A new CRISPR-based one-off procedure that lowers "bad" cholesterol has been approved to enter Phase I human trial. If ...
Gene-editing tools like CRISPR have unlocked new treatments for previously uncurable diseases. Now, researchers at the ...
ACM impedes the heart from pumping blood to the rest of the body, and is a leading cause of sudden cardiac death in young ...
People looking to lose weight and lower their blood sugar may someday be able to get a single injection that turns their cells into tiny factories churning out a protein that is essentially the active ...
A gene therapy candidate extended survival in a mouse model of SMARD1, supporting its testing in a clinical trial for this ...
A "gene silencer" (technically known as small interfering RNA, or siRNA), locally delivered by nanoparticles embedded in an ...
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Unlocking the secrets of gene therapy delivery: New insights into genome ejection from AAV vectors
A research team at the University of Osaka has unveiled the molecular mechanism behind genome ejection from adeno-associated virus (AAV) vectors, a crucial delivery vehicle in gene therapy. Subscribe ...
NanoMosaic, a leader in advanced analytical technologies for cell and gene therapy development and manufacturing, today announced that the FDA's Center for Biologics Evaluation and Research (CBER) has ...
Ocugen, Inc. gene therapy outlook: key 2026 catalysts, Phase 2/3 eye-disease data, BLA plans to 2028, and risks. Click for ...
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