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Study · Animal study (in vivo) · mouse · 2021

11 results

MOTS-c promotes phosphorodiamidate morpholino oligomer uptake and efficacy in dystrophic mice.

Ran N, Lin C, Leng L, Han G, Geng M, Wu Y, Bittner S, Moulton HM, Yin H

EMBO molecular medicine18 Dec 2020Cited by 12 (Europe PMC)

PMID33337582 (opens in a new tab)PMCIDPMC7863382 (opens in a new tab)DOI10.15252/emmm.202012993 (opens in a new tab)PepBankPUB-33337582
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Summary

Antisense oligonucleotide (AO)-mediated exon-skipping therapies show promise in Duchenne muscular dystrophy (DMD), a devastating muscular disease caused by frame-disrupting mutations in the DMD gene. However, insufficient systemic delivery remains a hurdle to clinical deployment. Here, we demonstrate that MOTS-c, a mitochondria-derived bioactive peptide, with an intrinsic muscle-targeting property, augmented glycolytic flux and energy production capacity of dystrophic muscles in vitro and in vivo, resulting in enhanced phosphorodiamidate morpholino oligomer (PMO) uptake and activity in mdx mice. Long-term repeated administration of MOTS-c (500 μg) and PMO at the dose of 12.5 mg/kg/week for 3 weeks followed by 12.5 mg/kg/month for 3 months (PMO-M) induced therapeutic levels of dystrophin expression in peripheral muscles, with up to 25-fold increase in diaphragm of mdx mice over PMO alone. PMO-M improved muscle function and pathologies in mdx mice without detectable toxicity. Our results demonstrate that MOTS-c enables enhanced PMO uptake and activity in dystrophic muscles by providing energy and may have therapeutic implications for exon-skipping therapeutics in DMD and other energy-deficient disorders.
CC BYText from Europe PMC. Highlighted sentences are the source of results.

Results from this study11

7 results from studies that gave the peptide. No result from this study comes from people.

  1. 01AnimalAnimal study (in vivo)Efficacy outcome

    In mdx mice, MOTS‑c augmented glycolytic flux of dystrophic muscle.

    In plain wordsIn mice with muscular dystrophy, MOTS‑c increased the sugar-burning activity of muscle.

    Peptide
    MOTS-c
    Outcome
    IncreaseGlycolytic flux of dystrophic muscle in vivo
    Study

    mousen: not reported in source

    mdx mice (Duchenne muscular dystrophy)
    Outcome group
    Mitochondrial function · Musculoskeletal
    Studied dose
    Dose not stated

    Source sentences

    Summary
    MOTS-c, a mitochondria-derived bioactive peptide, with an intrinsic muscle-targeting property, augmented glycolytic flux and energy production capacity of dystrophic muscles in vitro and in vivo
  2. 02AnimalAnimal study (in vivo)Efficacy outcome

    In mdx mice, MOTS‑c augmented the energy production capacity of dystrophic muscle.

    In plain wordsIn mice with muscular dystrophy, MOTS‑c increased the energy production of muscle.

    Peptide
    MOTS-c
    Outcome
    IncreaseEnergy production capacity of dystrophic muscle in vivo
    Study

    mousen: not reported in source

    mdx mice (Duchenne muscular dystrophy)
    Outcome group
    Mitochondrial function · Musculoskeletal
    Studied dose
    Dose not stated

    Source sentences

    Summary
    MOTS-c, a mitochondria-derived bioactive peptide, with an intrinsic muscle-targeting property, augmented glycolytic flux and energy production capacity of dystrophic muscles in vitro and in vivo
  3. 03AnimalAnimal study (in vivo)Efficacy outcome

    In mdx mice, MOTS‑c enhanced phosphorodiamidate morpholino oligomer (PMO) uptake.

    In plain wordsIn mice with muscular dystrophy, MOTS‑c helped muscle take up more of the exon-skipping drug PMO.

    Peptide
    MOTS-c
    Outcome
    IncreasePMO uptake in mdx mice
    Study

    mousen: not reported in source

    mdx mice (Duchenne muscular dystrophy)
    Outcome group
    Muscle injury and repair · Musculoskeletal
    Studied dose
    Dose not stated

    Source sentences

    Summary
    resulting in enhanced phosphorodiamidate morpholino oligomer (PMO) uptake and activity in mdx mice.
  4. 04AnimalAnimal study (in vivo)Efficacy outcome

    In mdx mice, MOTS‑c enhanced phosphorodiamidate morpholino oligomer (PMO) activity.

    In plain wordsIn mice with muscular dystrophy, MOTS‑c made the exon-skipping drug PMO work better.

    Peptide
    MOTS-c
    Outcome
    IncreasePMO activity in mdx mice
    Study

    mousen: not reported in source

    mdx mice (Duchenne muscular dystrophy)
    Outcome group
    Muscle injury and repair · Musculoskeletal
    Studied dose
    Dose not stated

    Source sentences

    Summary
    resulting in enhanced phosphorodiamidate morpholino oligomer (PMO) uptake and activity in mdx mice.
  5. 05AnimalAnimal study (in vivo)Efficacy outcome

    In mdx mice, repeated MOTS‑c with PMO increased dystrophin expression in the diaphragm by up to 25-fold over PMO alone.

    In plain wordsIn mice with muscular dystrophy, MOTS‑c plus PMO raised dystrophin in the diaphragm up to 25-fold more than PMO alone.

    Peptide
    MOTS-c
    Outcome
    IncreaseDystrophin expression in the diaphragm
    Study

    mousen: not reported in source

    mdx mice (Duchenne muscular dystrophy)
    Comparator
    PMO alone
    Outcome group
    Muscle injury and repair · Musculoskeletal
    Studied dose
    500 μgAs given in this study (mouse).

    Measurements

    Dystrophin increase over PMO alone
    ≤ 25-fold
    diaphragm

    Source sentences

    Summary
    Long-term repeated administration of MOTS-c (500 μg) and PMO at the dose of 12.5 mg/kg/week for 3 weeks followed by 12.5 mg/kg/month for 3 months (PMO-M) induced therapeutic levels of dystrophin expression in peripheral muscles, with up to 25-fold increase in diaphragm of mdx mice over PMO alone.
  6. 06LabIn vitro (cells)Efficacy outcome

    In vitro, MOTS‑c augmented glycolytic flux of dystrophic muscle.

    In plain wordsIn lab tests, MOTS‑c increased the sugar-burning activity of muscle from a muscular dystrophy model.

    Peptide
    MOTS-c
    Outcome
    IncreaseGlycolytic flux of dystrophic muscle in vitro
    Study

    n: not reported in source

    Dystrophic muscle (in vitro)
    Outcome group
    Mitochondrial function · Musculoskeletal
    Studied dose
    Dose not stated

    Source sentences

    Summary
    MOTS-c, a mitochondria-derived bioactive peptide, with an intrinsic muscle-targeting property, augmented glycolytic flux and energy production capacity of dystrophic muscles in vitro and in vivo
  7. 07LabIn vitro (cells)Efficacy outcome

    In vitro, MOTS‑c augmented the energy production capacity of dystrophic muscle.

    In plain wordsIn lab tests, MOTS‑c increased the energy production of muscle from a muscular dystrophy model.

    Peptide
    MOTS-c
    Outcome
    IncreaseEnergy production capacity of dystrophic muscle in vitro
    Study

    n: not reported in source

    Dystrophic muscle (in vitro)
    Outcome group
    Mitochondrial function · Musculoskeletal
    Studied dose
    Dose not stated

    Source sentences

    Summary
    MOTS-c, a mitochondria-derived bioactive peptide, with an intrinsic muscle-targeting property, augmented glycolytic flux and energy production capacity of dystrophic muscles in vitro and in vivo

Studies that did not test the peptide alone4

In this study, MOTS-c was a lab tool or a carrier, part of a mix, or the subject of a lab method. These results are not evidence about taking MOTS-c.

  1. 08AnimalNot a test of MOTS-c aloneAnimal study (in vivo)Efficacy outcome

    In mdx mice, repeated MOTS‑c with PMO induced therapeutic levels of dystrophin expression in peripheral muscles.

    In plain wordsIn mice with muscular dystrophy, repeated MOTS‑c plus PMO led to therapeutic levels of dystrophin in muscles.

    Peptide
    MOTS-c
    Outcome
    IncreaseDystrophin expression in peripheral muscles
    Study

    mousen: not reported in source

    mdx mice (Duchenne muscular dystrophy)
    Outcome group
    Muscle injury and repair · Musculoskeletal

    Source sentences

    Summary
    Long-term repeated administration of MOTS-c (500 μg) and PMO at the dose of 12.5 mg/kg/week for 3 weeks followed by 12.5 mg/kg/month for 3 months (PMO-M) induced therapeutic levels of dystrophin expression in peripheral muscles, with up to 25-fold increase in diaphragm of mdx mice over PMO alone.

    In this study, MOTS-c was a lab tool or a carrier, part of a mix, or the subject of a lab method. This result is not evidence about taking MOTS-c.

  2. 09AnimalNot a test of MOTS-c aloneAnimal study (in vivo)Efficacy outcome

    In mdx mice, the PMO-M regimen with MOTS‑c improved muscle function.

    In plain wordsIn mice with muscular dystrophy, the long-term MOTS‑c plus PMO treatment improved muscle function.

    Peptide
    MOTS-c
    Outcome
    IncreaseMuscle function with PMO-M
    Study

    mousen: not reported in source

    mdx mice (Duchenne muscular dystrophy)
    Outcome group
    Muscle injury and repair · Musculoskeletal

    Source sentences

    Summary
    PMO-M improved muscle function and pathologies in mdx mice
    1 context sentence
    Summary · context
    Long-term repeated administration of MOTS-c (500 μg) and PMO at the dose of 12.5 mg/kg/week for 3 weeks followed by 12.5 mg/kg/month for 3 months (PMO-M) induced therapeutic levels of dystrophin expression in peripheral muscles, with up to 25-fold increase in diaphragm of mdx mice over PMO alone.

    In this study, MOTS-c was a lab tool or a carrier, part of a mix, or the subject of a lab method. This result is not evidence about taking MOTS-c.

  3. 10AnimalNot a test of MOTS-c aloneAnimal study (in vivo)Efficacy outcome

    In mdx mice, the PMO-M regimen with MOTS‑c improved muscle pathologies.

    In plain wordsIn mice with muscular dystrophy, the long-term MOTS‑c plus PMO treatment improved signs of muscle damage.

    Peptide
    MOTS-c
    Outcome
    DecreaseMuscle pathologies with PMO-M
    Study

    mousen: not reported in source

    mdx mice (Duchenne muscular dystrophy)
    Outcome group
    Muscle injury and repair · Musculoskeletal

    Source sentences

    Summary
    PMO-M improved muscle function and pathologies in mdx mice
    1 context sentence
    Summary · context
    Long-term repeated administration of MOTS-c (500 μg) and PMO at the dose of 12.5 mg/kg/week for 3 weeks followed by 12.5 mg/kg/month for 3 months (PMO-M) induced therapeutic levels of dystrophin expression in peripheral muscles, with up to 25-fold increase in diaphragm of mdx mice over PMO alone.

    In this study, MOTS-c was a lab tool or a carrier, part of a mix, or the subject of a lab method. This result is not evidence about taking MOTS-c.

  4. 11AnimalNot a test of MOTS-c aloneAnimal study (in vivo)Safety outcome

    In mdx mice, no toxicity was detected with the PMO-M regimen with MOTS‑c.

    In plain wordsIn mice with muscular dystrophy, the long-term MOTS‑c plus PMO treatment caused no detectable toxicity.

    Peptide
    MOTS-c
    Outcome
    No significant changeToxicity with PMO-M
    Study

    mousen: not reported in source

    mdx mice (Duchenne muscular dystrophy)
    Outcome group
    Adverse events · Musculoskeletal

    Source sentences

    Summary
    without detectable toxicity.
    1 context sentence
    Summary · context
    Long-term repeated administration of MOTS-c (500 μg) and PMO at the dose of 12.5 mg/kg/week for 3 weeks followed by 12.5 mg/kg/month for 3 months (PMO-M) induced therapeutic levels of dystrophin expression in peripheral muscles, with up to 25-fold increase in diaphragm of mdx mice over PMO alone.

    In this study, MOTS-c was a lab tool or a carrier, part of a mix, or the subject of a lab method. This result is not evidence about taking MOTS-c.

AI wrote and checked these results. AI can make mistakes, so check important results against the source.

Version history

PepBank keeps every version of the data on this study. Status changes from Retraction Watch, Europe PMC and Crossref are listed with their source.

  1. Version 2Current

    9 Oct 2026, 18:17 UTC

    Study results updated

  2. Version 1

    9 Oct 2026, 02:47 UTC

    First added · source update from Europe PMC

Subjects

Terms as the source lists them.

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