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MitoSENS 2017

mitosens mitochondria sens gene therapy

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#1 Oki

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  • Location:SENS Research Foundation

Posted 10 March 2017 - 02:45 AM


In late 2016 we published our long-awaited manuscript describing the simultaneous rescue of two mitochondrial genes simultaneously. The breakthrough that started this project off was funded by a successful Longecity crowdfunding campaign in 2013. Less than 3 years later we published this manuscript describing the results:

 

https://academic.oup...1093/nar/gkw756

 

This manuscript broke ground in two fundamental ways:

 

1. We showed the first incontrovertible true complete rescue of a mitochondrial gene. Meaning we took cells that by all measures seemed incapable of performing any aerobic respiration and then added back the mutant gene to rescue the phenotype. 

2. We expressed two engineered mitochondrial genes simultaneously. This has big implications for future applications to aging disease as well as to complex mitochondrial disease because maybe if we can produce two genes at once then someday perhaps we can express all 13. 

 

 

What we are working on now:

 

Allotopic expression in a mouse. We haven't actually started working on live mice yet, but we have gotten AE to work in mouse cells. 

 

All the genes! Well maybe not all 13 simultaneously, but quite a few. We are trying to develop a platform technology that will allow us to do the same thing with all 13 genes separately, in groups, or all at once. Right now we mostly work on 1 at a time. 

 

 


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Also tagged with one or more of these keywords: mitosens, mitochondria, sens, gene therapy

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