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Educational Resources
Translation is the process of translating the sequence of a messenger RNA (mRNA) molecule to a sequence of amino acids during protein synthesis.
… process of translating the sequence of a messenger RNA (mRNA) molecule to a sequence of amino acids during protein … Gene, Gene Expression, Genetic Code, Messenger RNA (mRNA), Protein, Ribosome … Translation, as related to … through which information encoded in messenger RNA (mRNA) directs the addition of amino acids during protein …
Educational Resources
A stop codon is a trinucleotide sequence within a messenger RNA (mRNA) molecule that signals a halt to protein synthesis.
… codon is a trinucleotide sequence within a messenger RNA (mRNA) molecule that signals a halt to protein synthesis. … … Gene Environment Interaction, Genetic Code, Messenger RNA (mRNA), Protein, Translation, Gene … A stop codon is a … nucleotides (a trinucleotide) in DNA or messenger RNA (mRNA) that signals a halt to protein synthesis in the cell. …
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
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 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 of gene therapy to make genetic changes in the bone marrow of sickle …
Fact Sheet
Early in the COVID-19 pandemic, researchers used state-of-the-art genomic sequencers to quickly sequence the SARS-CoV-2 virus.
… or a small, circular piece of DNA. Plasmids are used in mRNA vaccine production because they are easy to replicate … can be produced within a couple of weeks, allowing new mRNA vaccines to be tested and distributed rapidly. …  …  … such as in a test tube) protocols to create mRNA in a method that mimics our own biological processes. …
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.
… called Gaucher disease. ​ … Gaucher disease, drug therapy for Gaucher disease, Ellen Sidransky Ph.D., GBA gene …
Staff
Dr. Randy Chandler is an associate investigator in the Organic Acid Research Section of the Metabolic Medicine Branch in NHGRI.
… research focused on adeno-associated virus (AAV) gene therapy for the treatment of hemophilia A, a genetically … models have helped enable two clinical trials: one uses mRNA therapy and the other, AAV-mediated nuclease-free genome … approaches, such as AAV gene delivery, genome editing, and mRNA therapy, to cure rare genetic disorders. The primary …
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 …
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 …
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 …