An international team led by Monash University researchers has uncovered the genetic code governing the way genetic mutations ...

Because the new method can replace a large stretch of defective DNA with a healthy sequence, the same retron-based package can fix any combination of mutations within that stretch of DNA without ...

To solve this critical challenge, he founded Nephrogen in 2022, a biotech startup that uses AI and advanced screening to ...

Key pointsMonash researchers have uncovered the hidden 'code' governing how genetic mutations affect RNA splicing and result in diseaseBreakthrough ...

The Boston biotech founded by Harvard professor David Sinclair is testing whether cellular reprogramming can turn back the ...

The Neuromuscular Disease Therapeutics Market is entering a period of accelerated growth, supported by breakthroughs in gene therapy, antisense oligonucleotides (ASOs), monoclonal antibodies, and ...

A new Northwestern University study using patient nervous tissue and lab-grown human neurons has uncovered how a key disease ...

Researchers announce breakthrough results for AMT-130, the first disease-modifying treatment for Huntington's disease, with ...

Gene therapy is becoming a powerful way to treat challenging diseases that don’t respond to traditional treatments, and researchers now report the first success in modifying genes to slow Huntington’s ...

A baby born with a rare and dangerous genetic disease is growing and thriving after getting an experimental gene editing treatment made just for him. The baby, KJ Muldoon of Clifton Heights, ...

Lozano is a rare disease mom, neuroscience Ph.D. candidate at UC Davis, and board member for the PURA Syndrome Foundation. In May, a historic moment in science and medicine was captured in a single ...

Subscribe to our newsletter for the latest sci-tech news updates. Published in Nature Communications, scientists from the Wellcome Sanger Institute reveal insights into how our genes influence the ...