Cytoreductive gene therapy: to deliver an exogenous gene that causes cell death or allows the application of toxic agents. Immunomodulatory gene therapy: to induce gene expression that enhances ...

For the last 10 years, the only effective treatment for hypophosphatasia (HPP) has been an enzyme replacement therapy that ...

A new gene editing treatment has been approved for use in the NHS, which will help treat sickle cell disease patients ...

Researchers are enhancing the best features of AAV capsids and overcoming their limitations to accelerate gene-based therapies.

For the last 10 years, the only effective treatment for hypophosphatasia (HPP) has been an enzyme replacement therapy that must be delivered by injection three-to-six times each week.

The California Institute for Regenerative Medicine has awarded a $6 million grant to USC investigators pioneering a new first ...

Britain's National Health Service (NHS) will provide a cutting-edge gene therapy that aims to cure sickle cell disease, the ...

President Trump signed an executive order on the day of his inauguration that created doubts about the future of a program that helps state Medicaid programs pay for expensive cell and gene therapy.

In two new papers, researchers from The Jackson Laboratory (JAX) report the successful use of two approaches—gene therapy and ...

Experts hailed exciting news for some people with sickle cell disease in England as the health watchdog decided to grant ...