Gene therapy may be 'one-shot stop' for rare bone disease
For the last 10 years, the only effective treatment for hypophosphatasia (HPP) has been an enzyme replacement therapy that ...
The Scientist1 天
A Nanoparticle Delivery System for Gene Therapy
Genetic medicines could treat certain diseases, however, the current delivery systems available—viral vectors and lipid nanoparticles (LNPs)—pose unique challenges for gene therapy applications. For ...
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 ...
UK health system to offer cutting-edge gene therapy for sickle cell disease
Britain's National Health Service (NHS) will provide a cutting-edge gene therapy that aims to cure sickle cell disease, the ...