RNA interference (RNAi) and antisense oligonucleotides (ASOs) are two approaches that are commonly used in small animal research to modulate gene expression. Both RNAi and ASOs can be used to selectively silence or inhibit the expression of specific genes, allowing researchers to investigate the roles of these genes in disease and test potential therapies. Here’s an overview of RNAi and ASOs in small animal research:

1. RNA interference: RNAi is a naturally occurring mechanism for post-transcriptional gene silencing. It involves the use of small double-stranded RNA molecules, such as small interfering RNAs (siRNAs) or short hairpin RNAs (shRNAs), to selectively degrade or inhibit the expression of specific messenger RNAs (mRNAs). RNAi can be used to selectively silence disease-causing genes in small animal models, allowing researchers to investigate the roles of these genes in disease and test potential therapies.

One advantage of RNAi is its specificity – siRNAs or shRNAs can be designed to target specific mRNAs, allowing for selective gene silencing. In small animal models, RNAi can be delivered using viral vectors, lipid-based transfection reagents, or electroporation. RNAi has been used to investigate the roles of specific genes in small animal models of cancer, viral infections, neurodegenerative diseases, and metabolic disorders.

2. Antisense oligonucleotides: ASOs are synthetic single-stranded DNA or RNA molecules that can bind to specific mRNAs, blocking their translation or promoting their degradation. ASOs can be designed to target specific disease-causing genes, allowing for selective gene inhibition. ASOs can be delivered using viral vectors, lipid-based transfection reagents, or direct injection into target tissues or organs.

One advantage of ASOs is their versatility – they can be designed to target a wide range of mRNAs, allowing for selective gene inhibition in various disease contexts. In small animal models, ASOs have been used to investigate the roles of specific genes in diseases such as cancer, neurodegenerative disorders, and genetic diseases. ASOs have also been used as a therapeutic approach in small animal models, with promising results in diseases such as muscular dystrophy, spinal muscular atrophy, and Huntington’s disease.

Overall, RNAi and ASOs are powerful tools for investigating gene function and developing new therapies in small animal models. By selectively silencing or inhibiting specific disease-causing genes, researchers can gain valuable insights into disease mechanisms and identify potential therapeutic targets. By optimizing transfection conditions and carefully considering safety and ethical considerations, RNAi and ASOs can be used to generate reliable and reproducible data that can translate into clinical applications.