associated virus (AAV) virions with altered capsid protein, where the AAV virions exhibit greater infectivity of retinal cells compared to wild-type AAV. (justia.com)
  • More recently, his group invented tools for cleavage-free large gene insertion via mining microbial recombination protein, and developed single-cell tracking approach for studying cancer biology and cancer immunology. (stanford.edu)
  • Charcot-Marie Tooth disease type 1A (CMT1A), the major type of CMT, is caused by duplication of peripheral myelin protein 22 ( PMP22 ) gene whose overexpression causes structural and functional abnormalities in myelination. (en-journal.org)
  • The present disclosure further provides methods of delivering a gene product to a retinal cell in an individual, and methods of treating ocular disease. (justia.com)