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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.
… the VTS-270 compound has to be given for the life of the mouse. The team is now investigating if a combination of the …
News Release
NIH has awarded grants of more than $28 million aimed at deciphering the language of how and when genes are turned on and off.
… million aimed at deciphering the language of how and when genes are turned on and off. These awards emanate from the … of NIH. … "There is a growing realization that the ways genes are regulated to work together can be important for … role in some diseases. The investigators will use a mouse model to study genomic mechanisms underlying immune …
Talking Glossary
La interacción genes–ambiente se refiere a interrelación de los genes (y en términos más amplios, de la función del genoma) y el entorno fÃsico y social.
… Interacción genes-ambiente … La interacción genes–ambiente se refiere a interrelación de los genes (y en términos más amplios, de la función del genoma) y …
News Release
NIH researchers have uncovered a key factor in understanding the elevated cancer risk associated with gene therapy.
… safety parameter related to the AAV gene therapy in our mouse models that is critical to understand before we move to … performed additional experiments to detect where in the mouse genome the AAV vector delivered the corrected gene and … not insert where it would elevate the expression of nearby genes and it did not cause liver cancer. The researchers …
Talking Glossary
A cancer-susceptibility gene is a gene that, when changed (or mutated), gives an individual an increased risk for developing cancer
… have inherited mutations in certain cancer-susceptibility genes have a lifetime risk of cancer that is significantly …
News Release
NHGRI researchers are studying the link between Parkinson's disease, and a rare disorder, Gaucher disease, by cross-breeding mice with the disease mutations.
… controlling movement. Midbrain neurons from the new mouse model. The green shows the clumped alpha-synuclein. … In the study, the authors used a Parkinson's disease mouse model that carried extra copies of a mutated form of … is different about the cross-bred mice. Ultimately, this mouse model can be used to develop and evaluate new …
News Release
NHGRI researchers identified 59 genes or gene regions linked to canine athletics.
… researchers reached this conclusion after identifying 59 genes or gene regions linked to canine athletics, including … flow and pain perception. What terriers lacked in sporty genes, they made up for in genes associated with physical attributes such as their …
News Release
An expert panel from the Clinical Genome Resource (ClinGen) has critically reevaluated the scientific evidence for all 17 reported genes, disputing nine of the genes and revealing only three of the genes to be definitively associated with the most common form of the disease.
… Ph.D. … NIH-funded ClinGen panel also validates three genes believed to be associated with long QT syndrome … … specialists around the world had previously reported 17 genes to cause long QT syndrome, a little-known inherited … reevaluated the scientific evidence for all 17 reported genes, disputing nine of the genes and revealing only three …
Staff
Lisa Garrett is an associate investigator and director of the NHGRI Transgenic Mouse Core.
… is a staff scientist and director of the NHGRI Transgenic Mouse Core at the NIH. In 1984, she received her M.S. at … 1994 and completed her thesis, The Tissue - Specificity of Mouse Ets-1 Regulatory Region in a Transgenic Model. In 1994, … is sufficient and necessary for synovial joint formation. Genes Dev, 18(19):2404-17. 2004. [ PubMed ] Crabtree, J.S., …
Fact Sheet
Understanding gene therapy for sickle cell disease