The U.S. Food and Drug Administration (FDA) has approved prademagene zamikeracel (Zevaskyn) for the treatment of blistering ...

The focus should be on respecting the evidence, and our lived experiences, by providing all children with an appropriate language. Proposing gene therapies for deaf children risks overlooking the ...

Viruses are known to use the genetic machinery of the human cells they invade to make copies of themselves. As part of the process, viruses leave behind remnants throughout the genetic material ...

Patients with severe dystrophic epidermolysis bullosa, or EB, have skin so fragile, the slightest touch can lead to ...

In April, the Deparment of Health and Human Services shuttered the committe that oversaw newborn screenings. Advocates hope ...

Researchers at the University of Minnesota have completed a first-in-human clinical trial testing a CRISPR/Cas9 gene-editing technique to help the immune system fight advanced gastrointestinal (GI) ...

A new gene therapy reversed heart failure in pigs by repairing heart function through cBIN1, showing major promise for future ...

Researchers have completed a first-in-human clinical trial testing a CRISPR/Cas9 gene-editing technique to help the immune system fight advanced gastrointestinal (GI) cancers. The results show ...

The only remaining autonomous 'jumping gene' can only attach to, and stitch a copy of itself into, DNA when it builds up into large clusters and only as cells divide.

Gene therapy has emerged as a promising approach to previously untreatable conditions, including Duchenne muscular dystrophy (DMD), a progressive disorder caused by DMD mutations that leads to early ...

Viruses are known to use the genetic machinery of the human cells they invade to make copies of themselves. As part of the ...

Researchers at the University of Minnesota have completed a first-in-human clinical trial testing a CRISPR/Cas9 gene-editing ...