Grant Award Details

A Treatment for Artemis-deficient Severe Combined Immunodeficiency using Non-Viral CRISPR-driven Safe Harbor Transgenesis in Hematopoietic Stem Cells
Grant Number: 
DISC2-14130
Investigator: 
Type: 
PI
Award Value: 
$1,809,372
Status: 
Pre-Active

Grant Application Details

Application Title: 
  • A Treatment for Artemis-deficient Severe Combined Immunodeficiency using Non-Viral CRISPR-driven Safe Harbor Transgenesis in Hematopoietic Stem Cells
Public Abstract: 

Research Objective

A Treatment for Artemis-deficient Severe Combined Immunodeficiency using Non-Viral CRISPR-driven Safe Harbor Transgenesis in Hematopoietic Stem Cells

Impact

We aim to develop a novel genome editing based therapy for Artemis-deficient severe combined immunodeficiency that would improve upon prior gene therapies in efficacy, safety, and scalability.

Major Proposed Activities

  • Discover optimal approach for nonviral targeted integration of a functional DCLRE1C transgene into AAVS1 genomic safe harbor in hematopoietic stem and progenitor cells (HSPCs)
  • Discover optimal approach for generating Artemis-deficient HSPCs and characterize resulting cells
  • Demonstrate ex vivo restoration of Artemis function using Artemis-deficient HSPCs
  • Demonstrate in vivo restoration of Artemis function using Artemis-deficient HSPCs
Statement of Benefit to California: 

Our target disease indication, ART-SCID, carries significant risk of death and long-term comorbidities and disproportionately affects descendants of Navajo and Apache Native Americans, although it can affect individuals of any genetic background. We aim to develop a genome editing therapy in blood stem cells using genome editing that will improve upon prior gene therapies with better efficacy, safety, and scalability, which will improve patient access..