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Gene Therapy

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 Therapy

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?

What is Genetic Engineering?

Microorganisms in Medicine and Therapeutics

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.

The Central Dogma

The Central Dogma

The central dogma explains the flow of genetic information from DNA nucleotides to the amino acid sequence of proteins.
RNA is the Missing Link Between DNA and Proteins
In the early 1900s, scientists discovered that DNA stores all the information needed for cellular functions and that proteins perform most of these functions. However, the mechanisms of converting genetic information into functional proteins remained unknown for many years. Initially, it was believed that a single gene is...

In-vitro Mutagenesis

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.

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Related Experiment Video

Updated: Jun 22, 2026

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

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

Published on: February 15, 2019

[Progress of human gene therapy].

Yasufumi Kaneda¹

¹Division of Gene Therapy Science, Graduate School of Medicine, Osaka University.

Nihon Rinsho. Japanese Journal of Clinical Medicine

|June 11, 2009

Summary

Gene therapy shows promise for hereditary diseases and cancers, with stem cell approaches and improved oncolytic viruses as key future directions. Despite early setbacks and adverse events, ongoing research continues to advance therapeutic gene vector applications.

Area of Science:

Biomedical research
Molecular biology
Genetics

Context:

Gene therapy research has been ongoing since the 1990s.
Early gene therapy trials faced challenges, including serious adverse effects like leukemia in X-linked severe combined immunodeficiency (X-SCID) patients.
Despite these challenges, research and clinical trials continue for hereditary diseases, cancers, and cardiovascular conditions.

Purpose:

To review the progress and future directions of gene therapy.
To highlight advancements in gene-loaded vectors and stem cell-based gene therapy.
To identify key areas for future investigation in cancer gene therapy.

Summary:

Therapeutic gene-loaded vectors are being developed for various diseases.
Commercially available gene drugs include p53-loaded adenovirus vector, oncolytic adenovirus, and HGF plasmid DNA.

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Published on: July 14, 2023

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Last Updated: Jun 22, 2026

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

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

Published on: February 15, 2019

A Validatable Droplet Digital Polymerase Chain Reaction Assay for the Detection of Adeno-Associated Viral Vectors in Bioshedding Studies of Tears

A Validatable Droplet Digital Polymerase Chain Reaction Assay for the Detection of Adeno-Associated Viral Vectors in Bioshedding Studies of Tears

Published on: July 14, 2023

Stem cell-based gene therapy is anticipated to be central to hereditary disease treatment.

Future cancer gene therapy research will focus on regulating anti-tumor immunity and enhancing gene-modified oncolytic viruses.

Impact:

Gene therapy holds potential for treating genetic disorders and cancers.
Advancements in vector technology and stem cell applications are crucial for therapeutic success.
Continued research is essential to overcome safety concerns and improve efficacy in gene therapy applications.