Level B· Emerging clinical evidence with positive signalsClinical TrialEurope PMCOpen access

Induced Pluripotent Stem Cells: Hope in the Treatment of Diseases, including Muscular Dystrophies

Gois Beghini D., Iwao Horita S., Cascabulho CM., Anastácio Alves L., Henriques-Pons A.

Clinical Trial, published in Int J Mol Sci (2020) — summary generated from the PubMed abstract.

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Level B· Emerging clinical evidence with positive signalsEvidence level of this study

Several human studies show positive signals, while research methods and sample sizes continue to develop.

  • Level A · Stronger Clinical Evidence
  • Level B · Emerging clinical evidence with positive signals
  • Level C · Early human research exploring benefits
  • Level D · Scientific groundwork from lab and animal studies
  • Emerging · Emerging topic under active research
Read the A–D evidence level guide

This page is generated from the PubMed record. The Thai description is an automated summary of bibliographic fields and the abstract, not a full translation, and is not medical advice.

Study type
Clinical Trial
Journal
Int J Mol Sci (2020)
Reported sample size
—
Source database
Europe PMC
PMID
32751747
PMCID
PMC7432218
DOI
10.3390/ijms21155467
Citations
10

Abstract (original English)

Induced pluripotent stem (iPS) cells are laboratory-produced cells that combine the biological advantages of somatic adult and stem cells for cell-based therapy. The reprogramming of cells, such as fibroblasts, to an embryonic stem cell-like state is done by the ectopic expression of transcription factors responsible for generating embryonic stem cell properties. These primary factors are octamer-binding transcription factor 4 (Oct3/4), sex-determining region Y-box 2 (Sox2), Krüppel-like factor 4 (Klf4), and the proto-oncogene protein homolog of avian myelocytomatosis (c-Myc). The somatic cells can be easily obtained from the patient who will be subjected to cellular therapy and be reprogrammed to acquire the necessary high plasticity of embryonic stem cells. These cells have no ethical limitations involved, as in the case of embryonic stem cells, and display minimal immunological rejection risks after transplant. Currently, several clinical trials are in progress, most of them in phase I or II. Still, some inherent risks, such as chromosomal instability, insertional tumors, and teratoma formation, must be overcome to reach full clinical translation. However, with the clinical trials and extensive basic research studying the biology of these cells, a promising future for human cell-based therapies using iPS cells seems to be increasingly clear and close.

What this study does not prove

  • • This study does not prove SVF is an approved treatment or a replacement for standard care.

Evidence level

Several human studies show positive signals, while research methods and sample sizes continue to develop.

How we grade evidence
HumansMuscular DystrophiesOrganic Cation Transport ProteinsProto-Oncogene Proteins c-mycGene Expression Regulation, DevelopmentalKruppel-Like Transcription FactorsSOXB1 Transcription FactorsInduced Pluripotent Stem CellsCellular ReprogrammingKruppel-Like Factor 4

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