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gene therapy

Going Viral: The AAV Approach to Curing Cancer
 Going Viral: The AAV Approach to Curing Cancer 1024 256 Emily Siebenmorgen
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According to Timothy Cripe, MD, PhD, chief of the Division of Hematology and Oncology at Nationwide Children’s Hospital, it’s an incredible time to be working on cancer treatment – and now, targeted cancer prevention. “There’s so much going on in the cancer world these days,” says Dr. Cripe, who is also a principal investigator in…

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FDA Conditionally Approves Micro-dystrophin: A Small Gene With Big Promise
 FDA Conditionally Approves Micro-dystrophin: A Small Gene With Big Promise 1024 632 Abbie Miller
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UPDATE: On June 22, 2023, the FDA announced accelerated approval for SRP-9001/ELEVIDYS for Duchenne muscular dystrophy (DMD) following decades of research in the Center for Gene Therapy at Nationwide Children’s Hospital. The FDA approved the treatment for pediatric patients 4-5 years old with DMD. Jerry Mendell, MD, principal investigator in the Center for Gene Therapy…

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A Single-Dose Cancer Immunotherapy via Gene Transfer
 A Single-Dose Cancer Immunotherapy via Gene Transfer 150 150 Mary Bates, PhD
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Researchers at Nationwide Children’s Hospital report proof-of-principle results for a new gene therapy cancer treatment. The off-the-shelf, single-dose immunotherapy serves as an alternative to CAR-T therapy and can be engineered to be on-demand. T cells redirected to cancer cells either via a chimeric antigen receptor (CAR-T) or a bispecific molecule have been game-changers in treating…

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