About the Event
Genome Editing of Stem Cells
A Next Generation of Curative Therapeutics
Genome editing is the mechanism by which one can change the DNA sequence of a cell with nucleotide precision. This precision can be utilized as a powerful research tool but also opens up a new approach to curing patients of disease. In this talk, Matt Porteus will discuss the advances that have been made in developing genome editing of somatic cells to treat disease. In addition, the precision and new found efficiency in the process has raised concerns about the potential application to create heritable (germline) modifications in the genome. Porteus will also will discuss some of the thoughts surrounding that issue.
About the Speaker
He was the first to demonstrate that gene correction could be achieved in human cells at frequencies that were high enough to potentially cure patients. More recently his group has made key discoveries on how to utilize the CRISPR/Cas9 system in getting high frequencies of genome editing in primary human T-cells and CD34+ hematopoietic stem and progenitor cells. He is considered one of the pioneers and founders of the field of genome editing.
Porteus currently practices on the Pediatric Hematopoietic Stem Cell Transplant service at the Lucille Packard Children’s Hospital at Stanford and runs a research lab in the Division of Stem Cell Transplantation and Regenerative Medicine, Hematology/Oncology and Human Gene Therapy, which is focused on developing genome editing of stem cells as therapy for multiple genetic and non-genetic diseases.
Administratively, he is the Associate Director of the Medical Scientist Training Program, Pediatric Hematology/Oncology Fellowship program, and the developing Center for Definitive and Curative Medicine at Stanford.
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