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ALS Research Breakthrough?
“Researchers identify a cellular defect common to familial and sporadic forms of ALS”
A recent NIH-funded study may point to a possible therapeutic target for the disease.
Read the announcement here:
What do you think of this breakthrough? Does it hold promise for ALS patients? Especially when it identifies a cell defect that is shown to be common in both sporadic and familial ALS.
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2 Replies
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This is where it gets interesting;
” If an antisense oligonucleotide drug, which stops cells from manufacturing specific proteins, was used to decrease the amount of CHMP7 within the ALS neurons, the pore never degraded. “
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If past history of so-called ALS treatment breakthroughs are any indication of any possible future ALS treatments, then this too will fizzy out into obscurity.
Richard
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Dr. Rothstein works in ALS research since a long time (~20 years?). But what is presented here is not very impressive. Working with iPSC-derived is something that could be done at little cost now, and (fortunately) many teams are working on ways to mitigate TDP-43 aggregates.
And it only pushes the goal post further away: What makes CHMP7 aggregate in the nucleus?I guess this work has two objectives:
1. Participating in the run to patent TDP-43 therapies.
2. Showing to funding parties that the lab is doing something with their money.
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