Rare and Orphan Disease Mouse Models
Search for mouse models by human disease term.
Rare and orphan diseases affect fewer than 200,000 individuals in the United States, and nearly 700,000 rare diseases have been described to date. The Jackson Laboratory offers the world's largest collection of spontaneous, targeted, and transgenic mutant mouse strains, hundreds of which model rare diseases.
Search for mouse models by human disease term.
Discover helpful tools for advancing your rare and orphan disease research.
The Rare and Orphan Disease Center brings together global mouse resources and the expertise of scientists at the Jackson Laboratory for a start-to-finish approach to model development.
At The Jackson Laboratory’s Rare and Orphan Disease Center, we are committed to helping CdLS families along their rare...
In honor for International Myotonic Dystrophy Awareness Day 2021, JAX's Laurent Bogdanik discusses the disease and potential...
JAX Professor Robert Burgess, Ph.D., investigates the genetic and molecular mechanisms underlying peripheral nerve degeneration in CMT, and...
Chris Brannigan is preparing to take off his boots and walk nearly 1200 miles down the eastern seaboard of the United...
Rare and Orphan Disease Research Tools and Online Resources
Resources for people who are interested in learning more about rare diseases, clinical trials, and research programs.
What is a mouse model? Find out in this Minute to Understanding from the Jackson Laboratory!
This in-person course is a modern survey of heredity, disease, genetics in experimental animals and humans, and molecular...
Efficacy studies are performed by JAX on mouse models for these diseases: C57BL10.mdx and D2.mdx for DMD, DyW for CMD1A, and A/J for...
JAX distributes well-characterized, preclinical mouse models to accelerate drug discovery for rare and orphan diseases.
We engineer new models and enhance existing models through genetic standardization and characterization to ensure reproducibility of data...
We partner with foundations, pharmaceutical and biotech companies, and other scientists to research treatments for uncommon...
Rare disease families and experts offer advice on how to navigate the rare disease odyssey.
Families have banded together from across the globe to make awareness and research for SMARD a reality.
Did you know that almost all of us have the sort of genetic mutations that cause rare genetic diseases?
Using the right mouse model can reveal various biological intricacies and may lead to potential therapeutic treatments. In a recent study,...
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