Treatment Uses Reduced-Intensity Preconditioning to Enhance Its Global Transportability After over a decade of preclinical research and development, a new gene therapy treatment for sickle c... Read more
Biological engineers design RNA circuits that enable precise control over the dose of therapeutic protein a patient receives. Delivering functional genes into cells to replace mutated genes,... Read more
Researchers at King’s College London have shown that rats with spinal cord injuries can re-learn skilled hand movements after being treated with a gene therapy People with spinal cord injury... Read more
Researchers from Drexel University reversed symptoms of Alzheimer’s disease in fruit flies by restoring the balance between two epigenetic enzymes that regulate gene expression, a study show... Read more
In a landmark study, scientists have used CRISPR gene editing to introduce beneficial natural mutations into blood cells to boost their production of foetal haemoglobin. In a landmark study... Read more
The results could open the door to new targeted therapies for this debilitating disease in the future In the largest study of its kind, nine novel genes for osteoarthritis have been discover... Read more
Researchers from the University of Pennsylvania have developed a gene therapy that successfully treats a form of macular degeneration in a canine model. The work sets the stage for translati... Read more
FINDINGS Through gene therapy, researchers engineered blood-forming stem cells (hematopoietic stem/progenitor cells, or HSPCs) to carry chimeric antigen receptor (CAR) genes to make cells th... Read more
A ‘cure’ for haemophilia is one step closer, following results of a groundbreaking gene therapy trial led by the NHS in London. Clinical researchers at Barts Health NHS Trust and... Read more
Study shows first-of-its kind gene therapy can restore vision to people with an inherited retinal disorder Patients who had lost their sight to an inherited retinal disease could see well en... Read more
Injecting DNA into injured horse tendons and ligaments can cure lameness, new research involving scientists at Kazan Federal University, Moscow State Academy and The University of Nottingham... Read more
Multiple sclerosis can be inhibited or reversed using a novel gene therapy technique that stops the disease’s immune response in mouse models, University of Florida Health researchers have f... Read more
Researchers from Genethon, the AFM-Téléthon laboratory, Inserm (UMR 1089, Nantes) and the University of London (Royal Holloway) demonstrated the efficacy of an innovative gene therapy in the... Read more