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Understanding gene therapy for sickle cell disease
… Sickle cell disease is the most common inherited blood disorder in … are only a handful of options to manage or treat sickle cell disease.  Now, researchers are developing new and emerging gene therapies to treat sickle cell disease. To help navigate these new therapies, more than …
News Release
NIH awards more than $64 million to for a database of human cellular responses called the Library of Integrated Network-based Cellular Signatures or LINCS.
… Cellular Signatures (LINCS). Discovering such cell responses will improve scientists' understanding of cell … molecular and biochemical responses, and use computer analyses to uncover common patterns in these responses - … opportunities and gaps in biomedical research that no single NIH institute could tackle alone, but that the agency …
Educational Resources
HapMap (short for "haplotype map") is the nickname of the International HapMap Project, an international project that seeks to relate variations in human DNA sequences with genes associated with health.
… health. … International HapMap Project, Haplotype, SNPs, Single Nucleotide Polymorphisms, Chromsomes … HapMap (short … can refer to a combination of alleles or to a set of single nucleotide polymorphisms (SNPs) found on the same …
Talking Glossary
Mosaicism refers to the presence of cells in a person that have a different genome from the body’s other cells.
… later in development. Mosaicism can affect any type of cell and does not always cause disease. … Mosaicism refers to …
Educational Resources
A haplotype is a set of DNA variations, or polymorphisms, that tend to be inherited together.
… tend to be inherited together. … Haplotype, Chromosomes, Single Nucleotide Polymorphisms, SNPs, International HapMap …
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.
… approved two gene therapies for the treatment of sickle cell disease in patients 12 years and older. About 100,000 … and millions of people around the world have sickle cell disease , a hereditary disease common among those whose … technology is critical to the diagnosis of sickle cell disease.  NIH researchers successfully edited the …
News Release
Learn what happens when two genetic neighborhoods merge in brain tumor cells after a gene controlled by a gene-control switch turned on a cancer-growth gene.
News Release
NHGRI researchers asked patients, parents and physicians in the sickle cell disease community (SCD) what they wanted and needed to know about genome editing to make informed decisions about participating in genome-editing clinical trials.
… asked patients, parents and physicians in the sickle cell disease community what they wanted and needed to know … treatments, which appear to provide a potential for sickle cell, are among the most widely publicized medical advances … of the Bioethics Core at NHGRI. “By talking to sickle cell disease stakeholders ahead of time, we can learn more …
Talking Glossary
A gamete is a reproductive cell of an animal or plant.
… Gamete … A gamete is a reproductive cell of an animal or plant. … A gamete is a reproductive cell of an animal or plant. In animals, female gametes are … called sperm. Ova and sperm are haploid cells, with each cell carrying only one copy of each chromosome. During …
News Release
A team from the NIH has surmounted a major obstacle to testing potential drug therapies for a rare, genetic condition called Gaucher disease.
… spleen, bone marrow and nervous system. The main type of cell that is affected by accumulated glucocerebroside is the macrophage, a type of white blood cell. Macrophages are cellular housekeepers that dispose of cell debris and pathogens. According to the study, …