Testing the efficacy of CRISPRi in FSHD2

Budget

•  $1,070,000 USD

Timeline

• 18 Months

Lay Abstract

Investigators: Takako Jones, PhD and Charis L. Himeda, PhD; University of Nevada, Reno School of Medicine

The goal of this proposal is to test the utility of our CRISPR inhibition (CRISPRi) approach as a viable therapeutic strategy for FSHD2 patients. To date, CRISPRi has only been tested in FSHD1 cells. In addition, FSHD2 is caused by a wide variety of mutations in the SMCHD1 gene, suggesting a personalized medicine approach may be necessary to ensure different FSHD2 genetic makeups can be therapeutically silenced. Thus, we will validate and optimize our all-in-one CRISPRi system both in vitro (in FSHD2 muscle cells in culture) and in vivo (in two mouse models of FSHD, including a xenograft mouse containing human FSHD2 patient muscle) using FSHD2 patient-derived cells.