Novel Genome Editing Strategies to Treat Bone Marrow Failure in Patients with Dyskeratosis Congenita
Abstract
A specific set of mutations that impair the maintenance of chromosome integrity cause the fatal early childhood bone marrow failure syndrome dyskeratosis congenita. The standard clinical treatment is bone marrow transplantation. This treatment requires a suitable donor and can have severe side effects due to the rejection of the donor cells by the host. The experiments described in this proposal test genome engineering strategies that would allow to correct the underlying defect of chromosome maintenance in the childrens own blood. If successful, this strategy will allow the reconstitution of the childrens blood with their own bone marrow, circumventing the need for a matched donor and alleviating the risks of transplant rejection.
Document Details
- Document Type
- DoD Grant Award
- Publication Date
- Nov 19, 2019
- Source ID
- W81XWH1910586
Entities
People
- Dirk Hockemeyer
Organizations
- United States Army
- University of California, Berkeley