The Complexity of Rare Disease
Learn about the complexity of rare disease and the unique difficulty it presents to patients and families by exploring the patient journey—from diagnosis to research to drug development.
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Rare Disease
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Exploring Rare Disease Through Translational Research
Learn how mouse models help researchers disentangle the complexity of rare disease and help drive preclinical drug development.
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JAX Tech Talk Episode 62: Let’s Talk About the Uncommon: Rare Diseases (Feb. 7, 2023)
February 7, 2023 - In this episode, we discuss the importance of studying rare diseases and the critical preclinical steps leading to diagnosis and treatment development.
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HuMouse models for the influence of genetic variation on the immune control of Epstein Barr
Christian Münz, Ph.D. - Professor and Co-director of the Institute of Experimental Immunology, University of Zürich, Switzerland Talk Title: Humanized mouse models for the influence of genetic variati
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A View on ALS Drug Discovery - Learnings From the Front Lines of Translational Disease Modeling
Join Cat Lutz, Ph.D., Senior Director In Vivo Pharmacology & Mouse Repository at JAX as she'll discuss neurodegenerative diseases, rare diseases, and her group’s latest work in preclinical studies usi
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CD34+ Hematopoietic Stem Cell Humanized Mice: The Basics
Frequently Asked Questions about CD34+ HSC Humanized Mice Humanized mouse models serve as an innovative and cost-effective platform to simulate trials, evaluate multiple drugs alone or in...
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Mouse Model Generation: From Idea to Data
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Proficiency with Larger Knockins
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Mouse Model of Niemann-Pick Type C Disease Reveals the Correlation Between Neuronal Cell Death and Motor Deficits
For decades, animal models have proven especially useful in revealing molecular changes for various conditions, including those in neurobiology. When researchers want to test existing or novel...
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Women in science: Huda Zoghbi discovered the genetic basis of Rett syndrome
Dr. Huda Zoghbi was born Huda El Hibri in 1955 in Beirut, Lebanon. Her father owned a business manufacturing olive oil and soap, and her mother stayed at home to raise Huda and her four siblings....
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Understanding the connection between heart and nerves in a rare form of SMA
SMARD is a disease that affects nerve cells. These are the cells that relay information from the brain through the spinal cord to different muscles in your body. This is kind of like the wires...
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Collectively, rare diseases are not so rare
Bruce Curran, right, speaks with JAX researcher David Bergstrom, Ph.D., during a Rare Disease Day 2016 event at the Maine State House. Curran is battling Inclusion Body Myositis, a rare...
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Light Up Your Research: Use and Selection of Optogenetic Mouse Models
This video reviews the basics of optogenetic mouse models and how researchers are using them to investigate neural circuitries underlying complex behaviors and neural diseases.
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Tips & Tricks for Care of NSG Mice
The NSG™ mouse model is used by investigators worldwide to advance research in cancer, infectious diseases, and immunological disorders. Watch this video to learn how to care for NSG™ Mice.
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The Versatile Mouse Model for Rare Disease Research
More than 7,000 rare (or orphan) diseases have been described so far and affect over 350 million people worldwide. Unfortunately, less than 10% of these diseases have an approved treatment, and...
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CRISPR/Cas9 Explained
Did you know that CRISPR engineering can sometimes yield more than the allele you originally intended? Learn more about mosaicism, how to resolve it, and other CRISPR basics in this video.
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Disaster Proofing Your Knowledge House: Tips to prepare your in vivo research for the worst
We’ve all heard the famous Ben Franklin quote, “if you fail to plan, you are planning to fail,” and any researcher knows, he was right. Planning is an essential part of any study: you plan for...
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NSG Models: The Most Versatile Immunodeficient Strain
View the suite of next generation NSG models.
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Strategies To Minimize Genetic Drift And Maximize Experimental Reproducibility In Mouse Research
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Know Thy Mouse: Variability in Aged Mice
Before undertaking a large study, it’s very beneficial to understand the biology of the model you are going to work with. This is particularly important...
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