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What is Genetic Engineering?

What is Genetic Engineering?

Gene Therapy

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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...

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Genome editing technologies allow scientists to modify an organism’s DNA via the addition, removal, or rearrangement of genetic material at specific genomic locations. These types of techniques could potentially be used to cure genetic disorders such as hemophilia and sickle cell anemia. One popular and widely used DNA-editing research tool that could lead to safe and effective cures for genetic disorders is the CRISPR-Cas9 system. CRISPR-Cas9 stands for Clustered Regularly Interspaced...

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The CRISPR-Cas system serves as a bacterial defense mechanism against invading genetic elements such as viruses and plasmids, forming the foundation for its adaptation as a powerful genome-editing tool. Originally discovered in prokaryotes, this system has been repurposed to revolutionize genetic engineering across a wide range of organisms, including plants, animals, and humans. The core component, Cas9, is an endonuclease derived from Streptococcus pyogenes, capable of introducing...

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Related Experiment Video

Updated: May 4, 2026

Mouse Genome Engineering Using Designer Nucleases

Mouse Genome Engineering Using Designer Nucleases

Published on: April 2, 2014

Genetic correction using engineered nucleases for gene therapy applications.

Hongmei Lisa Li¹, Takao Nakano, Akitsu Hotta

¹Department of Reprogramming Science, Center for iPS cell Research and Applications (CiRA), Kyoto University, Kyoto, Japan; Japan Society for the Promotion of Science (JSPS), Tokyo, Japan.

Development, Growth & Differentiation

|December 17, 2013

Summary

This summary is machine-generated.

Gene therapy advances, using tools like CRISPR/Cas9, offer precise human genome editing to correct genetic disorders. This review highlights recent progress and clinical applications for gene correction therapies.

Keywords:

gene repair genome editing human genetics

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Last Updated: May 4, 2026

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Area of Science:

Genetics
Molecular Biology
Biotechnology

Background:

Genetic mutations cause congenital disorders and unique phenotypic traits.
Gene therapy has long promised cures for genetic disorders but faced technical hurdles.
Recent advancements in gene editing technologies have emerged.

Purpose of the Study:

To review recent progress in human genome correction approaches.
To emphasize the clinical applications of gene therapy.

Main Methods:

Review of gene editing technologies including meganucleases (MNs), zinc finger nucleases (ZFNs), TAL effector nucleases (TALENs), and CRISPR/Cas9.
Analysis of gene correction strategies for the human genome.
Focus on clinical translation of gene therapy.

Main Results:

Gene editing technologies enable precise modification of target sites in the human genome.
Significant progress has been made in developing gene correction approaches.
Clinical applications of gene therapy are expanding.

Conclusions:

Tailor-made nucleases have revolutionized the potential for precise genome editing.
Gene therapy is increasingly viable for treating genetic disorders.
Further research and development promise expanded clinical use of gene correction.