Abstract
Rare neurodegenerative disorders encompass a diverse group of conditions characterized by the progressive degeneration of the nervous system. Usually, a combination of genetic, biochemical, and clinical features characterizes these disorders. An overview, classification, and investigation of the genetic mutations and variants linked to rare neurodegenerative diseases are included in this chapter on genetic therapy for these diseases. The article discusses novel approaches to treating genetic illnesses, including gene therapy, CRISPR-based interventions, and RNA-based therapeutics. It includes case studies and particular instances of effective genetic treatments as well as ongoing clinical trials. In addition, difficulties and moral issues are discussed, covering issues like delivery strategies, side effects, and moral questions about the use of gene editing in the treatment of various illnesses. Lastly, future outlooks and conjectures regarding possible developments, avenues for further study, and the prospects for genetic therapies in the treatment of uncommon neurodegenerative illnesses are discussed.