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

MRNA acts as the key intermediate between DNA, the genetic blueprint, and proteins that carry out most of the work in cells, with RNA splicing ensuring proper reading of the blueprint by removing some ...

Scientists have reversed diabetic heart failure with a genetic therapy in mice and miniature human "hearts" grown from stem ...

A one-time gene therapy using a patient’s own stem cells has effectively cured a deadly immune disorder in 95% of treated ...

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

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, ...

Gene therapies for rare diseases are frequently developed then discarded by drug companies because they can’t afford to produce the treatment for more patients. Gene therapies for rare diseases are ...

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 ...

A new breakthrough in a rare genetic disease which causes children to age rapidly has been discovered using 'longevity genes' ...

Victor Sung, M.D., director of the Division of Movement Disorders in the University of Alabama at Birmingham Department of ...

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