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Related Concept Videos

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...
Tissue Transplantation01:24

Tissue Transplantation

Tissue transplantation is a significant medical procedure involving the transfer of cells, tissues, or organs from a donor to a recipient, with the primary aim of restoring lost functions. This procedure is crucial in treating a broad spectrum of diseases, including kidney diseases, liver failure, heart disease, and certain types of cancers.
The Biology of Tissue Transplantation
The biology of tissue transplantation hinges on the Major Histocompatibility Complex (MHC) molecules. These molecules...
Bone Marrow Sampling and Transplants01:22

Bone Marrow Sampling and Transplants

Bone marrow transplant is a potential cure for several diseases, including cancer and specific genetic disorders. Notably, this procedure is applicable for patients suffering from aplastic anemia, certain types of leukemia, severe combined immunodeficiency disease (SCID), Hodgkin's disease, non-Hodgkin's lymphoma, multiple myeloma, thalassemia, sickle-cell disease, and certain cancers.
The transplant begins with high doses of chemotherapy and radiation treatment, which aim to destroy the...
Kidney Transplant I: Introduction01:28

Kidney Transplant I: Introduction

A kidney transplant is a surgical approach that involves replacing a non-functioning kidney with a healthy one from a donor. This procedure is often a treatment option for end-stage renal disease (ESRD) patients. The method requires careful recipient selection, including evaluating various medical and psychosocial factors. These criteria vary between transplant centers but generally include assessments of the patient's overall health, adherence to medical recommendations, and lifestyle...
Forced Transdifferentiation01:28

Forced Transdifferentiation

Transdifferentiation, also known as lineage reprogramming, was first discovered by Selman and Kafatos in 1974 in silkmoths. They observed that the moths’ cuticle-producing cells transformed into salt-producing cells. Many such cases of natural transdifferentiation occur in organisms. In humans, pancreatic alpha cells can become beta cells. In newts, the loss of the eye’s lens causes the pigmented epithelial cells to transdifferentiate into the lens cells.
Artificial transdifferentiation occurs...

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Related Experiment Video

Updated: Jun 23, 2026

Preparation and Gene Modification of Nonhuman Primate Hematopoietic Stem and Progenitor Cells
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Preparation and Gene Modification of Nonhuman Primate Hematopoietic Stem and Progenitor Cells

Published on: February 15, 2019

Gene therapy in transplantation.

Jerome M Laurence1, Richard D M Allen, Geoffrey W McCaughan

  • 1Collaborative Transplantation Research Group, Bosch Insitute, Royal Prince Alfred Hospital and University of Sydney, NSW 2006, Australia.

Transplantation Reviews (Orlando, Fla.)
|May 12, 2009
PubMed
Summary

Gene therapy offers a novel approach to enhance organ transplant success by overcoming immune and non-immune barriers. This review examines candidate genes and strategies for clinical application.

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Last Updated: Jun 23, 2026

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Area of Science:

  • Transplantation immunology
  • Molecular biology
  • Regenerative medicine

Background:

  • Current transplant protocols have limitations in achieving long-term graft survival.
  • Immune rejection and non-immune complications remain significant challenges in transplantation.
  • Gene therapy presents a promising avenue for improving transplant outcomes.

Purpose of the Study:

  • To review candidate genes for therapeutic application in transplantation.
  • To explore strategies for delivering gene therapy to overcome transplant barriers.
  • To identify obstacles hindering the clinical implementation of gene therapy for transplants.

Main Methods:

  • Literature review of experimental models in gene therapy for transplantation.
  • Analysis of studies investigating gene candidates and delivery methods.
  • Examination of clinical translation challenges.

Main Results:

  • Various candidate genes show potential in preclinical models to mitigate immune responses and improve graft function.
  • Different gene delivery systems are being explored to enhance efficacy and safety.
  • Significant hurdles remain, including immunogenicity of vectors and efficient gene transfer.

Conclusions:

  • Gene therapy holds considerable promise for advancing transplant medicine.
  • Further research and development are crucial to address safety and efficacy concerns for clinical use.
  • Overcoming implementation obstacles is key to realizing the full potential of gene therapy in transplantation.