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Talking Glossary
Alternative splicing is a cellular process in which exons from the same gene are joined in different combinations, leading to different, but related, mRNA transcripts.
… different combinations, leading to different, but related, mRNA transcripts. … Alternative splicing is a cellular … different combinations, leading to different, but related, mRNA transcripts. These mRNAs can be translated to produce … different combinations, leading to different, but related, mRNA transcripts. … Talking Glossary …
Research at NHGRI
The Metabolic Medicine Branch houses clinical and laboratory research programs that focus on the development of new therapies for patients with genetic disorders.
… and their research teams develop gene addition therapy and gene editing approaches to treat metabolic … of single cells and explore the side-effects of gene therapy. The MMB is invested in mentorship and supports …
News Release
NHGRI researchers have identified new genes associated with the Erdheim-Chester disease (ECD) and some possible new therapies.
… and protect the body from infection. ECD has no standard therapy, although consensus guidelines for clinical …
News Release
NHGRI researchers have released new educational materials to help the sickle cell disease community learn about gene therapies for the disease.
… The Democratizing Education for Sickle Cell Disease Gene Therapy Project, led by Vence Bonham Jr., J.D., NHGRI acting … disease and their support networks to learn about the gene therapy clinical trial process, its benefits and risks, … community information about what participating in a gene therapy clinical trial entails,” said Kiana Amini, a …
Talking Glossary
cDNA (short for copy DNA; also called complementary DNA) is synthetic DNA that has been transcribed from a specific mRNA through a reaction using the enzyme reverse transcriptase
… is synthetic DNA that has been transcribed from a specific mRNA through a reaction using the enzyme reverse … is synthetic DNA that has been transcribed from a specific mRNA through a reaction using the enzyme reverse … is synthetic DNA that has been transcribed from a specific mRNA through a reaction using the enzyme reverse …
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.
… a rational approach to drug discovery and personalizing therapy. Oregon Health and Science University (OHSU), …
Profile
After major advances in hematology, David Bodine retires with an eye towards mentorship and a garden
David Bodine, founder of the Hematopoiesis Section and chief of the Genetics and Molecular Biology Branch, advanced the fields of gene therapy and hematology in his 38 years at the NIH. Now, he plans to continue his contributions to science as an emeritus and through training the next generation of scientists.
… you see as the most exciting future opportunities in gene therapy? Bodine: When we founded the American Society of Gene Therapy, we wanted to make these therapies low cost and widely available. Now, 20 years later, gene therapy has been very successful for treating any number of …
Educational Resources
A repressor is a protein that turns off the expression of one or more genes.
… … Gene, Gene Expression, Gene Regulation, Messenger RNA (mRNA), Protein, Transcription … A repressor, as related to … gene(s), which prevents the production of messenger RNA (mRNA). Repressor proteins are essential for the regulation of …
News Release
Researchers have discovered that FGF21 is elevated in mice with liver disease that mimics the same condition in patients with methylmalonic acidemia.
… allowed them to study the response to liver-directed gene therapy and to compare the findings in patients after liver … "Our new markers can accurately predict how effective a therapy, whether cellular or genomic, might be for the … acidemia, medical genomics, genetic disorders, gene therapy, Charles Venditti … Researchers have discovered that …
Fact Sheets
Genetics refers to the study of genes and their roles in inheritance. Genomics refers to the study of all of a person's genes (the genome).
… of pharmacogenetics is to create an individualized drug therapy that allows for the best choice and dose of drugs. … is the breast cancer drug trastuzumab (Herceptin). This therapy works only for women whose tumors have a particular … Asked Questions about Pharmacogenomics ). Stem Cell Therapy Stem cells have two important characteristics. First, …