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

The FDA has offered positive feedback on possible paths forward for accelerated or traditional approval of FLT201 using data from a single-arm study.

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.

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

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

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

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

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 ...

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 ...

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 ...