# The Future of Epigenetics: Emerging Technologies and Clinical Applications

**Authors:** Kavita A. Iyer, Rumiana Koynova-Tenchov, Janet M. Sasso, Trupti Thite, Yi Deng, Qiongqiong Angela Zhou

PMC · DOI: 10.1021/acsptsci.5c00729 · ACS Pharmacology & Translational Science · 2026-02-25

## TL;DR

Epigenetics is transforming medicine by linking gene regulation to environmental factors and disease, with new technologies and therapies showing promise across multiple conditions.

## Contribution

This report highlights recent transformative advances in epigenetic research and clinical translation using data from the CAS Content Collection.

## Key findings

- Environmental epigenetics shows how external factors induce heritable changes with transgenerational health implications.
- Cancer research leads epigenetic studies, with 13 FDA-approved drugs targeting hematological malignancies.
- The clinical pipeline includes 37 ongoing trials expanding into metabolic, neurological, and inflammatory disorders.

## Abstract

Epigenetics, the
study of heritable changes in gene expression
that do not involve alterations to the DNA sequence, has emerged as
a transformative field in biology and medicine, revealing how gene
expression is modulated in response to internal and external cues.
Its applications span from understanding fundamental biological processes
and disease mechanisms to developing novel diagnostics and therapies,
making it a cornerstone of modern biomedical research. This report
explores data from the CAS Content Collection to examine recent transformative
advances in epigenetic research, highlighting technological innovations
that are revolutionizing our understanding of gene regulation and
therapeutic applications. Environmental epigenetic research shows
strong interest, demonstrating how external factors induce heritable
epigenetic changes with implications for transgenerational inheritance.
These findings bridge environmental exposures with health outcomes
across generations, informing public health strategies and personalized
medicine approaches. Disease applications span multiple pathologies,
with cancer research leading epigenetic studies. Emerging applications
in aging research, metabolic diseases, and autoimmune conditions demonstrate
the field’s expanding clinical relevance. Clinical translation
has achieved significant success, with 13 FDA-approved epigenetic
drugs primarily targeting hematological malignancies through HDAC
inhibitors (6 drugs) and DNMT inhibitors (2 drugs). The robust clinical
pipeline includes 37 ongoing trials across novel targets, with encouraging
diversification beyond oncology into metabolic, neurological, and
inflammatory disorders.

## Linked entities

- **Diseases:** cancer (MONDO:0004992)

## Full-text entities

- **Genes:** PSG2 (pregnancy specific beta-1-glycoprotein 2) [NCBI Gene 5670] {aka CEA, PSBG2, PSG1}, MIR326 (microRNA 326) [NCBI Gene 442900] {aka MIRN326, hsa-mir-326, mir-326}, GDE1 (glycerophosphodiester phosphodiesterase 1) [NCBI Gene 51573] {aka 363E6.2, MIR16}, HDAC6 (histone deacetylase 6) [NCBI Gene 10013] {aka CPBHM, HD6, JM21, KDAC6, PPP1R90}, MAPT (microtubule associated protein tau) [NCBI Gene 4137] {aka DDPAC, FTD1, FTDP-17, MAPTL, MSTD, MTBT1}, F2R (coagulation factor II thrombin receptor) [NCBI Gene 2149] {aka CF2R, HTR, PAR-1, PAR1, TR}, TNF (tumor necrosis factor) [NCBI Gene 7124] {aka DIF, IMD127, TNF-alpha, TNFA, TNFSF2, TNLG1F}, IDH2 (isocitrate dehydrogenase (NADP(+)) 2) [NCBI Gene 3418] {aka D2HGA2, ICD-M, IDH, IDH-2, IDHM, IDP}, CRBN (cereblon) [NCBI Gene 51185] {aka MRT2, MRT2A}, BRD3 (bromodomain containing 3) [NCBI Gene 8019] {aka FSHRG2, ORFX, RING3L}, DNMT1 (DNA methyltransferase 1) [NCBI Gene 1786] {aka ADCADN, AIM, CXXC9, DNMT, HSN1E, MCMT}, AHRR (aryl hydrocarbon receptor repressor) [NCBI Gene 57491] {aka AHH, AHHR, bHLHe77}, SEPTIN9 (septin 9) [NCBI Gene 10801] {aka AF17q25, MSF, MSF1, PNUTL4, SEPT9, SINT1}, MIR126 (microRNA 126) [NCBI Gene 406913] {aka MIRN126, miRNA126, mir-126}, DNMT3A (DNA methyltransferase 3 alpha) [NCBI Gene 1788] {aka DNMT3A2, HESJAS, M.HsaIIIA, TBRS}, EP300 (EP300 lysine acetyltransferase) [NCBI Gene 2033] {aka KAT3B, MKHK2, RSTS2, p300}, HNMT (histamine N-methyltransferase) [NCBI Gene 3176] {aka HMT, HNMT-S1, HNMT-S2, MRT51}, F2RL3 (F2R like thrombin or trypsin receptor 3) [NCBI Gene 9002] {aka PAR4}, BDNF (brain derived neurotrophic factor) [NCBI Gene 627] {aka ANON2, BULN2}, FMR1 (fragile X messenger ribonucleoprotein 1) [NCBI Gene 2332] {aka FMRP, FRAXA, POF, POF1}, BRD9 (bromodomain containing 9) [NCBI Gene 65980] {aka LAVS3040, PRO9856, SMARCI2}, BRD2 (bromodomain containing 2) [NCBI Gene 6046] {aka BRD2-IT1, D6S113E, FSH, FSHRG1, FSRG1, NAT}, PRDM9 (PR/SET domain 9) [NCBI Gene 56979] {aka KMT8B, MEISETZ, MSBP3, PFM6, ZNF899}, TUBA1B (tubulin alpha 1b) [NCBI Gene 10376] {aka K-ALPHA-1}, TET1 (tet methylcytosine dioxygenase 1) [NCBI Gene 80312] {aka CXXC6, LCX, bA119F7.1}, APC (APC regulator of Wnt signaling pathway) [NCBI Gene 324] {aka BTPS2, DESMD, DP2, DP2.5, DP3, GS}, MECP2 (methyl-CpG binding protein 2) [NCBI Gene 4204] {aka AUTSX3, MRX16, MRX79, MRXS13, MRXSL, PPMX}, NOS3 (nitric oxide synthase 3) [NCBI Gene 4846] {aka EC-NOS, ECNOS, MYMY8, NOSIII, cNOS, eNOS}, INS (insulin) [NCBI Gene 3630] {aka IDDM, IDDM1, IDDM2, ILPR, IRDN, MODY10}, IRS1 (insulin receptor substrate 1) [NCBI Gene 3667] {aka HIRS-1}, SREBF1 (sterol regulatory element binding transcription factor 1) [NCBI Gene 6720] {aka HMD, IFAP2, SREBP1, bHLHd1}, ABCA1 (ATP binding cassette subfamily A member 1) [NCBI Gene 19] {aka ABC-1, ABC1, CERP, HDLCQTL13, HDLDT1, HPALP1}, KCNQ1 (potassium voltage-gated channel subfamily Q member 1) [NCBI Gene 3784] {aka ATFB1, ATFB3, JLNS1, KCNA8, KCNA9, KVLQT1}, PPARGC1A (PPARG coactivator 1 alpha) [NCBI Gene 10891] {aka LEM6, PGC-1(alpha), PGC-1alpha, PGC-1v, PGC1, PGC1A}, KMT2A (lysine methyltransferase 2A) [NCBI Gene 4297] {aka ALL-1, ALL1, CXXC7, GAS7, HRX, HTRX}, DNER (delta/notch like EGF repeat containing) [NCBI Gene 92737] {aka UNQ26, bet}, HDAC4 (histone deacetylase 4) [NCBI Gene 9759] {aka AHO3, BDMR, HA6116, HD4, HDAC-4, HDAC-A}, METTL14 (methyltransferase 14, N6-adenosine-methyltransferase non-catalytic subunit) [NCBI Gene 57721] {aka hMETTL14}, MIR34A (microRNA 34a) [NCBI Gene 407040] {aka MIRN34A, miRNA34A, mir-34, mir-34a}, APP (amyloid beta precursor protein) [NCBI Gene 351] {aka AAA, ABETA, ABPP, AD1, APPI, CTFgamma}, VHL (von Hippel-Lindau tumor suppressor) [NCBI Gene 7428] {aka HRCA1, RCA1, VHL1, pVHL}, DNMT3L (DNA methyltransferase 3 like) [NCBI Gene 29947], EZH2 (enhancer of zeste 2 polycomb repressive complex 2 subunit) [NCBI Gene 2146] {aka ENX-1, ENX1, EZH2b, KMT6, KMT6A, WVS}, EHMT2 (euchromatic histone lysine methyltransferase 2) [NCBI Gene 10919] {aka BAT8, C6orf30, G9A, GAT8, KMT1C, NG36}, PARP1 (poly(ADP-ribose) polymerase 1) [NCBI Gene 142] {aka ADPRT, ADPRT 1, ADPRT1, ARTD1, PARP, PARP-1}, METTL3 (methyltransferase 3, N6-adenosine-methyltransferase complex catalytic subunit) [NCBI Gene 56339] {aka IME4, M6A, MT-A70, Spo8, hMETTL3}, MIR148A (microRNA 148a) [NCBI Gene 406940] {aka MIRN148, MIRN148A, hsa-mir-148, mir-148a}, IL6 (interleukin 6) [NCBI Gene 3569] {aka BSF-2, BSF2, CDF, HGF, HSF, IFN-beta-2}, MEN1 (menin 1) [NCBI Gene 4221] {aka MEAI, SCG2}, NISCH (nischarin) [NCBI Gene 11188] {aka I-1, IR1, IRAS, hIRAS}, ERBB2 (erb-b2 receptor tyrosine kinase 2) [NCBI Gene 2064] {aka CD340, HER-2, HER-2/neu, HER2, MLN 19, MLN-19}, MIR375 (microRNA 375) [NCBI Gene 494324] {aka MIRN375, hsa-mir-375, miRNA375, mir-375}, MIR193A (microRNA 193a) [NCBI Gene 406968] {aka MIRN193, MIRN193A, mir-193a}, HOTAIR (HOX transcript antisense RNA) [NCBI Gene 100124700] {aka HOXAS, HOXC-AS4, HOXC11-AS1, NCRNA00072}, KDM1A (lysine demethylase 1A) [NCBI Gene 23028] {aka AIMAH3, AOF2, BHC110, CPRF, KDM1, LSD1}, CDKN2A (cyclin dependent kinase inhibitor 2A) [NCBI Gene 1029] {aka ARF, CAI2, CDK4I, CDKN2, CMM2, INK4}, TXNIP (thioredoxin interacting protein) [NCBI Gene 10628] {aka ARRDC6, EST01027, HHCPA78, THIF, VDUP1}, AHR (aryl hydrocarbon receptor) [NCBI Gene 196] {aka FVH3, RP85, bHLHe76}, SCN5A (sodium voltage-gated channel alpha subunit 5) [NCBI Gene 6331] {aka CDCD2, CMD1E, CMPD2, HB1, HB2, HBBD}, ARID1A (AT-rich interaction domain 1A) [NCBI Gene 8289] {aka B120, BAF250, BAF250a, BM029, C1orf4, CSS2}
- **Diseases:** Crohn's (MESH:D003424), Solid tumors (MESH:D009369), personality disorders (MESH:D010554), MS (MESH:D009103), fibrosis (MESH:D005355), ulcerative colitis (MESH:D003093), sickle cell anemia (MESH:D000755), STORM (MESH:D009901), glioblastoma (MESH:D005909), Autoimmune Diseases (MESH:D001327), SLE (MESH:D008180), diffuse midline gliomas (MESH:D005910), Metabolic diseases (MESH:D008659), acute myocardial infarction (MESH:D009203), CLL (MESH:D015451), Autism spectrum disorders (MESH:D000067877), mental health disorders (OMIM:603663), chronic inflammation (MESH:D007249), seizure (MESH:D012640), myelodysplastic syndromes (MESH:D009190), blood cancers (MESH:D019337), alcoholic hepatitis (MESH:D006519), adipose tissue dysfunction (MESH:D018205), arrhythmias (MESH:D001145), cutaneous T-cell lymphoma (MESH:D016410), ischemic heart disease (MESH:D017202), ALS (MESH:D000690), ALL (MESH:D054198), synovial sarcoma (MESH:D013584), cardiac remodeling (MESH:D020257), lymphomas (MESH:D008223), Cardiometabolic diseases (MESH:D024821), PMDA (MESH:D009471), Alzheimer's (MESH:D000544), immune dysregulation (OMIM:614878), hypertrophy (MESH:D006984), hypertension (MESH:D006973), breast cancer (MESH:D001943), heart failure (MESH:D006333), Neurological Disorders (MESH:D009461), T2D (MESH:D003924), Obesity (MESH:D009765), neurological, cardiovascular, and autoimmune diseases (MESH:D020274), depression (MESH:D003866), NMPA (MESH:D000069279), NAFLD (MESH:D065626), tumorigenesis (MESH:D063646), kidney and cardiovascular diseases (MESH:D007674), CHD (MESH:D004266), prostate and bladder cancers (MESH:D011471), metastasis (MESH:D009362), AML (MESH:D015470), autism (MESH:D001321), diabetic peripheral neuropathy (MESH:D010523), epilepsy (MESH:D004827), disease (MESH:D004194), insulin resistance (MESH:D007333), hepatitis B. (MESH:D006509), keloid (MESH:D007627), Neurodevelopmental disorders (MESH:D002658)
- **Chemicals:** Temozolomide (MESH:D000077204), CPI-0610 (MESH:C000623150), mocetinostat (MESH:C523184), salt (MESH:D012492), lysine (MESH:D008239), Dacogen (MESH:D000077209), folate (MESH:D005492), Tazverik (MESH:C000593333), sodium (MESH:D012964), Beleodaq (MESH:C487081), CUDC-101 (MESH:C549566), glucose (MESH:D005947), Istodax (MESH:C087123), I-BET762 (MESH:C554645), Apabetalone (MESH:C000628794), ruxolitinib (MESH:C540383), ATP (MESH:D000255), Vafidemstat (MESH:C000710213), vinclozolin (MESH:C025643), vitamin B12 (MESH:D014805), sucrose (MESH:D013395), givinostat (MESH:C575255), pracinostat (MESH:C557525), Ivosidenib (MESH:C000627630), 2-hydroxyglutarate (MESH:C019417), Farydak (MESH:D000077767), Dordaviprone (MESH:C585684), lactate (MESH:D019344), Vidaza (MESH:D001374), Psi (MESH:D011560), CFT8634 (-), thalidomide (MESH:D013792), enasidenib (MESH:C000605269), CAS (MESH:D002118), ASOs (MESH:D016376), pomalidomide (MESH:C467566), valproic acid (MESH:D014635), BPA (MESH:C006780), lipid (MESH:D008055), OTX015 (MESH:C000605331), quinolone (MESH:D015363), N6-methyladenosine (MESH:C010223), m6A (MESH:C005955), Tucidinostat (MESH:C547816), Zolinza (MESH:D000077337), alcohol (MESH:D000438)
- **Species:** Severe acute respiratory syndrome coronavirus 2 (no rank) [taxon 2697049], Human immunodeficiency virus 1 (no rank) [taxon 11676], Homo sapiens (human, species) [taxon 9606]
- **Mutations:** A-to-I, K219T, K27M
- **Cell lines:** MRG-201 — Mus musculus (Mouse), Hybridoma (CVCL_LN00), INT-1B3 — Homo sapiens (Human), Melanoma, Cancer cell line (CVCL_C682)

## Full text

_Full body text omitted from this summary view._ Fetch the complete paper as Markdown: https://tomesphere.com/paper/PMC12993786/full.md

## Figures

11 figures with captions in the complete paper: https://tomesphere.com/paper/PMC12993786/full.md

## References

634 references — full list in the complete paper: https://tomesphere.com/paper/PMC12993786/full.md

---
Source: https://tomesphere.com/paper/PMC12993786