Neurofibromatosis type 1 (NF1) is an inherited neurological disorder that affects 1 in 3,500 people worldwide. It is caused by mutations in the NF1 gene, which encodes neurofibromin, a protein ...
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Mouse models for ultra-rare disorder could pave the way for nervous system gene editing therapies
Scientists at The Jackson Laboratory (JAX) have developed mouse models that survive premature death and enable pre-clinical testing of alternating hemiplegia of childhood (AHC), a devastating and ...
Scientists from UC Davis Center for Surgical Bioengineering, the MIND Institute and UC Berkeley’s Murthy Lab are developing ...
A new study reveals a critical vulnerability in a brain receptor targeted by a rare autoimmune disease. Researchers have ...
Researchers at the Jan and Dan Duncan Neurological Research Institute (NRI) at Texas Children’s Hospital and Baylor College of Medicine recently made an important advance in neurodegenerative disease ...
Mouse models of neurodevelopmental disorders possess unique, sex-specific metabolic dysfunctions, according to a new study in eNeuro. Understanding the unique metabolic effects of each disorder in ...
A Northwestern Medicine study has uncovered a promising new therapeutic approach for a rare genetic brain disorder, according ...
Future Neurology. 2014;9(1):67-75. While differences in strain genetic backgrounds have contributed to variation in experimental results across different disease areas, these observations have also ...
A new “atlas” developed by researchers at Duke University School of Medicine, University of Tennessee Health Science Center, and the University of Pittsburgh will increase precision in measuring ...
Markus Terrey, a neuroscientist at The Jackson Laboratory’s Rare Disease Translational Center, led the development of the new AHC mouse models—breakthrough tools that are helping researchers ...
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