National Institute for Health and Care Excellence9 小时
Groundbreaking one-off gene therapy approved for severe sickle cell disease
People in England to benefit from groundbreaking one-off gene therapy for severe sickle cell disease approved by NICE ...
precisionmedicineonline1 小时
Fractyl Health Expects to Launch Clinical Trial of Type 2 Diabetes Gene Therapy in 2025
While the company is prioritizing RJVA-001 and a lead program in obesity, it's pausing some R&D efforts and laying off 17 percent of its workforce.
Gene therapy and bone marrow transplant alleviate ultra-rare genetic disease symptoms
In two new papers, researchers from The Jackson Laboratory (JAX) report the successful use of two approaches—gene therapy and ...
Genetic Swiss Army Knife: New Tool For Gene Editing And Therapy
This new tool, known as minimal, versatile genetic perturbation technology (mvGPT), combines the powers of gene editing, ...
EurekAlert!3 天
JAX researchers alleviate symptoms of ultra-rare genetic disease
In two new papers, researchers from The Jackson Laboratory (JAX) report the successful use of two approaches -- gene therapy ...
'Hope of a cure': Revolutionary gene-editing therapy for sickle cell approved for use on NHS
NHS officials estimate around 50 people a year will receive treatment now it has been approved for use for certain patients with severe sickle cell disease. | ITV National News ...