Not so pseudo anymore: pseudogenes as therapeutic targets

Area of Science:

• Genetics
• Molecular Biology
• Pharmacology

Background:

• Pseudogenes are gene remnants often considered non-functional DNA.
• They arise from gene duplication or retrotransposition events.
• Historically viewed as molecular fossils, their role is being re-evaluated.

Purpose of the Study:

• To discuss the emerging role of pseudogenes in gene expression regulation.
• To explore pseudogene-derived RNAs as regulators in human diseases.
• To highlight novel pharmacological strategies targeting pseudogene transcripts.

Main Methods:

• Review of existing literature on pseudogene function.
• Analysis of pseudogene-derived noncoding RNAs in gene regulatory circuits.
• Discussion of therapeutic approaches targeting pseudogene transcripts.

Main Results:

• Pseudogenes can be exapted for novel functions, particularly in gene regulation.
• Pseudogene-derived RNAs act as crucial components in RNA-based gene regulatory networks.
• Functional pseudogenes can be considered as active genes.

Conclusions:

• Pseudogene-derived RNAs play significant roles in human disease pathogenesis.
• Targeting pseudogene transcripts with antisense oligonucleotides, siRNAs, aptamers, or gene therapy presents promising pharmacological avenues.
• Reclassifying functional pseudogenes as 'genes' reflects their active regulatory roles.