Prenatal gene therapy via mRNA LNPs fixes faulty genes in fetal brain cells in animal model of neurodevelopmental syndrome.

Some prior research had shown that gene therapy would not lower the amount of time children spend on ventilator support.

A single treatment with, a CRISPR-Cas9 based gene editing therapy, is enough to replace the daily medication of patients with ...

Medera Inc. today announced that its clinical-stage gene therapy subsidiary, Sardocor Corp., has successfully completed its ...

Intellia has reported positive results from its ongoing Phase I/II trial of NTLA-2002, a CRISPR-based gene editing therapy ...

CHILDREN in Spain who have been deaf from birth are hearing for the first time after taking part in gene therapy trials. Four ...

Positive results for Intellia Therapeutics, Inc.'s NTLA-2002 in treating HAE patients. Stock price dips, but the program ...

Acquisition creates a leading, clinical-stage company focused on the development of gene therapy treatments for rare inherited retinal degenerations New OPGx-LCA5 Phase 1/2 6-month data demonstrate sa ...

Dyno Therapeutics has formed a new research partnership with Roche, focusing on the development of next-generation ...

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The big picture: The potential of CRISPR gene-editing technology was realized six months ago, when the Food and Drug Administration approved the first therapy that uses the system to treat sickle-cell ...

So, we’re voting, and the outcome may end the world, or not. So, we’re voting, and just reading the news this morning put me ...