The transcriptome libraries were sequenced by Annoroad Gene Technology Co., Ltd (Beijing, China) on an Illumina platform. For RNA-seq analysis, the quality of sequenced clean data was analyzed using ...

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 ...

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 ...

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. Researchers described the case in a new study ...

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 ...

A baby born with a rare genetic disease is "growing and thriving" after getting bespoke gene therapy. It's the first time anyone in the world has been given an experimental gene-editing treatment ...

Instead, KJ has made medical history. The baby, now 9 ½ months old, became the first patient of any age to have a custom gene-editing treatment, according to his doctors. He received an infusion ...

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 ...

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 ...