Models and Gene Therapies for AAT Deficiency
Overview
Organization: National Heart, Lung, and Blood Institute
Start Date: 08/2021
End Date: 07/2026
Primary Goal: Goal 4: Increase and sustain research to better understand the prevention, pathogenesis, diagnosis, treatment, and management of COPD.
Secondary Goal(s):
Objective(s):
- Help expand, coordinate, and optimize COPD research efforts
- Develop personalized medicine for COPD based on the pathogenesis and clinical heterogeneity
- Promote research that can lead to strategies that help prevent the onset and progression of COPD and improve the quality of COPD care and management
Collaboration: University of Massachusetts Medical School Worcester
Activity Type: Research
Activity Description
This NHLBI-funded research activity is being carried out by University of Massachusetts Medical School Worcester.In this study, we will develop new gene therapy drugs to treat genetic emphysema due to alpha-1 antitrypsin deficiency (AATD), a common genetic disease and form of chronic obstructive pulmonary disease (COPD). We will use advanced gene editing tools to create genetically defined animal models of AATD (known as transgenic animals), both in mice and in ferrets, which are a good model to study lung diseases. In the course of the study, we will use the most cutting-edge tools in gene therapy, gene editing, and immune modulation to treat AATD in both animal models, and determine the best ways to measure the success of gene therapy over time.
Audience
General Audience: Health professionals, Researchers
Focused Audience: n/a
Program Reach: National
Type of Area: N/A
Setting: Workplace
Cost and Funding Sources
This activity was funded through the National Heart, Lung, and Blood Institute.Impact Analysis
Results of program are in progressAdvice or Lessons Learned
In processRelated Links
Contact Information
National Heart, Lung, and Blood Institute
Program Analyst
Bethesda
Maryland