Related Experiment Video
Updated: May 16, 2026

CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications
Published on: August 9, 2022
Stem cell therapy: an exercise in patience and prudence
Huan-Ting Lin1, Makoto Otsu, Hiromitsu Nakauchi
1Center for Stem Cell Biology and Regenerative Medicine, IMSUT, 4-6-1 Shirokanedai Minato-ku, Tokyo, 108-8639, Japan.
Induced pluripotent stem cells (iPSCs) offer novel therapeutic potential for intractable diseases. However, their clinical application requires further validation of safety and efficacy beyond hematopoietic stem cell therapies.
Area of Science:
- Regenerative Medicine
- Epigenetics
- Stem Cell Biology
Background:
- Drug development faces limitations for intractable diseases like organ failure, degenerative disorders, and cancers.
- Stem cells possess self-renewal and multilineage differentiation capabilities, presenting therapeutic opportunities.
- Induced pluripotent stem cells (iPSCs) derived from epigenetic reprogramming are at the forefront of novel therapeutic strategies.
Purpose of the Study:
- To review the current status of stem cell therapy, focusing on induced pluripotent stem cells.
- To discuss the challenges and future prospects of stem cell-based regenerative medicine.
- To provide background on the regulatory networks governing pluripotency.
Main Methods:
- Literature review of stem cell therapy advancements.
- Analysis of epigenetic studies related to pluripotency and cellular reprogramming.
- Discussion of in vitro characterization versus in vivo physiological function of differentiated cells.
Main Results:
- Stem cell therapy, particularly using iPSCs, represents a promising alternative for diseases untreatable by conventional pharmacology.
- Few stem cell therapies, excluding hematopoietic stem cells, have demonstrated proven therapeutic benefit and safety in clinical settings.
- Understanding pluripotency regulation is crucial for advancing stem cell applications.
Conclusions:
- Stem cell therapy holds significant promise but requires rigorous validation for clinical translation.
- Addressing the gap between in vitro potential and in vivo efficacy is critical for widespread adoption.
- Future research should focus on safety, efficacy, and regulatory aspects of stem cell therapies, especially iPSCs.
More Related Videos
Related Concept Videos
Stem Cell Therapy for Tissue Regeneration
Types of Stem Cells used in Stem Cell Therapy
The two main cell types that...
Stem Cell Culture
Induced Pluripotent Stem Cells
Somatic cells are...
Induced Pluripotent Stem Cells
Embryonic Stem Cells
ES cells are grown in a culture medium where they can divide indefinitely, creating ES cell lines. Under certain conditions, ES cells can differentiate, either spontaneously into a variety of...
Embryonic Stem Cells

