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Educational Resources
Messenger RNA (abbreviated mRNA) is a type of single-stranded RNA involved in protein synthesis.
… Messenger RNA (mRNA) … Messenger RNA (abbreviated mRNA) is a type of single-stranded RNA involved in protein synthesis. … Messenger RNA, MRNA, Protein Synthesis, Cell Biology … Messenger RNA …
News Release
NIH researchers have uncovered a key factor in understanding the elevated cancer risk associated with gene therapy.
… the elevated cancer risk associated with gene therapy. They conducted research on mice with a rare disease … hoping their findings may eventually help improve gene therapy for humans. Researchers at the National Human Genome … into a cell. AAV viruses are uniquely suited for gene therapy applications. But one prior study did find an …
Fact Sheet
mRNA vaccines inject cells with instructions to generate a protein that is normally found on the surface of SARS-CoV-2, the virus that causes COVID-19.
… Key Points mRNA vaccines inject cells with instructions to generate a … system will recognize the virus and respond to it.   mRNA vaccines are safe and cannot alter your DNA, and you cannot get COVID-19 from the vaccine.   mRNA vaccines may seem to have arrived quickly, but this …
Fact Sheet
Understanding gene therapy for sickle cell disease
… the Democratizing Education for Sickle Cell Disease Gene Therapy Project, which is led by NHGRI in partnership with … 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
NIH researchers demonstrated that gene therapy may be the best method for correcting the single faulty gene that causes Niemann-Pick disease, type C1.
… (NIH) researchers have demonstrated in mice that gene therapy may be the best method for correcting the single … that causes Niemann-Pick disease, type C1 (NPC1). The gene therapy involved inserting a functional copy of the NPC1 gene … NPC1 protein to correct the cell defects.   The gene therapy involved inserting a functional copy of the NPC1 gene …
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.
… patients with sickle cell disease who are considering gene therapy fully understand the meaning and limitations of … 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 …
Fact Sheet
Today, gene therapy for sickle cell disease is considered experimental and only available to you if you participate in a clinical trial.
… 4: Preparation for stem cell collection (~90 days)   Gene therapy requires stem cells to be collected from your bone … collection. The process differs based on the type of gene therapy approach. The process does not always work. If it … month, you will have a follow-up visit to see how well the therapy is working and to track your overall health. Your …
Educational Resources
An anticodon is a trinucleotide sequence complementary to that of a corresponding codon in a messenger RNA (mRNA) sequence.
… to that of a corresponding codon in a messenger RNA (mRNA) sequence. … Codon, DNA, Transfer RNA, TRNA, Messenger RNA, MRNA, Complementary Sequence … A codon is a DNA or RNA … complementary to a corresponding codon in a messenger RNA (mRNA) sequence. Each time an amino acid is added to a growing …
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
Gene therapy is an experimental technique for treating disease by altering the patient's genetic material.
… Gene Therapy … Gene therapy is an experimental technique for treating disease by … Gene Mutation, Cancer Treatment, Healthcare … Gene therapy is a technique that uses a gene(s) to treat, prevent or cure a disease or medical disorder. Often, gene therapy works by adding new copies of a gene that is broken, …