Search Results
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.
… of Health (NIH) researchers have demonstrated in mice that gene therapy may be the best method for correcting … involved inserting a functional copy of the NPC1 gene into mice with the disease; the treated animals were then found to … types of Niemann-Pick disease; this study focused on mice that had been bred with a faulty NPC1 gene to model …
News Release
Researchers have found powerful clues to why certain processes and systems in the mouse are so different from those in people.
… evolutionary time and the genomic landscapes of humans and mice, an international group of researchers has found … better understand how similarities and differences between mice and humans are written in their genomes. … Their … used to control gene activity have many similarities in mice and humans, and have been conserved, or continued, …
Staff
Dr. Randy Chandler is an associate investigator in the Organic Acid Research Section of the Metabolic Medicine Branch in NHGRI.
… that AAV vectors can cause insertional mutagenesis in mice treated as neonates and that this toxicity is … cause of upregulation of oncogenes and tumorigenesis in mice treated with AAV. Importantly, his AAV integration … research has involved the use of gene editing and transgenic constructs to create murine models of human …
News Release
NIH researchers have uncovered a key factor in understanding the elevated cancer risk associated with gene therapy.
… associated with gene therapy. They conducted research on mice with a rare disease similar to one in humans, hoping … In prior MMA gene therapy studies, researchers showed that mice bred to develop the condition could be restored to … by AAV gene therapy injection shortly after birth. The mice in the study survived into adulthood and were free from …
Policy Issues
Scientific institutions are developing recommendations to support the responsible use of human gene-editing research.
… technology. For instance: NHGRI's Embryonic Stem Cell and Transgenic Mouse Core uses CRISPR to generate laboratory mice with specific gene alterations for the study of human … that NHGRI helped develop. It was designed to generate mice containing mutations in every gene of the mouse genome. …
News Release
TCF1 (for T cell factor-1) is essential for the creation and persistence of disease-fighting antibodies in the bloodstream.
… Genetic Disease Research Branch. "If we knock it out in mice, we don't get the T follicular helper cells, or very … In experiment after experiment, both in vitro and using transgenic mice infected with lymphocytic choriomeningitis virus (LCMV), …
Staff
Dr. Paul Liu is a senior investigator in NHGRI's Translational and Functional Genomics Branch.
​
… et al., Cell 1996). Using the Cbfb-MYH11 knock-in mice (in which human MYH11 was inserted into mouse Cbfb to … (Hyde et al., Blood 2010). With a clinically relevant transgenic mouse model, they demonstrated leukemogenic … include more functional studies of CBFβ-SMMHC with novel transgenic mouse models. The zebrafish is a useful model for …
Partner with NHGRI
NHGRI's technologies, inventions and discoveries are available for licensing.
Staff
Dr. Michael Erdos is an Associate Investigator in the Molecular Genetics Section of the Center for Precision Health Research.
… effect, it facilitated the generation of a humanized transgenic mouse model by inserting a human genomic DNA … successful at reducing the cardiovascular defects in young mice. In addition, it improved the cardiovascular histopathology when administered to older mice. This work supported the first clinical trial …
News Release
The zebrafish is a powerhouse tool that helps researchers better understand the genes that are implicated in disease.
… of human genetic diseases. There are projects that use transgenic fish with fluorescent markers to label specific … its staff has generated over 500 different mutant and transgenic lines. In addition, other NHGRI investigators have … generated large number of mutant lines. These mutant and transgenic fish make their home in the largest zebrafish …