Alisha Gruntman
University of Massachusetts Chan Medical School, MA·Biomedical Engineering
Alpha-1 antitrypsin deficiency (AATD) is an inherited condition in which the body lacks a protective protein called AAT. Testing gene therapies and genome-editing treatments for it requires animals that develop disease like human patients.
- Fields
Biomedical Engineering
Physiology
Medicine & Clinical Research
- Topics
alpha-1 antitrypsin deficiency
gene therapy
genome editing
- Grants
- 1 known grant, through Jul 2031
What they work on
- Breed and maintain AAT-Null-PiZ ferrets, AAT-Null and AAT-Null-PiZ mice, and wild-type mice as models of alpha-1 antitrypsin deficiency (AATD) and supply them to the program's three research projects.
- Provide gene therapy vector dosing, sample collection from living animals and necropsy for all projects testing AATD treatments.
- Characterize lung responses to gene therapy and genome editing in mouse and ferret AATD models using pulmonary function testing and bronchoalveolar lavage, which collects cells and fluid from the airways.
More about the research
It also requires clear, clinically meaningful measures of whether a treatment works. This core facility supplies those animal models and measurements to the three research projects of a larger AATD gene therapy program.
Why it matters
People with alpha-1 antitrypsin deficiency can develop serious lung disease. Reliable mouse and ferret models and standardized lung measurements are needed to judge whether gene therapy and genome editing approaches work before they are tried in patients. This core's work could help set clinically relevant benchmarks for these therapies.
Known grants
From NIH and NSF research grants. Labs often have other funding we can't see.
Closest labs
Allison Keeler-Klunk
University of Massachusetts Chan Medical School, MA·Microbiology & Immunology
Determine how adeno-associated virus-delivered CRISPR-Cas9 provokes B- and T-cell responses against Cas9 that clear edited cells, comparing dual-AAV delivery with an AAV plus lipid nanoparticle approach that expresses Cas9 only transiently.
Ronald G Crystal
Weill Medical College of Cornell University, NY·Medicine & Clinical Research
Secure Investigational New Drug approval and manufacture clinical-grade AAV8hAAT(AVL), a virus vector carrying an oxidation-resistant form of alpha 1-antitrypsin, the protein that blocks lung-digesting enzymes.
Matthew Zinter
University of California-San Francisco, CA·Medicine & Clinical Research
Identify blood correlates of lung injury after pediatric transplant by comparing gene-activity profiles between airway lavage fluid and blood in the same patient, and separating lung-based from non-lung contributors to mortality.
The school
University of Massachusetts Chan Medical School: 29 labs on PI Pages, $350M a year on research, 55 research doctorates a year.
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