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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...

Gene Therapy

Gene Therapy

Pharmacogenomics: Identification of New Drug Targets

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...

Inborn Errors of Metabolism

Inborn Errors of Metabolism

Phenylketonuria (PKU) is a protein metabolism disorder characterized by high blood levels of the amino acid phenylalanine. This results from a mutation in the gene responsible for phenylalanine hydroxylase, an enzyme that converts phenylalanine into tyrosine. When this enzyme is deficient, phenylalanine builds up in the blood, leading to symptoms such as vomiting, rashes, seizures, growth deficiency, and severe mental retardation. An early diagnosis and a diet restricting phenylalanine intake...

What is Genetic Engineering?

What is Genetic Engineering?

Regulation of Metabolism

Regulation of Metabolism

Cellular needs and conditions vary from cell to cell and change within individual cells over time. For example, the required enzymes and energetic demands of stomach cells are different from those of fat storage cells, skin cells, blood cells, and nerve cells. Furthermore, a digestive cell works much harder to process and break down nutrients during the time that closely follows a meal compared with many hours after a meal. As these cellular demands and conditions vary, so do the amounts and...

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Related Experiment Video

Updated: Mar 12, 2026

Lentiviral Vector-mediated Gene Therapy of Hepatocytes Ex Vivo for Autologous Transplantation in Swine

Lentiviral Vector-mediated Gene Therapy of Hepatocytes Ex Vivo for Autologous Transplantation in Swine

Published on: November 4, 2018

Gene Therapy for Metabolic Diseases.

Randy J Chandler¹, Charles P Venditti¹

¹Medical Genomics and Metabolic Genetics Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, Maryland, USA.

Translational Science of Rare Diseases

|November 18, 2016

Summary

Gene therapy offers a promising treatment for genetic metabolic diseases. Viral gene addition is the primary method used in current clinical trials for these conditions.

Keywords:

Gene Delivery Gene Therapy Genomic Medicine Inborn Errors of Metabolism Metabolic Disorders Therapy for Rare Disease

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Last Updated: Mar 12, 2026

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CRISPR/Cas9-mediated Targeted Integration In Vivo Using a Homology-mediated End Joining-based Strategy

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Published on: March 12, 2018

Area of Science:

Biomedical research
Genetics
Molecular biology

Background:

Gene therapy is a promising treatment for genetic metabolic diseases.
Viral gene addition is the most common approach in current clinical trials.
Gene editing technologies are rapidly advancing.

Purpose of the Study:

To review viral-mediated gene addition approaches for treating metabolic diseases.
To highlight the current clinical trial landscape for gene therapy in metabolic disorders.

Main Methods:

Focus on viral-mediated gene addition strategies.
Analysis of current clinical trial data for metabolic diseases.
Review of scientific literature on gene therapy for metabolic disorders.

Main Results:

Viral gene addition has shown significant success in treating metabolic diseases in animal models and human trials.
Most ongoing clinical trials for metabolic diseases employ viral gene addition.

Conclusions:

Viral gene addition remains a key strategy in the clinical application of gene therapy for metabolic diseases.
Further research and clinical trials are essential to optimize gene therapy outcomes.