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News Release

NIH researchers tackle thorny side of gene therapy

NIH researchers have uncovered a key factor in understanding the elevated cancer risk associated with gene therapy.

… in understanding the elevated cancer risk associated with gene therapy. They conducted research on mice with a rare disease … humans, hoping their findings may eventually help improve gene therapy for humans. Researchers at the National Human …

Fact Sheet

Gene therapy patient education materials

Understanding gene therapy for sickle cell disease

… Now, researchers are developing new and emerging gene therapies to treat sickle cell disease. To help navigate … for the Democratizing Education for Sickle Cell Disease Gene Therapy Project, which is led by NHGRI in partnership with …

News Release

Gene therapy shows promise for treating Niemann-Pick disease type C1

NIH researchers demonstrated that gene therapy may be the best method for correcting the single faulty gene that causes Niemann-Pick disease, type C1.

… of Health (NIH) researchers have demonstrated in mice that gene therapy may be the best method for correcting the single faulty gene that causes Niemann-Pick disease, type C1 (NPC1). The …

Fact Sheet

Navigating sickle cell disease gene therapy

Gene therapy for sickle cell disease is available to you through FDA-approved therapies and through clinical trials.

… Gene therapy for sickle cell disease is available to you through … there are a number of clinical trials for sickle cell gene therapy, the journey can be similar across all treatments. …

Research at NHGRI

Sickle cell disease gene therapy education project

The Democratizing Education for Sickle Cell Disease Gene Therapy project developed patient-focused educational materials for the sickle cell community.

… disease is an inherited disorder caused by a change in the gene that produces hemoglobin, the red blood cells that carry … the U.S. Food and Drug Administration (FDA) approved two gene therapies to treat sickle cell disease. More than 50 … the Democratizing Education for Sickle Cell Disease Gene Therapy Project, which is led by NHGRI in partnership with …

Educational Resources

Gene Therapy

Gene therapy is an experimental technique for treating disease by altering the patient's genetic material.

… Gene Therapy … Gene therapy is an experimental technique for treating disease by …

News Release

How do you toast a gene therapy trial? With tea.

It’s teatime and doctors, researchers and patients are at the table. They celebrate a decade of work and launch the first in-human gene therapy trial for children with a rare and devastating disease, GM1 gangliosidosis.

… celebrate a decade of work and launch the first in-human gene therapy trial for children with a rare and devastating … disease. Jojo was about to receive the first experimental gene therapy treatment for the rare disease, GM1 …

News Release

Genes associated with Erdheim-Chester disease also linked to cancer

NHGRI researchers have identified new genes associated with the Erdheim-Chester disease (ECD) and some possible new therapies.

… and protect the body from infection. ECD has no standard therapy, although consensus guidelines for clinical … were available for molecular testing, half had BRAF V600E gene mutations, which is sometimes seen in colon cancer, lung … Residency Program, accumulation of histocytes, new gene discovery … NHGRI researchers have identified new genes …

News Release

NIH statement on new FDA-approved gene therapies for sickle cell disease

The U.S. National Institutes of Health (NIH) has long invested in basic genetics and genomics research, clinical trials, as well as translational medicine and social science studies, to advance our understanding of this widespread illness to help develop effective therapies.

… the U.S. Food and Drug Administration (FDA) approved two gene therapies for the treatment of sickle cell disease in … One of the new sickle cell treatments uses the CRISPR gene-editing system, a first for humans in the U.S. NIH … The Democratizing Education for Sickle Cell Disease Gene Therapy Project , a collaborative effort that aims to help …

Fact Sheet

Understanding gene therapy approaches

A variety of approaches target the blood stem cells to relieve or reduce your symptoms of sickle cell disease.

… It is important to understand the components of gene therapy, how these components differ and other treatment … cells to reduce your symptoms of sickle cell disease.  Gene therapies use your own stem cells, while bone marrow …