Targeting and engineering long non-coding RNAs for cancer therapy.
Michela CoanSimon HaefligerSamir OunzainRory JohnsonPublished in: Nature reviews. Genetics (2024)
RNA therapeutics (RNATx) aim to treat diseases, including cancer, by targeting or employing RNA molecules for therapeutic purposes. Amongst the most promising targets are long non-coding RNAs (lncRNAs), which regulate oncogenic molecular networks in a cell type-restricted manner. lncRNAs are distinct from protein-coding genes in important ways that increase their therapeutic potential yet also present hurdles to conventional clinical development. Advances in genome editing, oligonucleotide chemistry, multi-omics and RNA engineering are paving the way for efficient and cost-effective lncRNA-focused drug discovery pipelines. In this Review, we present the emerging field of lncRNA therapeutics for oncology, with emphasis on the unique strengths and challenges of lncRNAs within the broader RNATx framework. We outline the necessary steps for lncRNA therapeutics to deliver effective, durable, tolerable and personalized treatments for cancer.
Keyphrases
- long non coding rna
- drug discovery
- poor prognosis
- cancer therapy
- genome editing
- crispr cas
- papillary thyroid
- small molecule
- genome wide identification
- squamous cell
- genome wide analysis
- drug delivery
- transcription factor
- nucleic acid
- palliative care
- genome wide
- squamous cell carcinoma
- protein protein
- dna methylation
- long noncoding rna
- single molecule
- bioinformatics analysis