BIH Stem Cell Lecture: Novel AAV variants for gene addition and genome editing in preclinical and clinical applications

Information only available in English.

The lecture is held by Leszek Lisowski, Ph.D, MDA, Childrens's Medical Research Institute, Sydney.

 

About Leszek Lisowski

 Leszek Lisowski's main interest is in genetic engineering of viral vectors for use in gene therapy approaches. He developed novel adeno-associated virus (rAAV) vectors capable of specific integration and transgene expression from safe genomic loci and demonstrated successful gene editing in induced pluripotent stem cells (iPS) and embyonic stem cells. He also performed AAV selection in vivo using xenograft mouse model repopulated with primary human hepatocytes. This led to the identification of the first human-specific rAAV vector with improved resistance to neutralization by human IgG antibodies.