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Despite this, only a small subset of these systems has yielded approved therapeutics
The Bottom Line As I navigate this space with my patients, I hold two truths simultaneously: the preclinical evidence for certain peptides is genuinely compelling, AND the lack of rigorous human safety data demands significant caution
PMID 7936021
ALS-associated mutant TDP-43 is known to be aberrantly localized in mitochondria, but suppressing its mitochondrial localization protects against neurotoxicity in TDP-43 A315T mice (Wang et al., 2016)
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