Search research articles

ABOUT JoVE

Overview Leadership Blog JoVE Help Center

AUTHORS

Publishing Process Editorial Board Scope & Policies Peer Review FAQ Submit

LIBRARIANS

Testimonials Subscriptions Access Resources Library Advisory Board FAQ

RESEARCH

JoVE Journal Methods Collections JoVE Encyclopedia of Experiments Archive

EDUCATION

JoVE Core JoVE Business JoVE Science Education JoVE Lab Manual Faculty Resource Center Faculty Site

Terms & Conditions of Use

Related Concept Videos

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...

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.

CRISPR

CRISPR

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...

You might also read

Related Articles

Articles linked to this work by shared authors, journal, and citation graph.

Sort by

Same author

Parent and professional experiences of a clinical trial of prenatal and postnatal stem cell therapy for severe osteogenesis imperfecta.

European journal of human genetics : EJHG·2026

Same author

Safety and efficacy analysis of in vivo lentiviral gene therapy in pre-clinical ARC syndrome models.

Nature communications·2026

Same author

Abdominal fat distribution phenotypes and metabolic risk in premenopausal Asian women: insights beyond general adiposity measures.

Scientific reports·2026

Same author

Surgeons' Perceptions on the Utility of a Conceptual Novel Force Sensor at the Surgeon-Tool Interface: Formative Interview Study.

JMIR formative research·2026

Same author

2025 Consensus Clinical Management Guidelines for Niemann-Pick Disease Type C.

Journal of inherited metabolic disease·2026

Same author

Longitudinal Phenotypic Trajectories in GNAO1-Related Disorders: Defining Disease Progression and Clinical Profiles.

Annals of neurology·2026

Same journal

Beyond Upper Airway Involvement: Evidence of Intrinsic Lung Disease in a Mouse Model of Mucopolysaccharidosis I.

Journal of inherited metabolic disease·2026

Same journal

Immune Dysregulation in Branched Chain Organic Acidemias.

Journal of inherited metabolic disease·2026

Same journal

Long Term Follow-Up After Transplantation in Propionic Acidemia: A Retrospective French Pediatric and Adult Cohort Study.

Journal of inherited metabolic disease·2026

Same journal

Tri-Parametric Assessment of α-Galactosidase A Activity, lysoGb3 and X-Inactivation Aids Genotype-Phenotype Categorization of Fabry Disease Female Patients.

Journal of inherited metabolic disease·2026

Same journal

Mapping the Severity of Phenylalanine Hydroxylase Deficiency.

Journal of inherited metabolic disease·2026

Same journal

Gaucher Disease Treated With Lentiviral-Mediated Gene Therapy: First Case.

Journal of inherited metabolic disease·2026

See all related articles

Search research articles

Related Experiment Video

Updated: Jul 23, 2025

A Novel Surgical Approach for Intratracheal Administration of Bioactive Agents in a Fetal Mouse Model

A Novel Surgical Approach for Intratracheal Administration of Bioactive Agents in a Fetal Mouse Model

Published on: October 31, 2012

Fetal gene therapy.

Simon N Waddington^1,2, William H Peranteau³, Ahad A Rahim⁴

¹EGA Institute for Women's Health, University College London, London, UK.

Journal of Inherited Metabolic Disease

|July 20, 2023

Summary

This summary is machine-generated.

Fetal gene therapy, once a distant concept, is nearing clinical reality. Advances in prenatal diagnosis and fetal surgery pave the way for in-utero gene correction of genetic diseases.

Keywords:

gene therapy inherited metabolic disease

More Related Videos

Intravenous and Intra-amniotic In Utero Transplantation in the Murine Model

Intravenous and Intra-amniotic In Utero Transplantation in the Murine Model

Published on: October 9, 2018

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

Related Experiment Videos

Last Updated: Jul 23, 2025

A Novel Surgical Approach for Intratracheal Administration of Bioactive Agents in a Fetal Mouse Model

A Novel Surgical Approach for Intratracheal Administration of Bioactive Agents in a Fetal Mouse Model

Published on: October 31, 2012

Intravenous and Intra-amniotic In Utero Transplantation in the Murine Model

Intravenous and Intra-amniotic In Utero Transplantation in the Murine Model

Published on: October 9, 2018

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

Area of Science:

Medical Science
Genetics
Biotechnology

Background:

Gene therapy has evolved from an immature concept to a clinical reality.
The trend in treating genetic diseases is shifting towards earlier intervention.
Prenatal diagnosis of genetic diseases is rapidly improving.

Purpose of the Study:

To review the historical development of fetal gene therapy.
To discuss advancements in fetal surgery and diagnostic capabilities.
To explore preclinical gene therapy for inherited metabolic diseases.

Main Methods:

Historical review of gene therapy.
Discussion of advances in fetal surgery and prenatal diagnosis.
Exploration of preclinical data for inherited metabolic diseases.

Main Results:

Gene therapy has matured significantly since the late 1990s.
Improved prenatal diagnostics and fetal surgery enable earlier intervention.
Preclinical research shows promise for fetal gene therapy in metabolic disorders.

Conclusions:

Fetal gene therapy is a developing field with a clear trajectory towards clinical application.
The convergence of technology and clinical will supports the advancement of in-utero gene therapy.
Further research into preclinical models is crucial for the success of fetal gene therapy.