Tier 3 — preclinical

Effective treatment of mitochondrial myopathy by nicotinamide riboside, a vitamin B3

Khan NA, Auranen M, Paetau I, Pirinen E, Euro L, Forsström S, Pasila L, Velagapudi V, Carroll CJ, Auwerx J, Suomalainen A
EMBO molecular medicine 2014 6; 6; 721-731

Bibliography

PubMed
PMID 24711540
PubMed Central
PMC4203351
Funding
European and Finnish public, university and charitable research support, including ERC, Academy of Finland, NIH and Swiss research programs.
Competing interests
A complete competing-interest declaration is not specified in the accessible publication information.

Study snapshot

DesignPreclinical animal study with supporting assays as applicable
ModelMice with mitochondrial myopathy
SampleMitochondrial-myopathy mouse experiments
InterventionOral NR
DurationEarly- and late-stage disease treatment protocols
EndpointsModel-specific physiological outcomes; Biochemical or functional measures reported in the study

What the study showed, in plain terms

NR improved mitochondrial biogenesis and selected disease features in a mouse model of mitochondrial myopathy.

Key findings

NR improved mitochondrial biogenesis and selected disease features in a mouse model of mitochondrial myopathy.

What this study can and cannot tell us

A disease-model experiment does not establish efficacy across genetically diverse human mitochondrial disorders.

Editorial review

Reviewed by the Biohack Blueprint research team

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