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Therapeutic Impact of Exosomes on Huntington’s Disease

  • Sagnik Nag,
  • Subhrojyoti Ghosh,
  • Anuvab Dey,
  • Srijita Paul,
  • Rashmi Pejakala Raveendra

摘要

Huntington’s disease (HD) is a rare neurodegenerative disorder, which significantly disrupts muscle coordination and results in cognitive decline and psychiatric problems. HD is predominantly caused by a genetic mutation that results in the abnormal production of a protein called huntingtin, which leads to the degeneration of neurons in the brain. According to estimates, the worldwide prevalence of HD ranges from 5 to 10 cases per 100,000 people. There are currently no known cures for HD, and treatment options are limited. Currently, the existing treatments focus on symptom management and enhancing the quality of life for individuals impacted by the disease. In recent years, there has been an increase in research focused on HD, including efforts to better understand the disease’s underlying mechanisms and to develop new treatments. Due to the advent of advances made in drug delivery and pharmaceutical sciences, extracellular vesicles or exosomes have been widely explored for the treatment of an array of neurodegenerative disorders, including HD. Exosomes, small membrane-bound vesicles that are released by cells, have emerged as a promising therapeutic approach for HD. Exosomes can carry a variety of molecules, including proteins, RNA, and microRNA, which can regulate gene expression and cellular functions. Recent studies have suggested that exosomes may have neuroprotective effects and could potentially be used to deliver therapeutic molecules to the brain. Exosome-based therapies have several potential advantages for HD treatment. They can be engineered to target specific cells or tissues and can potentially provide a sustained release of therapeutic molecules. Additionally, since exosomes are derived from cells in the body, they may have lower immunogenicity and fewer side effects than traditional drugs. Thus, this chapter tends to extrapolate the therapeutic potentials of extracellular vesicles or exosomes against HD, considering all the associated challenges and toxicological implications.