Abstract
Targeted genome and epigenome editing technologies have recently emerged as important tools for biomedical research and as potential reagents for therapies of gene-based diseases. This talk will focus on our recent work on both improving and defining the genome wide specificities of clustered regularly interspaced short palindromic repeat (CRISPR)-Cas nucleases, a robust platform for introducing targeted genome sequence alterations. The talk will also briefly describe the creation and validation of new technologies for modifying specific epigenomic marks on histones and DNA that can be used to induce targeted alterations in endogenous human gene expression. Taken together, these methodologies provide transformative tools for understanding human biology and offer promising pathways forward for developing new classes of therapeutics based on targeted alterations of gene sequence and/or gene expression.
Joung:Editas Medicine: Consultancy, Equity Ownership, Other, Patents & Royalties; Transposagen Biopharmaceuticals: Equity Ownership, Membership on an entity's Board of Directors or advisory committees, Patents & Royalties; Horizon Discovery: Consultancy, Membership on an entity's Board of Directors or advisory committees.
Author notes
Asterisk with author names denotes non-ASH members.
