Exploiting retroelements for targeted gene insertion
Michael received an A.B. from Harvard College in human developmental and regenerative biology in 2014. In May 2019 he completed his PhD in clinical neurosciences from the University of Cambridge. In his doctoral work, Michael studied the ways in which glial stem cells in the CNS change with ageing. His main focus was understanding how the ageing brain environment limits the regenerative capacity of the aged stem cell.
Advances in genome engineering have enabled precision gene editing, but to date, there is no available tool to seamlessly replace DNA in the postnatal human central nervous system (CNS). One potential solution to this problem is to repurpose retroelements, an abundant and diverse class of naturally occurring genetic elements that efficiently insert large amounts of genetic material into their host— retroelements are so efficient that these insertion events compose over half of the human genome. My goal is to re-engineer this highly effective gene insertion machinery to mediate large-scale, site-specific genomic rearrangements in the human CNS. Ultimately, a technology that efficiently inserts DNA into specific genomic loci could be used to rapidly study the effects of autism-associated genomic mutations and may eventually be harnessed to reverse genomic mutations in patients with the disorder.
Keywords: Genome engineering, gene therapy, delivery