#gene therapy

For the research team, the question was: “Can we turn something that is good for the cancer into something that will kill [it]?”

Potential cure strategy builds on unexpected findings about hematopoietic stem cells.

NIH-supported trial is the first step in developing a new approach to treat myasthenia gravis.

New research from Dana-Farber Cancer Institute has identified 116 genes as key molecular vulnerabilities for multiple myeloma.

Meet the eight innovative scientists and learn about the research Damon Runyon is funding.

Half of patients treated with the adenovirus vector-based gene therapy Adstiladrin experienced complete remission.

Researchers hope to combine gene editing with a less toxic stem cell transplant. Findings may apply to cancer and other illnesses.

Edited T cells survive and thrive in three patients in the first U.S. trial using this approach.

Researchers have developed a new gene therapy approach for children with severe combined immunodeficiency.

The accelerated approval pathway may offer a faster route to approval for new treatments in significant, unmet medical needs.

A common mutation makes mouse tumors more vulnerable to certain cancer drugs.

The animals have 100 times as many cells as humans but rarely get cancer. New gene studies may help explain why.

We need to make certain we assure patient safety and adequately characterize the potential risks and benefits of these products.

New immunotherapy approach could make engineering immune cells faster and cheaper.

This emerging area of treatment could hold incredible promise –– but critics are wary of its approach.

For example: Hep C cures have meant fewer people to treat and fewer transmissions to potential patients.