Researchers have developed an innovative new nanopore sequencing assay to identify carriers of fragile X syndrome (FXS), the ...

Researchers have developed an innovative new nanopore sequencing assay to identify carriers of fragile X syndrome (FXS), the ...

The approval was a landmark in the quest to design gene-editing therapies with curative potential. Casgevy shows exceptional efficacy, but so far the therapy has been administered to only a small ...

In order to investigate the stability of the FMR1 (Fragile X Mental Retardation 1) alleles from the normal population, when maternally inherited, we analyzed 75 mother-to-son transmissions.

The FDA has slapped a clinical hold on Rocket Pharmaceuticals’ pivotal gene therapy trial in response to a death. Rocket, which was aiming to report data in mid-2026, dropped its targeted trial ...

Rocket Pharmaceuticals acknowledged the death of a patient in a pivotal trial assessing its Danon disease gene therapy candidate RP-A501, a study that the FDA has placed on clinical hold.

You can reach Jason on Signal at JasonMast.05. Rocket Pharmaceuticals said Tuesday that a patient had died in a trial studying its experimental gene therapy for a rare heart condition called Danon ...

May 27 (Reuters) - The U.S. FDA has paused Rocket Pharmaceuticals' (RCKT.O), opens new tab mid-stage gene therapy trial after a patient died from serious complications, the drug developer said on ...

AP The families of two of the 10 contacted their fertility clinics after their children developed cancers associated with the gene variant TP53. The European Sperm Bank had supplied the sperm ...

For the first time, researchers have created a gene-edited spider that spins red fluorescent silk. Led by a team at the University of Bayreuth, the project tackled a long-standing challenge in ...

The reduction/absence of the functional FMRP protein, coded by the X-linked Fmr1 gene in humans, is responsible for the syndrome. Targets of FMRP, CLSTN1, and ICAM5, play critical roles in the ...