Gene therapy adeno-associated viruses (AAVs) - viruses that can be engineered to deliver DNA to target cells - are unlikely to cause cancer-triggering insertions in humans or monkeys and may contribute to long-term efficacy, according to new research from the University of Pennsylvania’s Gene Therapy Program (GTP).
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Posted on Nov 06, 2023
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The Foundation for Angelman Syndrome Therapeutics (FAST), a non-profit organization, has entered into an exclusive global collaborative research and development agreement with the University of Pennsylvania to develop an investigational adeno-associated virus (AAV) gene therapy for Angelman syndrome.
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Posted on Oct 17, 2023
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Nine researchers from the University of Pennsylvania’s Gene Therapy Program (GTP) presented abstracts highlighting their translational science and discovery research on gene therapy, gene editing, and adeno-associated virus (AAV) vector technology at the American Society of Cell and Gene Therapy (ASGCT)’s 26th Annual Meeting.
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Posted on May 16, 2023
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Forty years after the Orphan Drug Act passed, researchers advance drug development for neglected rare conditions everywhere from the lab bench to backstage.
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Posted on May 02, 2023
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