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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.
… 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, …
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 …
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 …
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.
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… 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 …
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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 …
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 …