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Review
. 2024 Mar 6:6:1304110.
doi: 10.3389/fgeed.2024.1304110. eCollection 2024.

Epigenetic editing for autosomal dominant neurological disorders

Jennifer J Waldo  1 , Julian A N M Halmai  1 , Kyle D Fink  1
Affiliations
Review

Epigenetic editing for autosomal dominant neurological disorders

Jennifer J Waldo et al. Front Genome Ed. .

Abstract

Epigenetics refers to the molecules and mechanisms that modify gene expression states without changing the nucleotide context. These modifications are what encode the cell state during differentiation or epigenetic memory in mitosis. Epigenetic modifications can alter gene expression by changing the chromatin architecture by altering the affinity for DNA to wrap around histone octamers, forming nucleosomes. The higher affinity the DNA has for the histones, the tighter it will wrap and therefore induce a heterochromatin state, silencing gene expression. Several groups have shown the ability to harness the cell's natural epigenetic modification pathways to engineer proteins that can induce changes in epigenetics and consequently regulate gene expression. Therefore, epigenetic modification can be used to target and treat disorders through the modification of endogenous gene expression. The use of epigenetic modifications may prove an effective path towards regulating gene expression to potentially correct or cure genetic disorders.

Keywords: CRISPR/Cas9; autosomal dominant; epigenetics; gene regulation; neurodenerative diseases.

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Conflict of interest statement

The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest.

Figures

FIGURE 1
FIGURE 1
Examples of gain of function pathogenesis. Created with Biorender.com.
FIGURE 2
FIGURE 2
Different DNA-binding domains and their DNA targeting structure. Created with Biorender.com.
FIGURE 3
FIGURE 3
Therapeutic approaches to the treatment of neurogenerative disorders using dCas9 epigenetic editors. Created with Biorender.com.
See this image and copyright information in PMC

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