Our Medical Impact

JAX research has contributed to many of today's medical treatments. 


Since 1929, JAX researchers have made significant advances in understanding human biology, and have directly contributed to the development of today's essential medical treatments. 

To date, 26 Nobel prizes are associated with Jackson Laboratory research, education programs and resources. JAX professor George Snell, Ph.D., won the 1980 Nobel Prize in Physiology or Medicine for providing an in-depth understanding of the immune system’s major histocompatibility complex, making organ transplants possible. Three graduates of the JAX Summer Student Program have gone on to win the Nobel Prize, and another two dozen Nobel Prizes have been awarded for research conducted using JAX® Mice.

Here are just a few JAX discoveries that have advanced clinical care around the world:

  • Elizabeth Russell’s work led to new treatments for blood and immunological diseases, including performing the first bone marrow transplant in a mammal.
  • In 1958, while examining a large testicular tumor on a mouse from the strain known as 129, Leroy Stevens noticed that it was composed of many kinds of tissue, including muscle, skin, bone and hair. In later studies, Stevens observed that the tumors, known as teratocarcinomas, produced not only the various kinds of tissues as expected, but also groups of undifferentiated cells with the capacity to grow into a wide range of tissue types. He dubbed the latter cells "pluripotent embryonic stem cells" and is acknowledged as the “father of stem cell research.”
  • Studies by Douglas Coleman led to the discovery of leptin, central to obesity and diabetes research. Leptin is also a treatment for rare cases of obese patients with a specific genetic disorder.
  • George Snell won the 1980 Nobel Prize in Physiology or Medicine for providing an in-depth understanding of the immune system’s major histocompatibility complex, making organ transplants possible.
  • Techniques now widely used in human fertility treatments, such as in vitro fertilization and cryopreservation (freezing) of embryos, were developed and refined over the past four decades at JAX to build and maintain the vast repository of mouse strains.
  • Se-Jin Lee pioneered the discovery of myostatin, a protein that inhibits muscle growth. His work has opened up new avenues to developing therapies to fight muscle loss due to diseases such as Duchenne’s muscular dystrophy or aging. Lee, whose research has relied on the use of mouse models of disease, joined the JAX faculty in 2017.
  • Edison Liu and colleagues found a molecular fingerprint of some of the most deadly cancers of women: a genomic configuration described as a tandem duplicator phenotype (TDP) that is significantly enriched in triple-negative breast cancer, serous ovarian cancer and endometrial carcinomas, and that responds to a specific chemotherapy (cisplatin).
  • Work by Cat Lutz resulted in the development of Spinraza®, the first medication used to treat Spinal Muscular Atrophy — a rare and fatal disease that causes spinal cord degeneration in infants. Similar methods are now being used to pursue therapeutics for more common diseases like Alzheimer’s, Parkinson’s and amyotrophic lateral sclerosis (ALS).



Two promising basic research discoveries at JAX are now entering the clinical trials pipeline:

  • New research from Simon John shows that adding vitamin B3 to the drinking water of mice predisposed to glaucoma can prevent the debilitating eye disease. The John lab is in discussion with a potential clinical partner for human trials.
  • Cyteir Therapeutics was founded to bring to clinical trials a new “genetic chemotherapy” approach to cancer treatment developed at The Jackson Laboratory. The Kevin Mills laboratory identified molecules that prevent repair of some cancer cells, providing an opportunity to target genetically defined cancers while significantly reducing the side effects of chemotherapy and potentially avoiding the development of treatment resistance.
  • Clinical trials using FcRn, based on work by JAX Professor Derry Roopenian, are progressing extremely well, especially for an autoimmune disease called hemolytic anemia.



These basic research findings hold promise for significantly advancing treatment options:

  • David Harrison demonstrated that treatment with rapamycin significantly lengthens the lives of aging mice and postpones the onset of the diseases of aging — the first intervention proven to extend the lifespan of a mammal. The findings are shedding light on the biological pathways involved in healthy aging.
  • David Serreze achieved a long-time goal in the diabetes research community of eliminating the B cells that begin the process of inducing type 1 diabetes. He successfully used a gene manipulation approach to identify a potential metabolic target.
  • Every cancer starts with a single cell, and Jennifer Trowbridge found a precise and reliable way — whole-genome profiling of open chromatin — to identify the kind of cell that leads to a given case of leukemia, a valuable key to cancer prognosis and outcome.
  • A research team led by Charles Lee identified two druggable targets for gastric cancer through a genomic molecular profiling technique, and validated the findings in mouse models capable of hosting human tumors.
  • Building off the success of Spinraza®, noted above, the JAX Rare and Orphan Disease Center is now working with a number of foundations to create and distribute better mouse models of various genetic diseases.
  • The FDA approved Mepsevii for use in pediatric and adult patients with mucopolysaccharidosis type VII (MPS VII), often referred to as Sly syndrome. The “Sly Syndrome” name is based on William Sly, MD, who first described MPSVII and worked with a JAX team to characterize a mouse model for the disease, which affects fewer than 150 patients worldwide.