Single protein is key to treating a deadly genetic heart disease targeting young athletes
A research team at the University of California San Diego has discovered a novel and promising method of…
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…
the events to watch for in the coming year
India’s Aditya-L1 spacecraft launched in 2023. Next year, it will observe the Sun during its peak activity phase.Credit:…
Novo Nordisk backs biotech start-up targeting aging
General Control enters Novo Nordisk’s Co-Creation Greenhouse to advance multiplex epigenetic editing for complex chronic disease. General Control,…
Cutting-edge treatments in amyotrophic lateral sclerosis: the role of molecular pathogenesis in targeted therapies | Stem Cell Research & Therapy
Feldman EL, Goutman SA, Petri S, Mazzini L, Savelieff MG, Shaw PJ, et al. Amyotrophic lateral sclerosis. Lancet.…
Muscle-driven gene therapy shows promise for protecting memory in Alzheimer’s
Alzheimer’s disease (AD) is a devastating cause of memory loss and cognitive decline, for which no curative treatment…
Harmful cholesterol levels cut in half with one-time gene editing drug in early trial
Altering a single gene may help people lower dangerously high levels of cholesterol and other fats in the…
New gene-editing method can correct many disease-causing mutations in mammalian cells
Some genetic disorders-such as cystic fibrosis, hemophilia and Tay Sachs disease-involve many mutations in a person’s genome, often…
Experimental gene therapy successfully treats dozens of children with rare fatal disorder
ADVERTISEMENT An experimental gene therapy has successfully treated dozens of children with a rare and fatal immune disorder,…
Experimental gene therapy restores immune system function in children with genetic immune disorder
An experimental gene therapy developed by researchers at UCLA, University College London and Great Ormond Street Hospital has restored…
Harnessing “DNA surgery” to develop therapeutic medicines for genetic liver and heart diseases
Genome editing is increasingly recognzed as a leading frontier in biomedical research and an essential driver of healthcare…