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 ...

Besides pain medications to relieve symptoms as well as antibiotics to treat infections, hydroxyurea—a bone marrow suppressive agent that ... All cell and gene therapy manufacturers face a ...

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 ...

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

For recurrent respiratory papillomatosis (RRP), novel gene therapy to jump-start immunity to the human papillomavirus (HPV) ...

Scientists looked at multiple techniques used to measure the modified viruses deployed in some gene therapy research and treatments. One technique, known as SEC-MALS, was the most precise and ...

The study revealed multiple "common" and "cell specific" changes caused by insulin-resistance representing new targets for pharmacological or targeted gene therapy approaches. Richard Coward ...

Factor VIII gene was introduced into hematopoietic stem cells in men with hemophilia. The annualized bleeding rate went from 10 to 0. No worrisome gene integrations were seen. The content of this ...

Lentiviral self-inactivating vectors are the retroviral vectors with the greatest potential for gene therapy Nondividing cells can be transduced by lentiviral vectors. They possess a low ...

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