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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...
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...
Targeted Cancer Therapies02:57

Targeted Cancer Therapies

The targeted cancer therapies, also known as “molecular targeted therapies,” take advantage of the molecular and genetic differences between the cancer cells and the normal cells. It needs a thorough understanding of the cancer cells to develop drugs that can target specific molecular aspects that drive the growth, progression, and spread of cancer cells without affecting the growth and survival of other normal cells in the body.
There are several types of targeted therapies against specific...
What is Genetic Engineering?00:49

What is Genetic Engineering?

Overview
Treatment Resistent Cancers02:56

Treatment Resistent Cancers

Cancer is the second leading cause of death in the United States. A cancer cell is genetically unstable and hence can mutate faster. They can also modify their microenvironment and escape immune surveillance. The difficulties in treating cancer are further compounded by the emergence of rapid resistance to anticancer drugs. The most common ways to attain resistance in cancer cells include alteration in drug transport and metabolism, modification of drug target, elevated DNA damage response, or...

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Related Experiment Video

Updated: Jul 10, 2026

In vivo Application of the REMOTE-control System for the Manipulation of Endogenous Gene Expression
08:54

In vivo Application of the REMOTE-control System for the Manipulation of Endogenous Gene Expression

Published on: March 29, 2019

[Gene therapy in The Netherlands].

E A M Schenk-Braat1, M M K B van Mierlo, G A P Hospers

  • 1Erasmus MC-Centrum, Postbus 2040, 3000 CA Rotterdam. e.schenk-braat@erasmusmc.nl

Nederlands Tijdschrift Voor Geneeskunde
|October 24, 2007
PubMed
Summary

Gene therapy shows promise for genetic disorders like severe combined immune deficiency and haemophilia B, with ongoing global research. While generally safe, its effectiveness for cancer treatment remains limited.

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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

Related Experiment Videos

Last Updated: Jul 10, 2026

In vivo Application of the REMOTE-control System for the Manipulation of Endogenous Gene Expression
08:54

In vivo Application of the REMOTE-control System for the Manipulation of Endogenous Gene Expression

Published on: March 29, 2019

In vivo Reprogramming of Adult Somatic Cells to Pluripotency by Overexpression of Yamanaka Factors
12:12

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

Published on: December 17, 2013

Area of Science:

  • Biomedical research
  • Clinical trials
  • Gene therapy

Context:

  • Global research on gene therapy's clinical utility is extensive.
  • Two gene therapy products recently approved in China.
  • Over 200 Dutch patients treated in gene therapy clinical trials.

Purpose:

  • To review the current status and clinical utility of gene therapy.
  • To assess the safety and efficacy of gene therapy for various conditions.

Summary:

  • Gene therapy is generally safe based on published results.
  • Effective for certain genetic disorders like severe combined immune deficiency and haemophilia B.
  • Efficacy in cancer treatment appears limited currently.

Impact:

  • Gene therapy offers potential for treating genetic disorders.
  • Further research is needed to improve cancer treatment efficacy.
  • Accumulating clinical experience informs future gene therapy applications.