Researchers have identified how variations in a gene called TRIO can influence brain functions and result in distinct ...

Scientists present current evidence for a new gene therapy for Duchenne muscular dystrophy called delandistrogene moxeparvovec. AAN Evidence in Focus articles highlight the strength of the current ...

Tessera Therapeutics Presented new preclinical data in non-human primates (NHP) for alpha-1 antitrypsin deficiency (AATD) and phenylketonuria (PKU), where RNA Gene Writer achieved an estimated 76% ...

Gene Hackman, Wes Anderson, Bill Murray on the set of 2001's 'The Royal Tenenbaums' Buena Vista Pictures/Courtesy Everett Collection Ahead of the Cannes premiere of his latest film, The Phoenician ...

In this editorial, the authors describe the foundations of an N-of-1 gene-editing study to treat an infant with a urea-cycle disorder. 2 Department of Chemistry and Biochemistry, University of ...

The one-off treatment highlights the promise of a new type of gene editing and the challenges of using it to treat extremely rare genetic diseases. Doctors say they constructed a bespoke gene ...

You can reach Megan on Signal at mmolteni.13. Leading trade organizations representing the makers of cell and gene therapies are calling for a 10-year international moratorium on the use of CRISPR ...

Get Instant Summarized Text (Gist) A child with severe carbamoyl phosphate synthetase 1 (CPS1) deficiency received a personalized CRISPR-based gene editing therapy, resulting in improved metabolic ...

The latest iteration of the editor, which utilized complex enzymes found in bacteria, can be programmed to insert an entire gene - or multiple genes - into a specific location in the human genome ...

News broke yesterday that researchers in Philadelphia appear to have successfully treated a 6-month-old baby boy, called KJ, with a personalized CRISPR gene-editing therapy. The treatment corrects ...

Doctors announced this week that they have treated a newborn baby with a rare genetic disease using the world’s first personalized gene editing therapy. Geoff Bennett discussed the treatment and ...