Epigenomics of neurological disorders

Olga Khorkova, Jane Hsiao, Claes Wahlestedt

Research output: Chapter in Book/Report/Conference proceedingChapter


Neurological disorders, including Alzheimer’s and Parkinson’s diseases, Dravet syndrome, amyotrophic lateral sclerosis, Angelman syndrome, fragile X mental retardation, spinal muscular atrophy, and many others, affect a significant part of world population. The proportion of familial cases in many of these disorders is small, indicating a significant role of environment, de novo mutations, multigenic predispositions, and epigenetic changes in their pathogenesis. Epigenetic changes in particular have been the subject of intense investigation in recent years. Besides epigenetic DNA and histone modification enzymes, long noncoding RNAs (lncRNAs) are emerging as essential components of epigenetic regulation. lncRNAs represent a major interest in the drug development process because they provide access to previously “undruggable” gene targets. Furthermore, lncRNA can be modulated using oligonucleotide-based compounds that have a shortened development pipeline. The synergy of the novel disease mechanisms (epigenetics), new therapeutic target class (lncRNA), an optimal therapeutic modality (oligonucleotide-based compounds), and unmet medical need in sporadic cases of major neurological disorders fuels intensive research in this area. We briefly review these studies.

Original languageEnglish (US)
Title of host publicationRosenberg’s Molecular and Genetic Basis of Neurological and Psychiatric Disease
Subtitle of host publicationVolume 1
Number of pages18
ISBN (Electronic)9780128139554
StatePublished - Jan 1 2020
Externally publishedYes


  • Alzheimer’s disease
  • Amyotrophic lateral sclerosis
  • Angelman syndrome
  • AntagoNAT
  • Dravet syndrome
  • Epigenetics
  • Fragile X mental retardation
  • LncRNA
  • Neurological disorders
  • Oligonucleotides
  • Parkinson’s disease
  • Spinal muscular atrophy

ASJC Scopus subject areas

  • Medicine(all)


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