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Related Concept Videos

Urine Studies I: Urinalysis01:29

Urine Studies I: Urinalysis

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Urinalysis is a widely used diagnostic test that analyzes urine's physical, chemical, and microscopic characteristics. Healthcare providers use it to detect and monitor various health conditions, including renal disease, urinary tract infections (UTIs), diabetes, and metabolic or systemic disorders.Components of UrinalysisUrinalysis consists of three primary components: physical, chemical, and microscopic examination. Each provides unique insights into the urine sample and, by extension, the...
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DMD-Null mice exhibit severe muscle weakness, impaired regeneration, and deficient satellite cell function.

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Splice Modulation Studies Using Urine-Derived Cells.

Katsuhiko Kunitake1, Yoshitsugu Aoki2

  • 1Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry (NCNP), Kodaira, Tokyo, Japan.

Methods in Molecular Biology (Clifton, N.J.)
|October 1, 2025
PubMed
Summary
This summary is machine-generated.

Antisense oligonucleotide therapy shows promise for Duchenne muscular dystrophy (DMD). This study details a method using human urine-derived cells (UDCs) to create muscle cells for evaluating exon-skipping treatments.

Keywords:
Antisense oligonucleotide (ASO)CD90Duchenne muscular dystrophy (DMD)Exon skippingHuman urine-derived cells (UDCs)ImmunoblottingImmunoblottingImmunocytochemistryImmunocytochemistryMYOD1RT-PCR

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Area of Science:

  • Regenerative Medicine
  • Genetics
  • Drug Discovery

Background:

  • Duchenne muscular dystrophy (DMD) results from dystrophin deficiency.
  • Antisense oligonucleotide (ASO)-based exon skipping is a key therapeutic strategy for DMD.
  • A reliable platform is needed to evaluate ASO efficacy for DMD.

Purpose of the Study:

  • To describe protocols for isolating and differentiating CD90-positive human urine-derived cells (UDCs) into myotubes.
  • To establish a preclinical screening platform for ASO-based exon skipping in DMD.
  • To evaluate the correction of DMD mRNA and protein levels following exon skipping in UDC-derived myotubes.

Main Methods:

  • Isolation of CD90-positive human urine-derived cells (UDCs).
  • Transduction of the MYOD1 gene to differentiate UDCs into myotubes.
  • Evaluation of exon skipping efficacy by assessing DMD mRNA and protein levels.

Main Results:

  • Protocols for UDC isolation and differentiation into myotubes are provided.
  • UDC-derived myotubes serve as a valuable model for evaluating exon-skipping therapies.
  • The method allows for assessment of DMD mRNA and protein correction after ASO treatment.

Conclusions:

  • Human urine-derived cells can be differentiated into myotubes for DMD research.
  • This platform facilitates early-stage preclinical identification of effective ASOs for DMD.
  • The described protocols support the development of splice-modulating therapies for muscular dystrophy.