Switching a transgene with a Morpholino oligo

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SUMMARY

The discussion centers on the innovative use of a Morpholino oligo to switch gene expression in AAV-delivered therapeutics, as detailed in the primary paper by Zhong et al. (2019). This study introduces a reversible RNA on-switch that effectively modulates gene expression in vivo, showcasing significant potential for therapeutic applications. The editorial by Giuseppe Ronzitti (2020) further emphasizes the implications of this technology in advancing gene therapy methodologies. The findings underscore a pivotal advancement in the field of molecular biology and gene therapy.

PREREQUISITES
  • Understanding of AAV (Adeno-Associated Virus) vector systems
  • Familiarity with Morpholino oligonucleotides and their applications
  • Knowledge of ribozyme technology and its mechanisms
  • Basic principles of gene expression modulation in vivo
NEXT STEPS
  • Research the mechanisms of AAV-delivered gene therapies
  • Explore the applications of Morpholino oligos in gene regulation
  • Investigate the design and function of ribozyme on-switches
  • Study recent advancements in RNA-based therapeutics
USEFUL FOR

Researchers in molecular biology, genetic engineers, and professionals involved in the development of gene therapies will benefit from this discussion, particularly those focused on innovative gene modulation techniques.

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TL;DR
New approach for controlling expression from a transgene
Giuseppe Ronzitti. Let’s switch on AAV! Sci Transl Med. 2020;12(579) doi:10.1126/scitranslmed.aba9016 (editorial)
https://stm.sciencemag.org/content/12/529/eaba9016

This describes a virally delivered ribozyme modulated by a Morpholino oligo.

Press release
https://www.eurekalert.org/pub_releases/2019-12/sri-nta122419.php

Primary paper
Zhong G, Wang H, He W, Li Y, Mou H, Tickner ZJ, Tran MH, Ou T, Yin Y, Diao H, Farzan M. A reversible RNA on-switch that controls gene expression of AAV-delivered therapeutics in vivo. Nat Biotechnol. 2019;[Epub ahead print] doi:10.1038/s41587-019-0357-y.
https://www.nature.com/articles/s41587-019-0357-y
 
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This is an interesting study! It looks like the authors have developed a novel ribozyme on-switch to modulate gene expression of AAV-delivered therapies in vivo. The press release highlights some of the potential applications for this technology. I'm looking forward to seeing more studies in this area and how this technology could be utilized to develop new treatments.