ARTICLE | Emerging Company Profile
Forward compatibility
Universal is using rAAV gene editing to create universal donor stem cell lines
May 2, 2016 7:00 AM UTC
Universal Cells Inc. is using an rAAV-based gene editing platform to create universal pluripotent stem cells for allogeneic cell therapies and transplants. The platform circumvents rejection caused by mismatched human leukocyte antigens or minor histocompatibility antigens by using rAAVs to engineer cells with a single exposed class I HLA molecule: HLA-E.
The company's AAVs edit genes by delivering a single-stranded DNA that contains a gene sequence flanked by homology arms. The homology arms pair with DNA at the desired insertion point, and the new sequence is copied into the genome by homologous recombination. ...