Investigators at Weill Cornell Medicine have developed a versatile and non-toxic technology for controlling the activity of ...

Although heart cells and skin cells contain identical instructions for creating proteins encoded in their DNA, they're able ...

Priority Research Communication by Professor Yogesh Dwivedi and colleagues at the University of Alabama at Birmingham reports original, peer-reviewed findings demonstrating that long noncoding RNAs ...

A team of researchers from the National Institutes for Quantum Science and Technology (QST) and Tokyo Metropolitan University has developed a biomaterial that could change how we treat muscle ...

Arc Institute, Gladstone Institutes, and University of California, San Francisco, scientists have developed an epigenetic editing platform that enables safe modification of multiple genes in primary ...

These long-term findings in a large patient cohort show the sustained clinical efficacy and safety of autologous CD34+ hematopoietic stem-cell lentiviral gene therapy for ADA-SCID, indicating that it ...

A Washington man appeared to be destined to develop Alzheimer’s disease — but against all genetic odds, he has eluded the common dementia for decades. Researchers at the Washington University School ...

AUSTIN, Texas, Oct. 14, 2025 /PRNewswire/ -- Genprex, Inc. ("Genprex" or the "Company") (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients ...

Regeneron is clearing a regulatory path for its investigational gene therapy DB-OTO after an early phase study demonstrated hearing improvements in children with a genetic form of deafness. The data, ...

Genetic deficiency of otoferlin, a protein critical to synaptic transmission by the sensory hair cells of the ear, causes congenital deafness. Medicines to treat the condition are lacking; children ...

Each year, a small number of babies are born mostly, if not fully, deaf because one of their genes isn’t working. The gene normally makes a protein that the hairs in our inner ears need to relay sound ...