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Designing nucleic acid-based therapeutics for cancer treatment: Updates on the state of the art.

Molecular therapy : the journal of the American Society of Gene Therapy 2026 Vol.34(1) p. 68-96

Peng B, Jayasinghe MK, Le AH, Tran NM, Le MTN

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As in art, the ability of nucleic acids to be designed and synthesized as a novel treatment modality is limited only by the imagination.

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BibTeX ↓ RIS ↓
APA Peng B, Jayasinghe MK, et al. (2026). Designing nucleic acid-based therapeutics for cancer treatment: Updates on the state of the art.. Molecular therapy : the journal of the American Society of Gene Therapy, 34(1), 68-96. https://doi.org/10.1016/j.ymthe.2025.09.041
MLA Peng B, et al.. "Designing nucleic acid-based therapeutics for cancer treatment: Updates on the state of the art.." Molecular therapy : the journal of the American Society of Gene Therapy, vol. 34, no. 1, 2026, pp. 68-96.
PMID 41017155

Abstract

As in art, the ability of nucleic acids to be designed and synthesized as a novel treatment modality is limited only by the imagination. Nucleic acids of virtually all sizes and forms can be synthesized on demand, from short antisense oligonucleotides to large mRNAs and to entire chromosomes. Given the genetic basis of cancer, nucleic acid-based therapy is a particularly promising avenue for anticancer therapeutic development. This has led to a profusion of studies exploring strategies to utilize nucleic acid-based drugs to treat cancer, with some approaches demonstrating great potential for clinical translation. In this review, we summarize the various nucleic acid-based strategies being developed for cancer therapy. We also provide a comprehensive overview of current efforts to enhance the potency and safety of nucleic acid-based drugs, exploring advances in nucleotide composition, design, and delivery strategies.

MeSH Terms

Humans; Neoplasms; Nucleic Acids; Genetic Therapy; Animals; Oligonucleotides, Antisense; Drug Delivery Systems; Antineoplastic Agents

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