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Genetic Disorders
Sickle cell disease is a group of inherited red blood cells disorders.
Sickle cell disease is a group of inherited red blood cell disorders. … Sickle cell disease is the most common inherited blood …
News Release
NHGRI researchers seek help from people with Sickle Cell Disease to find the factors - environmental, social and genetic - that impact the severity of symptoms.
… Jeannine Mjoseth … People with sickle cell disease (SCD) can experience excruciating pain all over their …
Research at NHGRI
The Democratizing Education for Sickle Cell Disease Gene Therapy project utilizes a deliberative stakeholder-engagement approach to develop patient educational materials for sickle cell disease gene therapy.
… It is imperative that patients with sickle cell disease who are considering a gene therapy clinical trial …
Fact Sheet
Today, gene therapy for sickle cell disease is considered experimental and only available to you if you participate in a clinical trial.
… a trial may be dependent on: Your age. Your diagnosis of sickle cell disease. Your response to other treatments. Whether you are …
News Release
Researchers explored patients', parents' and physicians' perspectives on the use of CRISPR-Cas9 gene-editing to reverse sickle cell disease.
… on the use of CRISPR-Cas9 gene-editing to reverse sickle cell disease (SCD).  … A First-of-its-Kind Study CRISPR-Cas9 is a …
News Release
Researchers used whole genome sequence data to investigate the origin of the mutation that causes "sickling" of red blood cells.
… debate about the multi-centric or unicentric origin of the sickle allele in favor of the latter, with an estimate that … inherited with the sickle allele that may influence disease severity. 4. We raised the possibility that the … of the sickle allele will help clinicians redefine sickle cell subgroups and treat patients more effectively. This …
News Release
NHGRI researchers have released new educational materials to help the sickle cell disease community learn about gene therapies for the disease.
… navigate emerging developments in gene therapies for sickle cell disease. … New educational materials to help the sickle cell
Clinical Research
The INSIGHTS study is exploring sickle cell disease in adults.
… into Microbiome and Environmental Contributions to Sickle Cell Disease and Leg Ulcers Study (INSIGHTS) study is exploring …
Educational Resources
Sickle cell disease is a hereditary disease seen most often among people of African ancestry.
Sickle Cell DiseaseSickle cell disease is a hereditary disease seen most often …
Fact Sheet
Your mental and physical health are deeply connected. Mental health is important for your health and quality of life. 
… and/or trusted friend. Write about how you feel about your disease and how it affects your life. Reflect on why you … these other factors. Risks of participation Check out the sickle cell disease gene therapy FAQ Cost What costs does the …