Breakthrough Parkinson’s Drug Targets Disease at Its Genetic Roots
Scientists have reported promising early results from a novel Parkinson’s treatment designed to suppress the activity of a…
New mouse model reveals primary trigger of rare muscle disorder
Myofibrillar myopathy type 6 (MFM6) is a rare genetic muscle disorder that leads to severe muscle weakness and…
New gene therapy improves hearing in patients with rare genetic deafness
A new international study co-led by investigators from Mass General Brigham and the Eye & ENT Hospital of…
Multicentre gene therapy for OTOF-related deafness followed up to 2.5 years
Yasunaga, S. et al. A mutation in OTOF, encoding otoferlin, a FER-1-like protein, causes DFNB9, a nonsyndromic form…
‘Deafness reversed’: Scientists restore hearing in 10 patients with a single-injection gene therapy breakthrough |
A clinical study has delivered one of the clearest signs yet that gene therapy can restore hearing in…
The Medicine Maker | mRNA Expands into Fertility Medicine
Credit: Mito Kanatsu-Shinohara
Researchers in Japan have demonstrated that messenger RNA (mRNA) therapy…
Gene-modified pancreas cells offer hope for people with type 1 diabetes
Key Insights A proof-of-concept trial in a single patient has shown that genetically engineered pancreas cells can…
Genetic mismatch identified as major risk factor in cord blood transplantation
A Japanese study of more than 7,400 patients has identified a genetic mismatch that sharply increases the risk…
Eyes of the world’s longest-living vertebrate, the Greenland shark, show little ageing
Swimming at a crawl with cloudy eyes and mottled skin, the Greenland shark looks like it’s seen better…
AskBio Announces FDA Acceptance of Investigational New Drug (IND) Application for AB-1009 Gene Therapy for Treatment of Late-Onset Pompe Disease (LOPD)
Durham, N.C., Jan. 08, 2026 (GLOBE NEWSWIRE) — AB-1009 program advances to Phase 1/Phase 2; clinical trial initiated…
7 medical breakthroughs of 2025
For most diseases, the challenge is not a lack of effort but the limits of existing tools and…
Science in 2026: what to expect this year – Nature
Science in 2026: what to expect this year NatureDrugs to Watch in 2026 Dermatology TimesScience in 2026: Breakthroughs Set to…