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Guangping Gao, PhD

Research Focus - Adeno-associated Virus: Biology, Vectorology, and Applications for In Vivo Gene Therapy

  • The molecular genetics and pathomechanism of inborn errors of metabolism in the central nervous system
  • Natural evolution, host interactions, and genetic fate of adeno-associated virus (AAV)
  • Vectorology, transduction biology, and structural biology of AAV
  • Novel enabling technology platform development for AAV vectors, genome editing, animal modeling, and RNA therapeutics
  • Interventional in vivo gene therapy for gene replacement, silencing, addition, and editing

Representative Publication

Wang D, Tai PWL, Gao G. Adeno-associated virus vector as a platform for gene therapy delivery. Nat Rev Drug Discov. 2019;18(5):358-378.

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In the News

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  • UMass Chan research explores differences in AAV vector manufacture, shows clear winner for method
    Research News

    UMass Chan research explores differences in AAV vector manufacture, shows clear winner for method

    A paper from the Horae Gene Therapy Center at UMass Chan Medical School may settle a longstanding debate about how to best manufacture adeno-associated virus vectors for gene therapy.

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  • UMass Chan research supports development of new suppressor-tRNA-based gene therapies
    Research News

    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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  • Flotte lab to develop gene therapy models for genetic lung disease
    Research News

    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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  • Behind the research: Jae-Hyuck Shim strives to unlock cure for devastating childhood bone disease
    Research News

    Behind the research: Jae-Hyuck Shim strives to unlock cure for devastating childhood bone disease

    Jae-Hyuck Shim, PhD, is inspired to develop a gene therapy for fibrodysplasia ossificans progressiva (FOP) after seeing the strength of families who have a child with the rare, crippling disease.

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  • Guangping Gao and Dan Wang partner with ASC Therapeutics to develop novel gene therapy for maple syrup urine disease
    Research News

    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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  • UMMS establishes gene therapy collaborative research agreement with Pfizer
    Research News

    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.

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  • Erik Sontheimer co-leading efforts to develop gene editing toolkit by NIH Somatic Cell Genome Editing Consortium
    Research News

    Erik Sontheimer co-leading efforts to develop gene editing toolkit by NIH Somatic Cell Genome Editing Consortium

    Six UMass Medical School scientists are among the members of the National Institutes of Health’s Somatic Cell Genome Editing Consortium to publish a paper in Nature outlining the program’s goals.

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  • Guangping Gao makes list of Nature Biotechnology Top 20 translational researchers
    Community News

    Guangping Gao makes list of Nature Biotechnology Top 20 translational researchers

    An influential scientific journal’s list of top translational biotech researchers for 2019 includes Guangping Gao, PhD. Rankings are based on total patents for the year, among other factors.

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  • Guangping Gao elected to American Academy of Microbiology
    Research News

    Guangping Gao elected to American Academy of Microbiology

    Guangping Gao, PhD, has been elected to the American Academy of Microbiology, the honorific leadership group of the American Society of Microbiology.

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  • Synergy among 40 scientists at Li Weibo Institute for Rare Diseases Research accelerates discovery
    Research News

    Synergy among 40 scientists at Li Weibo Institute for Rare Diseases Research accelerates discovery

    Inspired by patients and their families, 40 scientists at the The Li Weibo (李伟波) Institute for Rare Diseases Research are working together to discover cures for dozens of rare disorders, according to a Facebook Live interview with co-directors Michael Green, MD, PhD, and Guangping Gao, PhD.

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