From Promise to Practice: CRISPR-Based Therapies for Multiple Sclerosis
DOI:
https://doi.org/10.58445/rars.3246Keywords:
Immunology, CRISPR, Multiple Sclerosis, Central Nervous System, Autoimmune DiseaseAbstract
More than 2.3 million people have been diagnosed with multiple sclerosis (MS) worldwide (Doshi and Chataway). MS is an autoimmune condition caused by immune cells attacking the body’s own central nervous system (CNS). Currently, no treatments exist, and strategies are aimed at slowing the progression and symptoms of the disease. However, novel research has cast light on certain immune cells and genes responsible for the development and progression of MS. Given this, gene editing is a promising method to treat MS outright by editing the genes of cells implicated in MS. There has been admirable clinical success with editing genes in other diseases, like sickle cell anemia, which seeds hope for curing MS using gene editing. However, there are still major challenges to consider with editing the CNS. While there are new strategies in development, many safety and ethical factors must still be addressed before this technology can be used to treat patients. Overall, this paper presents a systematic review of the potential of CRISPR as a treatment method for MS. Some biological challenges include the presence of the blood-brain barrier (BBB), immune cell heterogeneity, and off-target effects, while technical challenges include the packaging and transportation efficiency to carry CRISPR. Parallelly, novel approaches to tackle these challenges will also be addressed, such that MS may be treated, rather than just managed.
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