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Related Concept Videos

Stem Cell Therapy for Tissue Regeneration01:21

Stem Cell Therapy for Tissue Regeneration

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Stem cell therapy is a method used in regenerative medicine to repair and restore function to damaged tissues and organs. Stem cells have the potential to proliferate and differentiate into various tissue types, making them ideal candidates for tissue regeneration. For example, hematopoietic stem cell transplants are commonly used in blood cancer treatment to replenish damaged bone marrow and restore healthy blood cells.
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Stem cell research aims to find ways to use stem cells to regenerate and repair cellular damage. Over time, most adult cells undergo the wear and tear of aging and lose their ability to divide and repair themselves. Stem cells do not display a particular morphology or function. Adult stem cells, which exist as a small subset of cells in most tissues, keep dividing and can differentiate into a number of specialized cells generally formed by that tissue. These cells enable the body to renew and...
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Gene Therapy00:59

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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...
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Embryonic Stem Cells00:57

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Embryonic stem (ES) cells were first discovered in mice in 1981 by Martin Evans. In 1998, James Thomson identified a method to isolate embryonic stem cells from humans. Human embryonic stem cells (hESCs) are obtained from 3-5 day old embryos that remain unused after an in vitro fertilization procedure.
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Embryonic stem (ES) cells are undifferentiated pluripotent cells, meaning they can produce any cell type in the body. This gives them tremendous potential in science and medicine since they can generate specific cell types for use in research or to replace body cells lost due to damage or disease.
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The ability of induced pluripotent stem cells or iPSCs to differentiate into most body cell types has stimulated repair and regenerative medicine research over the past few decades. iPSC-derived blood cells, hepatocytes, beta islet cells, cardiomyocytes, neurons, and other cell types can repair injuries or regenerate damaged tissue in diseases such as diabetes and neurodegenerative disorders.
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Intravenous and Intra-amniotic In Utero Transplantation in the Murine Model
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Fetal stem cell and gene therapy.

Russell Witt1, Tippi C MacKenzie1, William H Peranteau2

  • 1Division of Pediatric Surgery, University of California, San Francisco, San Francisco, CA, USA.

Seminars in Fetal & Neonatal Medicine
|June 17, 2017
PubMed
Summary
This summary is machine-generated.

In-utero stem cell therapy and gene therapy offer promising treatments for congenital diseases before symptoms appear. Advances in fetal interventions and imaging enhance cell delivery, potentially curing diseases early.

Keywords:
Fetal immunologic toleranceHematopoietic stem cellsIn-utero gene therapyIn-utero transplantationPrenatal gene therapyPrenatal stem cell therapyStem cell niche

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Area of Science:

  • Regenerative Medicine
  • Developmental Biology
  • Genetics

Background:

  • Recent advances in stem cell research, gene editing, prenatal imaging, and fetal interventions are enabling novel therapeutic approaches.
  • The fetal environment presents unique advantages for stem cell therapy, including accessible niches and immune tolerance.

Purpose of the Study:

  • To review existing studies and clinical trials of in-utero therapy for congenital diseases.
  • To discuss the challenges and future strategies for advancing in-utero therapeutic interventions.

Main Methods:

  • Review of preclinical animal models and human clinical attempts at in-utero stem cell transplantation and gene therapy.
  • Analysis of ultrasound-guided techniques for fetal vasculature access and cell delivery.

Main Results:

  • In-utero approaches leverage the fetal environment's stem cell niches and immune tolerance for potential disease cure.
  • Improved ultrasound guidance enhances the safety and efficacy of cell delivery to the fetus.

Conclusions:

  • In-utero therapy holds significant potential for early treatment of congenital diseases, offering social and economic benefits.
  • Overcoming current barriers is crucial for the successful clinical translation of these advanced fetal therapies.