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News Release: Revolutionary Gene Editing Method Could Fuel Next Generation of Cell and Gene Therapies
PHILADELPHIA - Researchers from the Perelman School of Medicine at the University of Pennsylvania have unveiled a groundbreaking technique that promises to enhance speed, efficiency, and minimize cellular toxicity in gene editing processes. The innovative method could significantly advance cell therapies for various diseases, including cancer, by offering an alternative to existing approaches.
The new approach leverages protein fragments utilized by some viruses to enter cells, enabling CRISPR-Cas gene editing molecules to penetrate the outer membranes of primary cells and access their DNA-contning nuclei with unprecedented efficacy while mntning low cellular toxicity. The findings were published in Nature Biotechnology this week.
This technique is expected to be particularly beneficial for modifying T-cells and other patient-derived primary cells into cell therapies, such as CAR T-cell therapy. In CAR T therapy, immune cells from a patient are removed, reprogrammed to target and destroy cancer cells after reintroduction into the bloodstream. The first FDA-approved CAR T therapy was developed at Penn Medicine and received approval in 2017.
We envision this new -a continuation of Penn Medicine's pioneering work in cell and gene therapies-to serve as a major enabler for next-generation treatments, sd lead author Junwei Shi, Ph.D., Assistant Professor of Cancer Biology and member of the Penn Epigenetics Institute and Abramson Family Cancer Research Institute.
The method was developed through a collaborative effort led by the laboratories of Rahul Kohli, MD, PhD, Associate Professor of Infectious Diseases and Biochemistry and Biophysics, and Gerd Blobel, MD, Ph.D., Frank E. Weise III Professor of Pediatrics and co-director of the Penn Epigenetics Institute.
The study was supported by funding from organizations including the National Institute of Health R01-HL119479, R01-GM138908, Parker Institute for Cancer Immunotherapy, and institutional funds from University of Pennsylvania.
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News Release: Revolutionary Gene Editing Method Could Fuel Next Generation Cell Therapies
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Revolutionary Gene Editing Method Enhances Speed and Efficiency Minimizes Cellular Toxicity Next Generation Cell Therapies Advanced CRISPR Cas Techniques Improved T cell and CAR T Cell Therapy