# Advances in Induced Pluripotent Stem Cell Reprogramming and Its Application in Amyotrophic Lateral Sclerosis: A Review

**Authors:** Yingliu Luo, Zhenru Xu, Zunxiong Li

PMC · DOI: 10.1096/fba.2025-00126 · FASEB BioAdvances · 2025-10-31

## TL;DR

This review discusses how iPSC technology has advanced and its use in studying and treating amyotrophic lateral sclerosis.

## Contribution

The paper reviews recent advancements in iPSC reprogramming and their specific applications in ALS research.

## Key findings

- iPSCs are now more efficiently generated using refined factors and delivery systems.
- ALS patient-derived iPSCs help model disease pathology and test potential therapies.
- Chemical reprogramming methods have expanded iPSC utility in neurodegenerative disease research.

## Abstract

Since Yamanaka's landmark achievement in reprogramming somatic cells into induced pluripotent stem cells (iPSCs) using the four key transcription factors—OCT4, SOX2, KLF4, and c‐Myc (OSKM)—iPSC technology has made significant strides. Notable advancements include refining reprogramming factors, delivery systems, somatic cell selection, and optimization of reprogramming conditions, along with developing chemical reprogramming methods. With their unparalleled proliferative capacity and near‐pluripotent differentiation potential, iPSCs have become invaluable tools for investigating neurodegenerative diseases, including amyotrophic lateral sclerosis (ALS). Neuronal models derived from ALS patient‐specific iPSCs, particularly iPSC‐derived motor neurons (iPSC‐MNs), offer a robust platform to recapitulate disease‐specific pathology and investigate the molecular mechanisms underpinning ALS, thereby accelerating the discovery of novel therapeutic strategies. This review highlights the evolution of iPSC technology and its transformative applications in ALS modeling, drug discovery, and therapeutic development.

iPSC Development and iPSC Application in ALS.

## Linked entities

- **Genes:** POU5F1 (POU class 5 homeobox 1) [NCBI Gene 5460], SOX2 (SRY-box transcription factor 2) [NCBI Gene 6657], KLF4 (KLF transcription factor 4) [NCBI Gene 9314], MYC (MYC proto-oncogene, bHLH transcription factor) [NCBI Gene 4609]
- **Diseases:** amyotrophic lateral sclerosis (MONDO:0004976), ALS (MONDO:0004976)

## Full-text entities

- **Genes:** POU5F1 (POU class 5 homeobox 1) [NCBI Gene 5460] {aka OCT3, OCT4, OCT4Borf1, OTF-3, OTF3, OTF4}, SOX2 (SRY-box transcription factor 2) [NCBI Gene 6657] {aka ANOP3, MCOPS3}, MYC (MYC proto-oncogene, bHLH transcription factor) [NCBI Gene 4609] {aka MRTL, MYCC, bHLHe39, c-Myc}, KLF4 (KLF transcription factor 4) [NCBI Gene 9314] {aka EZF, GKLF}
- **Diseases:** neurodegenerative diseases (MESH:D019636), ALS (MESH:D000690)
- **Species:** Homo sapiens (human, species) [taxon 9606]

## Full text

_Full body text omitted from this summary view._ Fetch the complete paper as Markdown: https://tomesphere.com/paper/PMC12577567/full.md

## Figures

3 figures with captions in the complete paper: https://tomesphere.com/paper/PMC12577567/full.md

## References

141 references — full list in the complete paper: https://tomesphere.com/paper/PMC12577567/full.md

---
Source: https://tomesphere.com/paper/PMC12577567