Grant Award Details

Overcoming barriers for airway stem cell gene therapy for Cystic Fibrosis
Grant Type: 
Grant Number: 
DISC0-14458
Project Objective: 
  • To develop an efficient and effective approach for editing the defective CFTR receptor in airways basal stem cells from cystic fibrosis patients, as the basis for a future gene therapy candidate.
Investigator: 
Disease Focus: 
Cystic Fibrosis
Respiratory Disorders
Human Stem Cell Use: 
Adult Stem Cell
Award Value: 
$1,472,858
Status: 
Pre-Active

Grant Application Details

Application Title: 
  • Overcoming barriers for airway stem cell gene therapy for Cystic Fibrosis
Public Abstract: 

Research Objective

This research will allow the targeting of airway stem cells for long lived gene therapy for Cystic Fibrosis and for other airway diseases

Impact

We will overcome the barriers to accessing airway basal stem cells for gene correction for Cystic Fibrosis (CF) and use a new gene correction strategy to correct >99% of all genetic changes causing CF

Major Proposed Activities

  • Determine the dosing and timing of detergent linked nanoparticles for accessing the airway stem cells
  • Package the gene correction cargo and perform delivery of this gene correction cargo into the stem cells
  • Perform successful gene correction of the mutated gene in the airway basal stem cells using the gene correction strategy
Statement of Benefit to California: 

Cystic 5brosis is one of the most common genetic disorders in the US. California is one of the U.S. states with the largest numbers of people living with CF at 2,386 people. These patients require lifelong, intense medical care both at home and in the hospital. Gene correction of this disease will have a major impact on these patients and their families and communities. It will also greatly reduce the cost of health care for these patients and for the state of California.

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