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Related Concept Videos

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...
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...
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...
Pharmacogenomics: Identification of New Drug Targets01:29

Pharmacogenomics: Identification of New Drug Targets

Advances in genomics have profoundly influenced drug discovery by increasing both the speed and accuracy of pharmaceutical development. Pharmacogenomics, which examines how genetic variation influences drug response, facilitates the identification of novel therapeutic targets and enables patient stratification for personalized treatment. These strategies contribute to improved drug efficacy, minimized adverse effects, and more efficient clinical trial design.Mapping genetic differences...
Microorganisms in Medicine and Therapeutics01:29

Microorganisms in Medicine and Therapeutics

Microorganisms play a fundamental role in vaccine development, gene therapy, and therapeutic production. Their biological properties are harnessed to advance medicine and public health. Beyond immunization, microorganisms contribute to gut health, antibiotic synthesis, and genetic disease treatment.Live Attenuated and Inactivated VaccinesLive attenuated vaccines, such as the measles, mumps, and rubella (MMR) vaccine, utilize weakened forms of pathogens to closely resemble natural infections.

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Related Experiment Video

Updated: May 22, 2026

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

Targeted gene therapies: tools, applications, optimization.

Olivier Humbert1, Luther Davis, Nancy Maizels

  • 1Departments of Immunology and Biochemistry, University of Washington School of Medicine, Seattle, WA 98195, USA.

Critical Reviews in Biochemistry and Molecular Biology
|April 26, 2012
PubMed
Summary
This summary is machine-generated.

Targeted gene therapies utilize rare-cutting endonucleases to correct or disable disease-causing genes. Advances in nuclease technology enhance precision and efficiency for treating genetic diseases.

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Non-Viral Engineering of Primary Human T Cells via Homology-Mediated End-Joining Targeted Integration of Large DNA Templates
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Non-Viral Engineering of Primary Human T Cells via Homology-Mediated End-Joining Targeted Integration of Large DNA Templates

Published on: May 9, 2025

Related Experiment Videos

Last Updated: May 22, 2026

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

Non-Viral Engineering of Primary Human T Cells via Homology-Mediated End-Joining Targeted Integration of Large DNA Templates
06:10

Non-Viral Engineering of Primary Human T Cells via Homology-Mediated End-Joining Targeted Integration of Large DNA Templates

Published on: May 9, 2025

Area of Science:

  • Molecular Biology
  • Genetics
  • Biotechnology

Background:

  • Many diseases stem from single-gene mutations or acquired genetic alterations.
  • Targeted gene therapies offer a promising treatment strategy for these conditions.
  • Current therapies often rely on rare-cutting endonucleases for precise DNA modification.

Purpose of the Study:

  • To review the principles and advancements in targeted gene therapies.
  • To highlight the advantages of gene targeting over traditional gene therapy methods.
  • To discuss future directions and challenges in the field.

Main Methods:

  • Gene targeting using rare-cutting endonucleases.
  • Homology-directed repair for gene correction.
  • Gene disruption for ablating disease genes.
  • Genome engineering techniques including mutation, insertion, and deletion.

Main Results:

  • Targeted gene therapies enable precise correction or disruption of disease-associated genes.
  • Gene targeting offers advantages over transgene-based gene therapy approaches.
  • Recent progress is driven by nuclease innovation and improved targeting strategies.

Conclusions:

  • Targeted gene therapies hold significant potential for treating a range of human diseases.
  • Continued advancements in nuclease technology and mechanistic understanding are crucial for future progress.
  • Minimizing off-target effects remains a key focus for safe and effective gene therapy.