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Updated: Feb 7, 2026

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Developments in lncRNA drug discovery: where are we heading?
Ilya Blokhin1, Olga Khorkova2, Jane Hsiao2
1a Center for Therapeutic Innovation and Department of Psychiatry and Behavioral Sciences , University of Miami Miller School of Medicine , Miami , FL , USA.
Long noncoding RNAs (lncRNAs) challenge the central dogma and offer new therapeutic targets for diseases. Advances in lncRNA targeting modalities are paving the way for novel treatments.
Area of Science:
- Molecular Biology
- Genetics
- Therapeutics
Background:
- Long noncoding RNAs (lncRNAs) have emerged as key regulators, challenging the traditional central dogma of molecular biology.
- lncRNAs play complex roles in cellular processes and are implicated in the pathogenesis of numerous diseases.
Purpose of the Study:
- To review current advances in lncRNA diversity and biological functions.
- To highlight the therapeutic potential of lncRNAs in various disease areas.
Main Methods:
- Literature review of lncRNA research.
- Analysis of therapeutic strategies targeting lncRNAs, including oligonucleotide-based drugs and CRISPR-Cas9.
Main Results:
- lncRNAs are involved in diverse biological functions and disease mechanisms.
- lncRNA-targeting modalities show promise for treating neurological, psychiatric, cardiovascular diseases, infectious diseases, and cancer.
Conclusions:
- lncRNA-dependent pathogenesis is a promising area for therapeutic intervention.
- Further research is needed to fully understand lncRNA biology, improve lncRNA-targeting drugs, and standardize nomenclature and databases.
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