The majority are in the very early preclinical stage of development, but applications for regulatory approval have been ...
Base editing utilizes enzymes to modify single amino acids at the most foundational unit of DNA, called a base.
A £1.65 million treatment has been approved for use for some NHS patients, offering some with an inherited blood disorder ...
This new tool, known as minimal, versatile genetic perturbation technology (mvGPT), combines the powers of gene editing, ...
In two new papers, researchers from The Jackson Laboratory (JAX) report the successful use of two approaches—gene therapy and bone marrow transplantation—to alleviate symptoms of multiple ...
There are 20 cell and gene therapies on Europe's market versus 43 in the U.S. Find out more about this ecosystem.
The UK has approximately 17,500 individuals living with sickle cell disease, a condition particularly prevalent amongst those ...
Investors often like to be on the cutting edge of new developments, and it doesn't get much more cutting edge than gene therapy and editing. The ability to make changes to a person's DNA – the ...
who is now able to hear thanks to gene therapy. It is now 12 months since the Food and Drug Administration rolled out the historic approval of a CRISPR therapy designed for patients suffering from ...
Pfizer’s research partnership with Sangamo Therapeutics produced a hemophilia A gene therapy that reached FDA discussions about a regulatory submission. That’s as far as the alliance will go.
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