Delivery of therapeutic genes is essential for gene therapy. Adeno-associated viruses (AAVs) are a prime vector for carrying ...
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New workflow boosts nuclear delivery for safer gene therapy
Gene therapy holds the promise of preventing and curing disease by manipulating gene expression within a patient's cells.
Gene-editing tools like CRISPR have unlocked new treatments for previously uncurable diseases. Now, researchers at the ...
The great hope for gene therapy is that one day it will be out of the clinic and into mainstream therapeutics. Bearing enormous potential to treat or cure disease, such technology can replace a ...
NEW YORK--(BUSINESS WIRE)--Neurogene Inc., a company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, today announced results of a well ...
Gene therapy is a medical treatment that works by changing or fixing a person's genes to treat or prevent disease ...
The disease leads to the progressive growth of fluid-filled cysts in the kidneys, often resulting in kidney failure and other ...
Gene editing is already being used to treat a handful of rare genetic diseases; however, most therapies involve breaking or inactivating genes. Precisely fixing disease-causing mutations is far more ...
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