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Related Concept Videos

Induced Pluripotent Stem Cells01:13

Induced Pluripotent Stem Cells

Stem cells are undifferentiated cells that divide and produce different types of cells. Ordinarily, cells that have differentiated into a specific cell type are post-mitotic—that is, they no longer divide. However, scientists have found a way to reprogram these mature cells so that they “de-differentiate” and return to an unspecialized, proliferative state. These cells are also pluripotent like embryonic stem cells—able to produce all cell types—and are therefore called induced pluripotent stem...
Induced Pluripotent Stem Cells01:06

Induced Pluripotent Stem Cells

Stem cells are undifferentiated cells that divide and produce different cell types. Ordinarily, cells that have differentiated into a specific cell type are terminally differentiated; however, scientists have found a way to reprogram these mature cells so that they dedifferentiate and return to an unspecialized, proliferative state. These cells are pluripotent like embryonic stem cells—able to produce all cell types—and are called induced pluripotent stem cells (iPSCs).
Somatic cells are...
Gene Therapy00:59

Gene Therapy

Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be inserted. The...
Gene Therapy00:59

Gene Therapy

Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be inserted. The...
Somatic to iPS Cell Reprogramming01:29

Somatic to iPS Cell Reprogramming

Reprogramming alters the gene expression in somatic cells, transforming them into induced pluripotent stem (iPS) cells over several generations. Scientists can reprogram cells by introducing genes for four transcription factors—Oct4, Sox2, Klf4, and c-Myc (OSKM) by viral or non-viral methods. These factors are also known as Yamanaka factors after Shinya Yamanaka, who first generated iPS cells using mouse skin cells. Yamanaka was awarded the Nobel Prize in Physiology or Medicine in 2012 for this...
Methods of Nuclear Reprogramming01:24

Methods of Nuclear Reprogramming

Nuclear reprogramming is a process of transforming one cell type into an unrelated cell type by epigenetic changes that alter the cell’s original gene expression pattern. Such epigenetic changes force cells to express a different set of genes, which play a significant role in inducing transformation into other cell types. Nuclear reprogramming offers applications in reproductive cloning for livestock propagation and regenerative medicine — developing patient-specific cells for injury repair.

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In vivo Reprogramming of Adult Somatic Cells to Pluripotency by Overexpression of Yamanaka Factors
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In vivo Reprogramming of Adult Somatic Cells to Pluripotency by Overexpression of Yamanaka Factors

Published on: December 17, 2013

Inducing pluripotency using in vivo gene therapy.

Roman Gardlik1

  • 1Institute of Molecular Biomedicine, Faculty of Medicine, Comenius University, Sasinkova 4, 811 08 Bratislava, Slovakia. romangardlik@gmail.com

Medical Hypotheses
|May 19, 2012
PubMed
Summary

We hypothesize that in vivo reprogramming of somatic cells could offer an alternative to ex vivo methods for patient-specific therapy. This approach involves direct DNA delivery to target tissues, potentially leveraging natural cellular environments, though safety and efficiency require further investigation.

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Area of Science:

  • Stem Cell Biology
  • Regenerative Medicine
  • Gene Therapy

Background:

  • Induced pluripotent stem (iPS) cells have advanced significantly since 2006.
  • Reprogramming somatic cells into iPS cells is key for patient- and disease-specific therapies.
  • Current ex vivo reprogramming methods face efficiency limitations.

Purpose of the Study:

  • To propose and explore the hypothesis of in vivo reprogramming as an alternative to ex vivo iPS cell generation.
  • To investigate the potential benefits of reprogramming cells within their natural tissue environment.
  • To identify challenges and safety concerns associated with in vivo reprogramming.

Main Methods:

  • The study proposes a conceptual framework for in vivo reprogramming.
  • It suggests direct administration of DNA encoding reprogramming factors into target tissues.
  • Focus is placed on overcoming limitations and addressing safety concerns like tumorigenesis.

Main Results:

  • No direct experimental results are presented as this is a hypothesis-driven concept.
  • The potential for in vivo reprogramming to utilize intrinsic tissue factors is highlighted.
  • Initial data from inflammatory bowel disease studies suggest potential benefits of in vivo gene therapy.

Conclusions:

  • In vivo reprogramming presents a novel therapeutic strategy with potential advantages over ex vivo methods.
  • Significant research is needed to address safety, efficiency, and feasibility.
  • Early indications suggest possible beneficial effects in specific disease contexts like inflammatory bowel disease.