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

10 results

SS-31 efficacy in a mouse model of Friedreich ataxia by upregulation of frataxin expression.

Liu Y, Cai J, Shen J, Dong W, Xu L, Fang M, Lin Y, Liu J, Ding Y, Qiao T, Li K

Human molecular genetics1 Dec 2021Cited by 13 (Europe PMC)

PMID34387346 (opens in a new tab)DOI10.1093/hmg/ddab232 (opens in a new tab)PepBankPUB-34387346
Publisher pagePubMedAsk about this study
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Summary

The text of this summary is not shown here, because the article license does not allow reuse. Read it at the source (opens in a new tab)

Text rights: Publisher copyright. Results still quote short attributed sentences.

Results from this study10

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

  1. 01AnimalAnimal study (in vivo)Efficacy outcome

    In Friedreich ataxia mice, SS‑31 significantly improved motor function.

    In plain wordsIn mice with Friedreich ataxia, SS‑31 improved movement ability.

    Peptide
    Elamipretide (SS-31)
    Outcome
    IncreaseMotor function
    Study

    mousen: not reported in source

    GAA expansion-based Friedreich ataxia models (Y47 and YG8R Fxn KIKO mice)
    Outcome group
    Exercise and physical function · Nervous system and cognition
    Studied dose
    1 mg/kg once daily · intraperitoneal · 1 monthAs given in this study (mouse).

    Source sentences

    Summary
    we observed the significant improvement of motor function.
    2 context sentences
    Summary · context
    To further explore the potential of SS-31, we used the GAA expansion-based models, including Y47 and YG8R (Fxn KIKO) mice, primary neurons and macrophages from the mice and cells derived from FRDA patients.
    Summary · context
    After once-daily intraperitoneal injection of 1 mg/kg SS-31 for 1 month, we observed the significant improvement of motor function.
  2. 02AnimalAnimal study (in vivo)Efficacy outcome

    In Friedreich ataxia mice, the vacuolation in dorsal root ganglia was repaired after SS‑31 treatment.

    In plain wordsIn mice with Friedreich ataxia, SS‑31 treatment repaired nerve tissue damage in the dorsal root ganglia.

    Peptide
    Elamipretide (SS-31)
    Outcome
    DecreaseVacuolation in dorsal root ganglia
    Study

    mousen: not reported in source

    GAA expansion-based Friedreich ataxia models (Y47 and YG8R Fxn KIKO mice)
    Outcome group
    Neuroprotection · Nervous system and cognition
    Studied dose
    1 mg/kg once daily · intraperitoneal · 1 monthAs given in this study (mouse).

    Source sentences

    Summary
    The vacuolation in dorsal root ganglia
    3 context sentences
    Summary · context
    were all repaired after SS-31 treatment.
    Summary · context
    To further explore the potential of SS-31, we used the GAA expansion-based models, including Y47 and YG8R (Fxn KIKO) mice, primary neurons and macrophages from the mice and cells derived from FRDA patients.
    Summary · context
    After once-daily intraperitoneal injection of 1 mg/kg SS-31 for 1 month, we observed the significant improvement of motor function.
  3. 03AnimalAnimal study (in vivo)Efficacy outcome

    In Friedreich ataxia mice, lesions in dentate nuclei were repaired after SS‑31 treatment.

    In plain wordsIn mice with Friedreich ataxia, SS‑31 treatment repaired lesions in a brain region called the dentate nuclei.

    Peptide
    Elamipretide (SS-31)
    Outcome
    DecreaseLesions in dentate nuclei
    Study

    mousen: not reported in source

    GAA expansion-based Friedreich ataxia models (Y47 and YG8R Fxn KIKO mice)
    Outcome group
    Neuroprotection · Nervous system and cognition
    Studied dose
    1 mg/kg once daily · intraperitoneal · 1 monthAs given in this study (mouse).

    Source sentences

    Summary
    The vacuolation in dorsal root ganglia, lesions in dentate nuclei and the lost thickness of myelin sheath of spinal cord were all repaired after SS-31 treatment.
    2 context sentences
    Summary · context
    To further explore the potential of SS-31, we used the GAA expansion-based models, including Y47 and YG8R (Fxn KIKO) mice, primary neurons and macrophages from the mice and cells derived from FRDA patients.
    Summary · context
    After once-daily intraperitoneal injection of 1 mg/kg SS-31 for 1 month, we observed the significant improvement of motor function.
  4. 04AnimalAnimal study (in vivo)Efficacy outcome

    In Friedreich ataxia mice, the lost thickness of the spinal cord myelin sheath was repaired after SS‑31 treatment.

    In plain wordsIn mice with Friedreich ataxia, SS‑31 treatment restored thickness of the nerve insulation in the spinal cord.

    Peptide
    Elamipretide (SS-31)
    Outcome
    IncreaseMyelin sheath thickness in spinal cord
    Study

    mousen: not reported in source

    GAA expansion-based Friedreich ataxia models (Y47 and YG8R Fxn KIKO mice)
    Outcome group
    Neuroprotection · Nervous system and cognition
    Studied dose
    1 mg/kg once daily · intraperitoneal · 1 monthAs given in this study (mouse).

    Source sentences

    Summary
    The vacuolation in dorsal root ganglia, lesions in dentate nuclei and the lost thickness of myelin sheath of spinal cord were all repaired after SS-31 treatment.
    2 context sentences
    Summary · context
    To further explore the potential of SS-31, we used the GAA expansion-based models, including Y47 and YG8R (Fxn KIKO) mice, primary neurons and macrophages from the mice and cells derived from FRDA patients.
    Summary · context
    After once-daily intraperitoneal injection of 1 mg/kg SS-31 for 1 month, we observed the significant improvement of motor function.
  5. 05AnimalAnimal study (in vivo)Efficacy outcome

    In Friedreich ataxia mice, SS‑31 dramatically reduced hypertrophic cardiomyocytes.

    In plain wordsIn mice with Friedreich ataxia, SS‑31 strongly reduced enlarged heart muscle cells.

    Peptide
    Elamipretide (SS-31)
    Outcome
    DecreaseHypertrophic cardiomyocytes
    Study

    mousen: not reported in source

    GAA expansion-based Friedreich ataxia models (Y47 and YG8R Fxn KIKO mice)
    Outcome group
    Heart function · Cardiovascular
    Studied dose
    1 mg/kg once daily · intraperitoneal · 1 monthAs given in this study (mouse).

    Source sentences

    Summary
    In addition, the hypertrophic cardiomyocytes and disarrayed abnormal Purkinje cells were dramatically reduced.
    2 context sentences
    Summary · context
    To further explore the potential of SS-31, we used the GAA expansion-based models, including Y47 and YG8R (Fxn KIKO) mice, primary neurons and macrophages from the mice and cells derived from FRDA patients.
    Summary · context
    After once-daily intraperitoneal injection of 1 mg/kg SS-31 for 1 month, we observed the significant improvement of motor function.
  6. 06AnimalAnimal study (in vivo)Efficacy outcome

    In Friedreich ataxia mice, SS‑31 dramatically reduced disarrayed abnormal Purkinje cells.

    In plain wordsIn mice with Friedreich ataxia, SS‑31 strongly reduced disordered Purkinje nerve cells.

    Peptide
    Elamipretide (SS-31)
    Outcome
    DecreaseDisarrayed abnormal Purkinje cells
    Study

    mousen: not reported in source

    GAA expansion-based Friedreich ataxia models (Y47 and YG8R Fxn KIKO mice)
    Outcome group
    Neuroprotection · Nervous system and cognition
    Studied dose
    1 mg/kg once daily · intraperitoneal · 1 monthAs given in this study (mouse).

    Source sentences

    Summary
    In addition, the hypertrophic cardiomyocytes and disarrayed abnormal Purkinje cells were dramatically reduced.
    2 context sentences
    Summary · context
    To further explore the potential of SS-31, we used the GAA expansion-based models, including Y47 and YG8R (Fxn KIKO) mice, primary neurons and macrophages from the mice and cells derived from FRDA patients.
    Summary · context
    After once-daily intraperitoneal injection of 1 mg/kg SS-31 for 1 month, we observed the significant improvement of motor function.
  7. 07AnimalAnimal study (in vivo)Mechanism

    In Friedreich ataxia mice, SS‑31 upregulated frataxin (FXN) expression at the mRNA level in vivo.

    In plain wordsIn mice with Friedreich ataxia, SS‑31 raised the level of frataxin, the protein that is low in the disease.

    Peptide
    Elamipretide (SS-31)
    Outcome
    IncreaseFXN mRNA expression in vivo
    Study

    mousen: not reported in source

    GAA expansion-based Friedreich ataxia models (Y47 and YG8R Fxn KIKO mice)
    Outcome group
    Receptor activity and mechanism · Nervous system and cognition
    Studied dose
    1 mg/kg once daily · intraperitoneal · 1 monthAs given in this study (mouse).

    Source sentences

    Summary
    Interestingly, we found that SS-31 treatment upregulated FXN expression not only at the translational levels as observed in cell culture but also at mRNA levels in vivo.
    2 context sentences
    Summary · context
    To further explore the potential of SS-31, we used the GAA expansion-based models, including Y47 and YG8R (Fxn KIKO) mice, primary neurons and macrophages from the mice and cells derived from FRDA patients.
    Summary · context
    After once-daily intraperitoneal injection of 1 mg/kg SS-31 for 1 month, we observed the significant improvement of motor function.
  8. 08AnimalAnimal study (in vivo)Mechanism

    In Friedreich ataxia mice, mitochondrial morphology was greatly improved in all tested tissues after SS‑31.

    In plain wordsIn mice with Friedreich ataxia, SS‑31 greatly improved mitochondrial shape in all tested tissues.

    Peptide
    Elamipretide (SS-31)
    Outcome
    IncreaseMitochondrial morphology in tested tissues
    Study

    mousen: not reported in source

    GAA expansion-based Friedreich ataxia models (Y47 and YG8R Fxn KIKO mice)
    Outcome group
    Mitochondrial function · Nervous system and cognition
    Studied dose
    1 mg/kg once daily · intraperitoneal · 1 monthAs given in this study (mouse).

    Source sentences

    Summary
    Consequently, mitochondrial morphology and function were greatly improved in all tested tissues.
    2 context sentences
    Summary · context
    To further explore the potential of SS-31, we used the GAA expansion-based models, including Y47 and YG8R (Fxn KIKO) mice, primary neurons and macrophages from the mice and cells derived from FRDA patients.
    Summary · context
    After once-daily intraperitoneal injection of 1 mg/kg SS-31 for 1 month, we observed the significant improvement of motor function.
  9. 09AnimalAnimal study (in vivo)Mechanism

    In Friedreich ataxia mice, mitochondrial function was greatly improved in all tested tissues after SS‑31.

    In plain wordsIn mice with Friedreich ataxia, SS‑31 greatly improved mitochondrial function in all tested tissues.

    Peptide
    Elamipretide (SS-31)
    Outcome
    IncreaseMitochondrial function in tested tissues
    Study

    mousen: not reported in source

    GAA expansion-based Friedreich ataxia models (Y47 and YG8R Fxn KIKO mice)
    Outcome group
    Mitochondrial function · Nervous system and cognition
    Studied dose
    1 mg/kg once daily · intraperitoneal · 1 monthAs given in this study (mouse).

    Source sentences

    Summary
    Consequently, mitochondrial morphology and function were greatly improved in all tested tissues.
    2 context sentences
    Summary · context
    To further explore the potential of SS-31, we used the GAA expansion-based models, including Y47 and YG8R (Fxn KIKO) mice, primary neurons and macrophages from the mice and cells derived from FRDA patients.
    Summary · context
    After once-daily intraperitoneal injection of 1 mg/kg SS-31 for 1 month, we observed the significant improvement of motor function.
  10. 10AnimalAnimal study (in vivo)Efficacy outcome

    In Friedreich ataxia mice, maintaining the therapeutic benefits needed continuous SS‑31 administration.

    In plain wordsIn mice with Friedreich ataxia, the benefits of SS‑31 needed continued dosing to last.

    Peptide
    Elamipretide (SS-31)
    Outcome
    Not applicableMaintenance of benefit after stopping treatment
    Study

    mousen: not reported in source

    GAA expansion-based Friedreich ataxia models (Y47 and YG8R Fxn KIKO mice)
    Outcome group
    Neuroprotection · Nervous system and cognition
    Studied dose
    1 mg/kg once daily · intraperitoneal · 1 monthAs given in this study (mouse).

    Source sentences

    Summary
    Importantly, our data provided additional evidence that the maintenance of the therapeutic benefits needed continuous drug administration.
    2 context sentences
    Summary · context
    To further explore the potential of SS-31, we used the GAA expansion-based models, including Y47 and YG8R (Fxn KIKO) mice, primary neurons and macrophages from the mice and cells derived from FRDA patients.
    Summary · context
    After once-daily intraperitoneal injection of 1 mg/kg SS-31 for 1 month, we observed the significant improvement of motor function.

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

    10 Oct 2026, 08:19 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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