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News Release
It’s teatime and doctors, researchers and patients are at the table. They celebrate a decade of work and launch the first in-human gene therapy trial for children with a rare and devastating disease, GM1 gangliosidosis.
… a decade of work and launch the first in-human gene therapy trial for children with a rare and devastating … Jojo was about to receive the first experimental gene therapy treatment for the rare disease, GM1 gangliosidosis. A … extended to the entire team on the day of the gene therapy trial. Photo Credit: Ernesto Del Aguila III, NHGRI. …
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 … through which information encoded in messenger RNA (mRNA) directs the addition of amino acids during protein …
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. …
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 … nucleotides (a trinucleotide) in DNA or messenger RNA (mRNA) that signals a halt to protein synthesis in the cell. …
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 …
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.
… Gaucher disease, drug therapy for Gaucher disease, Ellen Sidransky Ph.D., GBA gene …
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 … transplants use the stem cells of a matched donor. Gene therapy  In gene therapy , your stem cells are changed by altering part of … welcomes your feedback about the sickle cell disease gene therapy resources. Please email your comments or questions to …
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 … Chandler’s research has focused on the efficacy of gene therapy as a treatment for rare genetic disorders, such as … models have helped enable two clinical trials: one uses mRNA therapy and the other, AAV-mediated nuclease-free genome …
Fact Sheet
Your mental and physical health are deeply connected. Mental health is important for your overall health and quality of life.
… 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 can … for your mental and physical health as you consider a gene therapy clinical trial.  Focus on your thoughts and feelings. …
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 … If you are eligible and decide to receive  gene therapy, your clinical team and healthcare providers will …