Molecular biology reportsJul 3, 2026

RNA changes as new drug targets: developing precise treatments and delivery methods for cancer immunotherapy and metabolic diseases

mRNA Technology Weekly Brief ↗PubMed ↗DOI ↗

Updated Jul 4, 2026

Abstract

Epitranscriptomic RNA modifications, including N6-methyladenosine (m6A), are linked to cancer progression and metabolic disorders.

RNA modifications play a significant role in regulating gene expression beyond traditional genetic mechanisms.
Dysregulation of m6A, 5-methylcytosine (m5C), and pseudouridine is associated with cancer, immune evasion, and resistance to therapies.
Recent developments in targeting RNA-modifying enzymes may offer new therapeutic approaches for cancer and metabolic diseases.
Advancements in delivery systems, such as lipid nanoparticles and polymeric carriers, enhance the stability and specificity of RNA-targeted therapies.
Challenges remain in clinical translation, particularly regarding delivery efficiency and patient variability.

Simplified

RNA modifications have emerged as an important regulatory layer that influences gene expression beyond conventional genetic and epigenetic mechanisms. Among the various epitranscriptomic modifications, N6-methyladenosine (m6A), 5-methylcytosine (m5C), and pseudouridine (Ψ) have been extensively investigated for their roles in RNA stability, splicing, translation, immune regulation, and metabolic homeostasis. Increasing evidence suggests that dysregulation of these modifications contributes to cancer progression, immune evasion, therapeutic resistance, and metabolic disorders, suggesting their potential as therapeutic targets. This review summarizes recent advances in endogenous epitranscriptomic RNA modifications and discusses their relevance in cancer immunotherapy and metabolic diseases. In addition, emerging therapeutic approaches targeting RNA-modifying enzymes, including writers, erasers, and readers, are discussed along with the development of antisense oligonucleotides, RNA-based therapeutics, and delivery systems. Recent progress in lipid nanoparticles, polymeric carriers, and targeted delivery platforms has improved the stability, specificity, and translational potential of RNA-targeted therapies. The review also highlights current challenges associated with clinical translation, including delivery efficiency, therapeutic specificity, and patient heterogeneity. Overall, epitranscriptomic RNA modifications may provide new opportunities for the development of precision therapeutic strategies for cancer and metabolic diseases.

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Competing interests

Declarations. Consent for publication: Not applicable. Competing interests: The authors declare no competing interests. Regarding the use of generative AI and AI-assisted technologies in writing During the preparation of this manuscript, the authors used Google Gemini, OpenAI ChatGPT, and Grok AI tools to improve language clarity, grammar, and overall readability. After using these tools, the authors carefully reviewed, revised, and edited the content as necessary and take full responsibility for the accuracy and integrity of the final manuscript.

PubMed

RNA changes as new drug targets: developing precise treatments and delivery methods for cancer immunotherapy and metabolic diseases

Abstract

Full Text

Funding

Competing interests

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