Splicingin

Splicingin is a term sometimes used in genetics and molecular biology to describe deliberate modification of RNA splicing by introducing new splice elements or exons into a gene or transcript. In practice, splicingin refers to strategies intended to alter which regions of a transcript are joined together during RNA processing, thereby changing the mature mRNA and the protein it encodes. The term is not widely standardized and appears mainly in discussions of experimental approaches to control splicing.

Mechanisms and methods: Splicingin can be achieved at the DNA level by genome editing to insert exons, splice sites, or regulatory elements, typically using CRISPR-based techniques with donor templates for precise insertion. At the RNA level, splice-switching strategies employ antisense oligonucleotides or small molecules to enhance or suppress the use of specific splice sites, enabling inclusion or skipping of exons without altering the genome. Some concepts also include spliceosome-mediated trans-splicing, where exons from different transcripts are joined to create a chimeric mRNA.

Applications: Splicingin is used in basic research to study gene function by comparing splice variants and in therapeutic contexts to correct mis-splicing or to elicit beneficial protein isoforms. Antisense therapies that modulate splicing have been explored for inherited disorders; exon inclusion or skipping strategies aim to restore or modify protein function.

Challenges: Efficiency, tissue specificity, off-target effects, immunogenicity, and delivery are major hurdles. Ethical and regulatory considerations apply particularly to clinical uses. The long-term effects of altering splicing patterns are not fully understood.

See also: Splicing, spliceosome, exon skipping, splice-switching oligonucleotides, antisense therapy, trans-splicing.