sciencenewsnet.in

Companion Penn Medicine Studies on AAV-based Gene Therapies in Non-Human Primates Suggest Integration into Human DNA is Unlikely to Drive Cancer Mutations While Offering the Potential for Durable Expression of the Transgene

November 6, 2023 sarah JonasResearch Results

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).

Reaching toward a cure for sickle cell disease

April 2, 2020 sarah JonasFeature

The National Heart, Lung, and Blood Institute (NHLBI) has awarded Case Western Reserve University up to $3.7 million to assess emerging genome-editing based therapies being tested for curing sickle cell disease (SCD) at leading U.S research universities and hospitals.

SCD is the most well-known among a group of inherited blood disorders, affecting about 100,000 people in the United States and about 20 million worldwide, according to a 2018 National Institutes of Health (NIH) statement announcing the NHLBI Cure Sickle Cell Initiative.

Virus surfaces help MTU engineers study vaccine and gene therapy applications

January 8, 2020 sarah JonasResearch Results

An isoelectric point is a common way to characterize viruses. However, it’s not easy. To improve manufacturing for vaccines and gene therapy, a Michigan Tech team uses surface charge to determine the isoelectric point of different viruses. Specifically, they use a single-particle method with atomic force microscopy (AFM).