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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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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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Stem Cell Culture01:17

Stem Cell Culture

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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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Embryonic Stem Cells00:57

Embryonic Stem Cells

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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 Cells00:58

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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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Chemogenetic Regulation in Reprogrammed Stem Cell-derived Precursor Cells in Treating Neurodegenerative Diseases
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Gene therapy using stem cells.

Erin R Burnight1, Luke A Wiley1, Robert F Mullins1

  • 1The Stephen A. Wynn Institute for Vision Research, Department of Ophthalmology and Visual Sciences, University of Iowa, Iowa City, Iowa 52242.

Cold Spring Harbor Perspectives in Medicine
|November 15, 2014
PubMed
Summary
This summary is machine-generated.

Gene therapy and patient-derived stem cells offer new hope for inherited retinal degenerations. These approaches can restore vision by targeting cells or replacing lost ones.

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Area of Science:

  • Ophthalmology
  • Regenerative Medicine
  • Gene Therapy

Background:

  • Viral-mediated gene augmentation therapy shows promise for retinal degenerative disorders.
  • Animal models are crucial for assessing the efficacy and safety of gene therapies.
  • In some cases, in vivo gene therapy is limited by the absence of target cells.

Purpose of the Study:

  • To explore the potential of gene therapy and patient-derived stem cells for vision restoration.
  • To address limitations of current gene therapy approaches in retinal degenerative diseases.

Main Methods:

  • Review of viral-mediated gene augmentation therapy.
  • Exploration of somatic cell reprogramming for autologous cell transplantation.
  • Discussion of therapeutic strategies for inherited retinal degenerations.

Main Results:

  • Gene therapy has demonstrated success in restoring vision in preclinical studies.
  • Somatic cell reprogramming offers a source of autologous cells for transplantation.
  • Combined approaches may overcome limitations of single-modality treatments.

Conclusions:

  • Gene therapy and stem cell-based treatments are promising for inherited retinal degenerations.
  • Patient-derived stem cells provide a viable alternative when target cells are absent.
  • Further development of these strategies holds potential for widespread vision restoration.