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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...
What is Genetic Engineering?00:49

What is Genetic Engineering?

Overview
In-vitro Mutagenesis01:16

In-vitro Mutagenesis

To learn more about the function of a gene, researchers can observe what happens when the gene is inactivated or “knocked out,” by creating genetically engineered knockout animals. Knockout mice have been particularly useful as models for human diseases such as cancer, Parkinson’s disease, and diabetes.
Combination Therapies and Personalized Medicine02:50

Combination Therapies and Personalized Medicine

Combining two or more treatment methods increases the life span of cancer patients while reducing damage to vital organs or tissue from the overuse of a single treatment. Combination therapy also targets different cancer-inducing pathways, thus reducing the chances of developing resistance to treatment.
The combination of the drug acetazolamide and sulforaphane is a good example of combination therapy to treat cancer. The cells in the interior of a large tumor often die due to the hypoxic and...
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...

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Related Experiment Video

Updated: May 13, 2026

Defining Gene Functions in Tumorigenesis by Ex vivo Ablation of Floxed Alleles in Malignant Peripheral Nerve Sheath Tumor Cells
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Defining Gene Functions in Tumorigenesis by Ex vivo Ablation of Floxed Alleles in Malignant Peripheral Nerve Sheath Tumor Cells

Published on: August 25, 2021

Gene therapy--why can it fail?

Georg F Weber1

  • 1University of Cincinnati Medical Center, Winkle College of Pharmacy, Cincinnati, OH 45267-0004, USA. georg.weber@uc.edu

Medical Hypotheses
|March 15, 2013
PubMed
Summary
This summary is machine-generated.

Gene therapy shows promise for treating diseases, but success requires understanding more than just gene sequences. Factors like gene dosage and regulation are crucial for effective gene-based treatments.

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Looking for Driver Pathways of Acquired Resistance to Targeted Therapy: Drug Resistant Subclone Generation and Sensitivity Restoring by Gene Knock-down
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Looking for Driver Pathways of Acquired Resistance to Targeted Therapy: Drug Resistant Subclone Generation and Sensitivity Restoring by Gene Knock-down

Published on: December 11, 2017

Related Experiment Videos

Last Updated: May 13, 2026

Defining Gene Functions in Tumorigenesis by Ex vivo Ablation of Floxed Alleles in Malignant Peripheral Nerve Sheath Tumor Cells
09:37

Defining Gene Functions in Tumorigenesis by Ex vivo Ablation of Floxed Alleles in Malignant Peripheral Nerve Sheath Tumor Cells

Published on: August 25, 2021

Looking for Driver Pathways of Acquired Resistance to Targeted Therapy: Drug Resistant Subclone Generation and Sensitivity Restoring by Gene Knock-down
08:59

Looking for Driver Pathways of Acquired Resistance to Targeted Therapy: Drug Resistant Subclone Generation and Sensitivity Restoring by Gene Knock-down

Published on: December 11, 2017

Area of Science:

  • Molecular Biology
  • Genetics
  • Biotechnology

Background:

  • Reductionism in medicine enables experimental gene expression.
  • Gene therapy offers potential for permanent disease symptom reversal.
  • Recent setbacks in gene therapy raise concerns despite technical advances.

Purpose of the Study:

  • To discuss the hypothesis that DNA sequences alone are insufficient for gene therapy success.
  • To highlight additional factors critical for effective gene therapy.
  • To provide insight into factors influencing gene therapy outcomes.

Main Methods:

  • Review and discussion of factors influencing gene expression and therapy.
  • Analysis of physiological consequences of gene expression.
  • Comparison with principles of pharmacokinetics and pharmacodynamics in drug therapy.

Main Results:

  • Knowledge of DNA sequences is insufficient for successful gene therapy.
  • Gene dosage, transcriptional regulation, and gene product interdependence are critical.
  • Cellular variations in these factors impact therapeutic outcomes.

Conclusions:

  • Gene therapy success hinges on understanding gene dosage, regulation, and interactions.
  • Insights into these factors are analogous to understanding pharmacokinetics/pharmacodynamics in drug therapy.
  • Controlling these factors is key to realizing gene therapy's potential.