Kosanke · Molecular therapy : the journal of the American Society of Gene Therapy 2021 · in vitro genomic study · n=30 hiPSC lines

Reprogramming enriches for somatic cell clones with small-scale mutations in cancer-associated genes.

Cited 18 times in the scientific literature.

Level 5 - mechanism / opinion, no new human data

Bench research analyzing human cell lines (CEBM Level 5).

PubMed 33831558 · doi:10.1016/j.ymthe.2021.04.007 · record verified 2026-08-31

What was done

Whole-exome sequencing was performed on 30 human induced pluripotent stem cell (hiPSC) lines. High-accuracy amplicon sequencing was used to trace identified small-scale variants back to parental somatic cell populations. A short hairpin RNA (shRNA) knockdown approach was applied to assess the impact of candidate genes on reprogramming efficiency.

What was found

All analyzed small-scale genetic variants pre-existed in parental cells rather than arising purely de novo. Mutations present in small subpopulations of parental cells became enriched among hiPSC clones during reprogramming. Enriched mutations affected genes governing cell-cycle control, cell death, and pluripotency. The abstract reports no numerical values, frequencies, or effect sizes.

Why it matters

This study shows reprogramming can act as a selective pressure that enriches rare oncogenic and tumor-suppressor mutations from parental cells. It highlights safety concerns and the need for genetic screening before using patient-derived hiPSCs in clinical cell therapies.

Limits

The analysis is restricted to in vitro bench research on 30 hiPSC lines. The abstract provides no exact numerical data, confidence intervals, or in vivo functional tumorigenicity testing.

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