Steve Wilton says " that tissue biopsies suggest dystrophin is being produced throughout the bodies of boys who received high doses of the bandage."

 

Through out the body at high dosage (:-o), does this mean the heart and diaphragm also?. Fingers crossed.

 

http://www.newscientist.com/article/mg20627544.700-gene-bandage-rej...

 

 

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Diaphragm should, that's what they saw in animals...heart not too sure BUT this is a huge step it it works. Fingers crossed!

"Last week, Wilton told the World Congress of Internal Medicine in Melbourne, Australia, that tissue biopsies suggest dystrophin is being produced throughout the bodies of boys who received high doses of the bandage.

It is not yet clear if the dystrophin will increase the boys' muscle strength, but Wilton points out it did in animals. The protein resembles the version found in men with the milder Becker's MD, who live into their 60s."
So they have started clincial trials with this? This is different than exon skipping?
It's exon skipping. AVI Biopharma UK systemic trial results. PMO skipping 51.

Kristi Koop said:
So they have started clincial trials with this? This is different than exon skipping?
This looks encouraging, Ben's mutation is on exon 59, will they be able to alter the treatment to work on other exons
From my understanding, PMO does produce dystrophin, however it is patchy and the amounts required for benifit are huge. The reason is that PMO is taken up by passive diffusion. I have also read that there was minimal uptake into the heart.

PPMO's on the other hand are much more promising as they have a peptide which either tagets muscles or is used to help permeate them (not sure which) and it also gets into the heart.
We do not know how high a PMO dose is needed at this point. A dose as high as 200 mg/kg was needed to skip 3 exons in dogs in order to show improvement in function. That might equate to a lower than 60 mg/kg for one exon in humans, there is a lot of discussion around this subject. The UK trial results should bring more light.

PPMOs were more effecient in animals, however they had some toxicity issues at some doses. AVI did not decide if they will continue development.

Jonathan said:
From my understanding, PMO does produce dystrophin, however it is patchy and the amounts required for benifit are huge. The reason is that PMO is taken up by passive diffusion. I have also read that there was minimal uptake into the heart.

PPMO's on the other hand are much more promising as they have a peptide which either tagets muscles or is used to help permeate them (not sure which) and it also gets into the heart.
No dosing hasnt been worked out in either PMO's or PPMO's, however I would guess there will be a better idea after the current round of trials.

Even though there is a level of toxicity with the PPMO, the hope is that with an spaced out dosing regime, the toxicity will be abel to be managed. Just have to hope AVI continue their trials and look at ways to get around the toxicity problem.


Ofelia Marin said:
We do not know how high a PMO dose is needed at this point. A dose as high as 200 mg/kg was needed to skip 3 exons in dogs in order to show improvement in function. That might equate to a lower than 60 mg/kg for one exon in humans, there is a lot of discussion around this subject. The UK trial results should bring more light.

PPMOs were more effecient in animals, however they had some toxicity issues at some doses. AVI did not decide if they will continue development.

Jonathan said:
From my understanding, PMO does produce dystrophin, however it is patchy and the amounts required for benifit are huge. The reason is that PMO is taken up by passive diffusion. I have also read that there was minimal uptake into the heart.

PPMO's on the other hand are much more promising as they have a peptide which either tagets muscles or is used to help permeate them (not sure which) and it also gets into the heart.
I am assuming that if AVI decides not to continue development of PPMOs someone else will. There are other research groups working with different peptides attached AOs. Not sure of the timing though, personally I would rather have PMOs soon (2-3-4-5 years) if they make them work as effective as they did in animals and buy more time to wait for the PPMOs. Clock is ticking. :(

Jonathan said:
No dosing hasnt been worked out in either PMO's or PPMO's, however I would guess there will be a better idea after the current round of trials.

Even though there is a level of toxicity with the PPMO, the hope is that with an spaced out dosing regime, the toxicity will be abel to be managed. Just have to hope AVI continue their trials and look at ways to get around the toxicity problem.


Ofelia Marin said:
We do not know how high a PMO dose is needed at this point. A dose as high as 200 mg/kg was needed to skip 3 exons in dogs in order to show improvement in function. That might equate to a lower than 60 mg/kg for one exon in humans, there is a lot of discussion around this subject. The UK trial results should bring more light.

PPMOs were more effecient in animals, however they had some toxicity issues at some doses. AVI did not decide if they will continue development.

Jonathan said:
From my understanding, PMO does produce dystrophin, however it is patchy and the amounts required for benifit are huge. The reason is that PMO is taken up by passive diffusion. I have also read that there was minimal uptake into the heart.

PPMO's on the other hand are much more promising as they have a peptide which either tagets muscles or is used to help permeate them (not sure which) and it also gets into the heart.

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