Gene therapy using adeno-associated virus (AAV) vectors
Neurological and metabolic diseases
Therapeutic genome editing
RNA-based approaches to treating genetic diseases
Representative Publications
Wang J, Zhang Y, Mendonca CA, Yukselen O, Muneeruddin K, Ren L, Liang J, Zhou C, Xie J, Li J, Jiang Z, Kucukural A, Shaffer SA, Gao G, Wang D. AAV-delivered suppressor tRNA overcomes a nonsense mutation in mice. Nature. 2022. Read Publication
Gene therapy developed for maple syrup urine disease shows promise, new UMass Chan study reports
UMass Chan researchersdemonstrated the promise of a gene therapy to correct a gene mutation causing maple syrup urine disease.
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Jul 3, 2024
UMass Chan BRIDGE Fund commits $2M in six faculty projects
BRIDGE Innovation and Business Development at UMass Chan Medical School has set aside nearly $2 million in funding for six faculty-led research projects that hold promise for translation to clinical application and commercialization.
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Apr 21, 2022
UMass Chan research supports development of new suppressor-tRNA-based gene therapies
Guangping Gao, PhD, and Dan Wang, PhD, show the first evidence that a suppressor transfer RNA therapy, delivered by a recombinant adeno-associated virus, can restore protein production up to six months after treatment in a mouse model of the rare genetic disease mucopolysaccharidosis type I.
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Nov 18, 2021
Flotte lab to develop gene therapy models for genetic lung disease
Researchers from UMass Chan Medical School have received a five-year $13.6 million program project grant from the National Heart, Lung and Blood Institute to develop new gene therapy models for alpha-1 antitrypsin deficiency, a chronic, debilitating genetic lung disease that shortens the lifespan.
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Jul 29, 2021
Guangping Gao and Dan Wang partner with ASC Therapeutics to develop novel gene therapy for maple syrup urine disease
UMass Medical School researchers Guangping Gao, PhD, and Dan Wang, PhD, are working with ASC Therapeutics to bring a gene therapy for maple syrup urine disease (MSUD) to the clinic.
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Apr 8, 2021
UMMS establishes gene therapy collaborative research agreement with Pfizer
UMass Medical School has entered into a three-year, collaborative research agreement with Pfizer to evaluate determinants that influence the manufacturing quality and yield of viral vectors used in gene therapy. The research at UMMS is being performed under the direction of Guangping Gao, PhD, and Dan Wang, PhD.
