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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...
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.
What is Genetic Engineering?00:49

What is Genetic Engineering?

Overview
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...

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Related Experiment Video

Updated: May 29, 2026

Suspension Culture Production and Purification of Adeno-Associated Virus by Iodixanol Density Gradient Centrifugation for In Vivo Applications
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Suspension Culture Production and Purification of Adeno-Associated Virus by Iodixanol Density Gradient Centrifugation for In Vivo Applications

Published on: February 9, 2024

Updates on current advances in gene therapy.

Jowy Tani1, Faustine, Jomiany Tani Sufian

  • 1Department of Neurology, Wanfang Hospital, Taipei Medical University, Taipei,Taiwan. jowy_tani@yahoo.com

The West Indian Medical Journal
|September 28, 2011
PubMed
Summary

Gene therapy uses genetic manipulation to treat diseases, facing challenges in safety and efficacy. Ongoing research explores viral and non-viral vectors for various conditions, including cancer and genetic disorders.

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Area of Science:

  • Biotechnology
  • Molecular Biology
  • Genetics

Background:

  • Gene therapy aims to treat diseases by altering genetic material.
  • Viral vectors like retroviruses and adenoviruses are common, while non-viral methods are emerging.
  • Successful trials exist for SCID and Leber's congenital amaurosis, but safety concerns persist.

Purpose of the Study:

  • To review current gene therapy approaches, vectors, and applications.
  • To discuss challenges and advancements in gene therapy safety and efficacy.
  • To highlight gene therapy strategies for cancer treatment.

Main Methods:

  • Review of viral vectors (retroviruses, adenoviruses, AAVs, etc.) and non-viral methods (physical, chemical).
  • Analysis of gene therapy applications in genetic diseases, cancers, and other disorders.
  • Examination of safety concerns and proposed improvements.

Main Results:

  • Gene therapy shows promise for genetic disorders and cancers.
  • Viral vectors differ in gene delivery mechanisms (genome integration vs. nuclear).
  • Adverse events highlight the need for enhanced safety protocols.

Conclusions:

  • Gene therapy holds significant potential but requires further development for safe and effective clinical use.
  • Oncolytic viruses, suicide-gene therapy, and immunotherapy are key cancer strategies.
  • Continued research is crucial for broader gene therapy applications.