University of Illinois Urbana-Champaign · EngineeringGene Editing for Neurodegenerative Diseases Lab
Thomas Gaj · Champaign, Illinois
Research Focus
Dr. Thomas Gaj's lab focuses on developing innovative gene-editing therapies for amyotrophic lateral sclerosis (ALS), a devastating neurodegenerative disease caused by mutations in the SOD1 gene. Using a cutting-edge technique called CRISPR base editing, the lab aims to safely and effectively turn off the production of harmful mutant proteins in the nervous system. Their work not only targets ALS but also paves the way for potential treatments for other neurological disorders.
CRISPR base editingALSgene therapySOD1 mutationsneurodegenerationAAV deliverygenetic diseasepermanent gene modification
Research Aims
5AIM 01Develop a CRISPR base editing strategy to permanently inactivate mutant SOD1 in ALS models.
AIM 02Optimize the targeting specificity and editing capabilities of the gene therapy.
AIM 03Enhance the pharmacokinetics and safety profile of the therapy.
AIM 04Investigate the long-term effects of the gene therapy in animal models.
AIM 05Establish a foundation for using base editing in treating various neurodegenerative diseases.
Skills the Lab Values
CRISPR base editinggene delivery with adeno-associated virus (AAV)in vivo modelsmouse geneticsmolecular cloningDNA extraction and analysiscell culturePCRimmunology techniquespharmacokinetics assessment
Output against the department
Publications61bottom 9% of dept.252nd of 278
this lab
most publishedleast published
Citations8.9ktop 41% of dept.114th of 278
this lab
most citedleast cited
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