Review

Epigenome Editing: Emerging Tools, Therapeutic Applications, and Challenges in Human Disease Treatment

Number: 35 March 5, 2026
  • Maryam Mirahmadi
  • Nader Hashemi
  • Sayed Hassan Tabatabaee
  • Forough Shams
  • Yong Teng
  • Azam Rahimpour *

Epigenome Editing: Emerging Tools, Therapeutic Applications, and Challenges in Human Disease Treatment

Abstract

Epigenetic modifications, including histone alterations, non-coding RNA interactions, and DNA methylation, regulate gene expression without altering the underlying DNA sequence. These modifications are essential for normal biological processes; however, their aberrant regulation is linked to numerous life-threatening disorders. Genome editing nucleases such as zinc finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs), and CRISPR/Cas systems offer promising tools for the precise correction of epigenetic abnormalities. This review explores epigenetic mechanisms, genome editing technologies for epigenetic modulation, and their applications in disease contexts, such as cancer and neurodegeneration, with reference to both in vitro and in vivo studies demonstrating therapeutic potential. For instance, aberrant histone acetylation and methylation patterns are frequently observed in cancer. Abnormal DNA methylation and disruptions in histone modifications have been implicated in neurological disorders, such as Alzheimer’s and Huntington’s disease. Although ZFNs and TALENs are foundational tools, their use has been limited by challenges in protein engineering and nonspecific targeting. CRISPR/Cas systems have become a versatile platform. Catalytically inactive Cas9 (dCas9) can be fused to epigenetic editing domains, such as histone deacetylases and DNA methyltransferases, to precisely regulate gene expression. For example, dCas9 has been used to reactivate the BRCA1 tumor suppressor gene in cancer cells. Although epigenetic editing holds significant promise in biomedical research and precision medicine, several challenges remain. These include unintended epigenetic alterations, the efficient delivery of editing tools to target cells, and limited in vivo validation. Future studies using animal models are essential to evaluate the translational potential and clinical applicability of this approach.

Keywords

Supporting Institution

Work supported by Ministry of Economy and Competitiveness (MEC) grants from Spanish government (“EpiFarm”: AGL2013–41047–R) and Aquagenomics (CDS2007-0002) to FP LR was supported by an Epifarm contract.

Project Number

Grant Number 29763, Ethics Code: IR.SBMU.RETECH.REC.1400.655

Ethical Statement

Not applicable

Thanks

The authors wish to thank Cellular and Molecular Biology Research Center, Shahid Beheshti University of Medical Sciences, Tehran, Iran, and School of Advanced Technologies in Medicine, Shahid Beheshti University of Medical Sciences, Tehran, Iran, for their support.

References

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Details

Primary Language

English

Subjects

Epigenetics

Journal Section

Review

Authors

Sayed Hassan Tabatabaee This is me
0009-0008-7162-0860
Iran

Yong Teng This is me
0000-0002-1856-7289
United States

Azam Rahimpour * This is me
0000-0002-3296-5881
United States

Early Pub Date

November 25, 2025

Publication Date

March 5, 2026

Submission Date

February 11, 2025

Acceptance Date

October 20, 2025

Published in Issue

Year 2026 Number: 35

APA
Mirahmadi, M., Hashemi, N., Tabatabaee, S. H., Shams, F., Teng, Y., & Rahimpour, A. (2026). Epigenome Editing: Emerging Tools, Therapeutic Applications, and Challenges in Human Disease Treatment. Biotech Studies, 35, 1830114. https://doi.org/10.38042/biotechstudies.1830114
AMA
1.Mirahmadi M, Hashemi N, Tabatabaee SH, Shams F, Teng Y, Rahimpour A. Epigenome Editing: Emerging Tools, Therapeutic Applications, and Challenges in Human Disease Treatment. Biotech Studies. 2026;(35):1830114. doi:10.38042/biotechstudies.1830114
Chicago
Mirahmadi, Maryam, Nader Hashemi, Sayed Hassan Tabatabaee, Forough Shams, Yong Teng, and Azam Rahimpour. 2026. “Epigenome Editing: Emerging Tools, Therapeutic Applications, and Challenges in Human Disease Treatment”. Biotech Studies, nos. 35: 1830114. https://doi.org/10.38042/biotechstudies.1830114.
EndNote
Mirahmadi M, Hashemi N, Tabatabaee SH, Shams F, Teng Y, Rahimpour A (March 1, 2026) Epigenome Editing: Emerging Tools, Therapeutic Applications, and Challenges in Human Disease Treatment. Biotech Studies 35 1830114.
IEEE
[1]M. Mirahmadi, N. Hashemi, S. H. Tabatabaee, F. Shams, Y. Teng, and A. Rahimpour, “Epigenome Editing: Emerging Tools, Therapeutic Applications, and Challenges in Human Disease Treatment”, Biotech Studies, no. 35, p. 1830114, Mar. 2026, doi: 10.38042/biotechstudies.1830114.
ISNAD
Mirahmadi, Maryam - Hashemi, Nader - Tabatabaee, Sayed Hassan - Shams, Forough - Teng, Yong - Rahimpour, Azam. “Epigenome Editing: Emerging Tools, Therapeutic Applications, and Challenges in Human Disease Treatment”. Biotech Studies. 35 (March 1, 2026): 1830114. https://doi.org/10.38042/biotechstudies.1830114.
JAMA
1.Mirahmadi M, Hashemi N, Tabatabaee SH, Shams F, Teng Y, Rahimpour A. Epigenome Editing: Emerging Tools, Therapeutic Applications, and Challenges in Human Disease Treatment. Biotech Studies. 2026;:1830114.
MLA
Mirahmadi, Maryam, et al. “Epigenome Editing: Emerging Tools, Therapeutic Applications, and Challenges in Human Disease Treatment”. Biotech Studies, no. 35, Mar. 2026, p. 1830114, doi:10.38042/biotechstudies.1830114.
Vancouver
1.Maryam Mirahmadi, Nader Hashemi, Sayed Hassan Tabatabaee, Forough Shams, Yong Teng, Azam Rahimpour. Epigenome Editing: Emerging Tools, Therapeutic Applications, and Challenges in Human Disease Treatment. Biotech Studies. 2026 Mar. 1;(35):1830114. doi:10.38042/biotechstudies.1830114


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