Switching a transgene with a Morpholino oligo

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In summary, the conversation discusses a study published in the journal Sci Transl Med, which introduces a novel ribozyme on-switch for modulating gene expression in AAV-delivered therapies. The press release also mentions potential applications for this technology. The primary paper, published in Nat Biotechnol, provides more details on the reversible RNA on-switch.
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JonMoulton
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TL;DR Summary
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.
 

FAQ: Switching a transgene with a Morpholino oligo

What is a transgene?

A transgene is a gene that has been artificially introduced into an organism, typically through genetic engineering techniques. It is different from the organism's natural genes and can have a different function or expression pattern.

What is a Morpholino oligo?

A Morpholino oligo is a type of synthetic molecule that is designed to bind to specific RNA sequences, effectively blocking their function. It is commonly used in genetic studies to manipulate gene expression.

How does switching a transgene with a Morpholino oligo work?

Switching a transgene with a Morpholino oligo involves injecting the oligo into an organism that carries the transgene. The oligo will then bind to the transgene's RNA, preventing it from being translated into protein. This can effectively turn off the transgene's expression.

What are the benefits of using a Morpholino oligo to switch a transgene?

Using a Morpholino oligo to switch a transgene offers several advantages. It is a quick and efficient method, as the oligo can be easily synthesized and injected into the organism. It also allows for precise control over the timing and level of gene expression, as the oligo can be removed or added at specific stages of development.

Are there any potential drawbacks to switching a transgene with a Morpholino oligo?

While using a Morpholino oligo to switch a transgene can be a useful tool, there are some potential drawbacks to consider. The oligo may not completely block the transgene's expression, leading to incomplete or variable results. Additionally, there is a risk of off-target effects, where the oligo may bind to unintended RNA sequences and interfere with their function.

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