- This topic has 5 replies, 4 voices, and was last updated 3 years, 5 months ago by
Frankie Perazzola.
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January 25, 2019 at 8:12 am #11401
FA Forums Moderator
KeymasterIncreased levels of a protein called GRP75 appear to repair frataxin deficiency and mitochondrial changes in Friedreich’s ataxia (FA), a cell study reports. Click here to read more about it.
What are your thoughts about this news?
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January 25, 2019 at 9:32 am #11418
Matt Lafleur
MemberI don’t mean to be a bummer. I should probably be super excited that any progress is being made…but part of me thinks, “This isn’t my first rodeo.” Seems like incremental steps have been all there is to see for twenty years. I’m ready for a breakthrough. Sorry, I need to be more optimistic, but those are my thoughts.
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January 25, 2019 at 4:41 pm #11431
Shandra
ParticipantI thought this was interesting, because it takes a different route to rescuing Frataxin. I’ll explain.
Our cells make proteins like this: DNA -> RNA -> Protein
So normally Frataxin is made like this:
Frataxin Gene (DNA) -> Frataxin mRNA -> Frataxin Protein
Because of GAA repeats in the DNA, FA patients have trouble with the first step ( DNA -> RNA ), which is known as transcription. Since we don’t make sufficient Frataxin mRNA, we don’t have enough Frataxin protein. But, we do have some.
The cool thing about this study is it takes a post-transcriptional approach. In other words, instead of trying to get around the issues caused by the GAA repeats in the DNA, the protein in the study (GRP75) works with the small amount of Frataxin we already have, and basically helps keep it around longer and get to the mitochondria where it can then do its job.
It’s definitely preliminary work, but I think it’s pretty cool. Instead of trying to make our bodies produce more Frataxin, or simply mopping up the damage caused by not having much Frataxin, this research shows we might be able to just help the Frataxin we already have!
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January 25, 2019 at 4:54 pm #11432
Shandra
ParticipantHere is the full paper, if anyone is interested in reading: https://drive.google.com/file/d/1qaEvn_0GQAbyvI0nVcaXGpaD7beigj8R/view?usp=sharing
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January 25, 2019 at 4:58 pm #11435
Matt Lafleur
MemberYou are like our secret weapon and I am honored to have you here.
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January 26, 2019 at 4:13 pm #11442
Frankie Perazzola
KeymasterI agree with Matt, we’re so fortunate!
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