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News Release
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.
… a decade of work and launch the first in-human gene therapy trial for children with a rare and devastating … longer a concern. This demonstrated to the U.S. Food and Drug Administration that they were ready to test the … and enough to move from the clinical trial phase to a new drug approval in two years. However, there was also a feeling …
Educational Resources
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 … Gene Mutation, Cancer Treatment, Healthcare … Gene therapy is a technique that uses a gene(s) to treat, prevent or cure a disease or medical disorder. Often, gene therapy works by adding new copies of a gene that is broken, …
News Release
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.
… Today, the U.S. Food and Drug Administration (FDA) approved two gene therapies for the … The Democratizing Education for Sickle Cell Disease Gene Therapy Project , a collaborative effort that aims to help … Researchers have worked hard to find a long-term, durable therapy for sickle cell disease. Research has enabled the use …
Fact Sheet
A variety of approaches target the blood stem cells to relieve or reduce your symptoms of sickle cell disease.
… transplants use the stem cells of a matched donor. Gene therapy  In gene therapy , your stem cells are changed by altering part of your genes. Types of gene therapy include gene addition and gene editing . There are …
News Release
Investigators with The Cancer Genome Atlas Research Network have identified new potential therapeutic targets for a major form of bladder cancer.
… therapies - second choices for treatments when the initial therapy does not work - and no approved targeted agents for … remodeling. Overall, the researchers identified potential drug targets in 69 percent of the tumors evaluated. They … affecting different organs look very similar. As targeted drug agents go through preclinical and clinical development, …
News Release
Doctors and researchers reunite with patient who received gene therapy for GM1 gangliosidosis.
… and researchers reunite with patient who received gene therapy for GM1 gangliosidosis. … In a sun-lit conference … after Jojo attended the last tea party, she received gene therapy for her rare disorder, GM1 gangliosidosis. … Only … within the altered protein.  But one approach — gene therapy — goes to the root of the problem. Instead of leaving …
Fact Sheet
Currently, there are different gene therapy approaches available through clinical trials.
… … To understand sickle cell disease and gene therapy , you should first know about blood cells and the … welcomes your feedback about the sickle cell disease gene therapy resources. Please email your comments or questions to … are meant to promote your general understanding of gene therapy for sickle cell disease. We encourage you to use …
Health FAQ
Discuss these questions with your doctor or health care provider.
… welcomes your feedback about the sickle cell disease gene therapy resources. Please email your comments or questions to … are meant to promote your general understanding of gene therapy for sickle cell disease. We encourage you to use … your healthcare provider or a clinical trial team. … Gene therapy is an experimental treatment that may improve the …
Educational Resources
Pharmacogenomics is a branch of pharmacology concerned with using DNA and amino acid sequence data to inform drug development and testing.
… with using DNA and amino acid sequence data to inform drug development and testing. … Amino Acids, Deoxyribonucleic … with using DNA and amino acid sequence data to inform drug development and testing. … Educational Resources …
News Release
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 … the most common treatment for ECD has been interferon , a drug that interferes with the division of cancer cells and …