Outline of Our Project

Introduction: 45 seconds

  • How many people are affected by autoimmune diseases:
    • Approximately 50 million Americans, 20 percent of the population or one in five people, suffer from autoimmune diseases
    • Women are more likely than men to be affected; some estimates say that 75 percent of those affected–some 30 million people–are women.
  • How many people are affected by multiple sclerosis:
    • 947,000 people in the US and 2.3 million people worldwide

Healthcare costs:

  • “The average annual cost per patient for the country was $29,339 (2010), $20,956 (2011), $23,892 (2012), $24,148 (2013), and $22,688 (2014). Drug therapy represented 86.1% of the total cost.” https://www.ncbi.nlm.nih.gov/pubmed/29605798 (link provided, new information gained for this post)

Other background information:

  • affects the nervous system
    • Brain
    • demyelination of nerves
    • immune system attacks the Myelin sheath and oligodendrocyte cells surrounding/protecting your peripheral and central nervous system
    • cognitive and physical impairments

Current Technology/Treatment: 75 seconds

Current Technology:

  • Gene Therapy: MS itself is not genetic; but there are genetic risk factors involved in the disease. Researchers currently have found 159 variants of genes that are potentially linked to MS
  • modified microglia: adapted the microglia to become anti-inflammatory, creating an environment where remyelination was possible
  • t-cell therapy: certain vaccines containing certain antigens have been tested in mice
  • ability to convert astrocytes within the CNS into oligodendroglia in vivo in order to combat the myelin destruction associated with this disease by forcing the expression of the miR-302/367 cluster in astrocytes

Why isn’t it good enough?

  • Many of the current treatments are not necessarily focused on treating MS but instead are adapted versions of treatments for other diseases
  • The current gene therapy has not gotten beyond identifying certain variations of genes that amplify the risk factor of getting the disease. There has not been further action or study as to how these could be edited out.
  • Modifying microglia as well as converting oligodendroglia into astrocytes only helped create better conditions; did not solve/eliminate the disease
  • Current t-cell therapy including vaccines caused allergic reactions

Your Technology: 3 minutes

How it works:

  • Gene therapy and gene editing could remove the genes causing MS directly from the cells while giving the cells regenerative properties that could help safeguard them from developing MS in the future.
  • Gene editing could also be used to modify the cells in the body that are responsible for t-cell production to stop them from producing t-cells that attack the body/carry MS?
  • Also possibly research into the Deinococcus radiodurans bacterium and applying genes that allow it to repair itself to neurons

Why it’s better:

  • It is specific to MS, but also has potential applications to other autoimmune diseases
  • Potentially less expensive: The main cost of treating MS comes from lifelong expenses for drugs and medications. Gene editing may reduce these costs by offering a one-time or short-term treatment.


  • Possible information on T-cell therapy from Dr. Susan Koegel and her research with T-cell development and function (still waiting on email response)

Things that are an issue:

  • There is not any work being done on adults for cell-therapy (CRISPR/Cas9, Car T-cells) as it would be very difficult to alter the genome in every single adult cell. In addition, cells in which the genome has not been altered would replicate alongside the altered cells. The CRISPR/Cas9 enzyme most probably wouldn’t be able to infiltrate all the cells targeted with one patient session, and therefore it is unknown how many gene therapy treatments would be needed which could be expensive and exhaustive.
  • Some ideas to fix this: We could only focus on a few specific type of cells such as the myelin sheath cells, oligodendrocyte cells, bone marrow cells that make T-cells, and the T-cells that attack the nervous system. We could then identify the specific genes that produce a negative immune response and change only the genomes in those types of cells.

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