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Genomics and Medicine

Starting in 2019, the American Journal of Human Genetics has published an annual feature identifying ten key advances in applying genomic information to clinical care that were reported in the previous 12 months of published literature. The Genomic Medicine Working Group has authored these reviews, based on its broader effort to identify notable accomplishments in genomic medicine on a monthly basis. From this larger set of published accomplishments, the working group has continued to select ten papers annually to be highlighted as the most significant.

… feature identifying ten key advances in applying genomic information to clinical care that were reported in the … feature identifying ten key advances in applying genomic information to clinical care that were reported in the … feature identifying ten key advances in applying genomic information to clinical care that were reported in the …

Health FAQ

Sickle cell disease gene therapy FAQ

Discuss these questions with your doctor or health care provider.

… long hospital stays. It is important to obtain specific information about the clinical trial or therapy you are … ask to speak with a genetic counselor if you need more information. … Every person’s experience may be different. … issues, may arise. Visit our resource page for additional information on how to combat common issues in the caregiver …

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.

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.

… For example: NIH investments in genome sequencing technology have led to the costs of whole genome sequencing … than a couple of decades ago. Affordable sequencing technology is critical to the diagnosis of sickle cell … of the National Human Genome Research Institute … For more information, visit: Sickle cell disease gene therapy …

For Patients and Families

Genetic Disorders

A list of genetic, orphan and rare diseases under investigation by researchers at or associated with the National Human Genome Research Institute.

Fact Sheet

Your mental health and gene therapy

Your mental and physical health are deeply connected. Mental health is important for your overall health and quality of life.

Fact Sheet

Gene therapy patient education materials

Understanding gene therapy for sickle cell disease

… and biomedical research communities. They include helpful information and resources for people with sickle cell disease …

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.

Genetic Disorders

About Cystic Fibrosis

Cystic fibrosis is a genetic disease that causes the body to produce thick, sticky mucus that clogs the lungs, leads to infection, and blocks the pancreas.

… aeruginosa. Gene Database Speeds Research As the amount of information about CF grows, scientists have recognized the … their research findings. To facilitate this sharing of information, the Cystic Fibrosis Foundation funds Cystic … for data derived from gene expression studies. By pooling information, researchers hope to accelerate the process of …

Genetic Disorders

About Huntington's Disease

Huntington's disease is an inherited neurological illness causing involuntary movements, severe emotional disturbance and cognitive decline.

… pregnancy. Additional Resources for Huntington's Disease Information NINDS Huntington's Disease Information Page Medline Plus: Huntington's Disease …