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Fact Sheet
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 …
Fact Sheet
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
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 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
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
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 …
Clinical Research
MINI Section invites caregivers of children with mitochondrial disease to participate in a survey about the use of immune therapies in children with mitochondrial disease.
… … For More Information … Contacts … Use of Immunoglobulin Therapy in the Mitochondrial Disease Community … MINI Section …
Educational Resources
Gene regulation is the process of turning genes on and off.
… Gene Regulation … Gene regulation is the process of turning genes on and off. … Gene Expression, Genes, DNA, Transcription Factors … Gene regulation is the process used to control the timing, …
Fact Sheet
Your mental and physical health are deeply connected. Mental health is important for your overall health and quality of life.
… actions you may want to take as you think about receiving gene therapy. …  There are many unknowns about what to expect from gene therapy. Your thoughts, feelings, beliefs and attitudes …
Educational Resources
The gene is the basic physical unit of inheritance.
… Gene … The gene is the basic physical unit of inheritance. … Inheritance, Gene, Double Helix, DNA, DNA Sequence … The gene is considered the basic unit of inheritance. Genes are …