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...

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...

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.

Teratogenicity

Teratogenicity

The ability of a drug to produce structural deformations and functional abnormalities in the developing embryo or the fetus is called teratogenicity, and the drug producing this effect is known as a teratogen. Teratogenic effects include stillbirth, miscarriage, intrauterine growth restriction, and neurocognitive delay. A teratogen may affect the embryo at different stages of development, which is important in determining the type and extent of the damage. During blastocyst formation, the early...

What is Genetic Engineering?

What is Genetic Engineering?

Genomic Imprinting and Inheritance

Genomic Imprinting and Inheritance

Diploid organisms inherit genetic material through chromosomes from both parents. Copies of the same gene are known as alleles. In most cases, both alleles are simultaneously expressed and allow various cellular processes to function optimally. If one of the alleles is missing or mutated, the expression of the other allele can compensate; however, this is not true for all genes.
The expression of some genes depends on which parent passed the gene to the offspring, through a phenomenon known as...

You might also read

Related Articles

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

Sort by

Same author

Utility of Neonatal Chest X-ray in Patients with Prenatally Diagnosed Congenital Lung Malformations.

Journal of pediatric surgery·2026

Same author

Not All Lucencies are Equal: Distinguishing Pneumatosis in Congenital Heart Disease from Necrotizing Enterocolitis.

Pediatric cardiology·2026

Same author

Combinatorial base editing couples disease correction with lineage amplification in hematopoietic stem and progenitor cells.

bioRxiv : the preprint server for biology·2026

Same author

In Utero Hematopoietic Cell Transplantation in Fetuses with Alpha Thalassemia Major: A Phase 1 Clinical Trial.

Blood advances·2026

Same author

Single-cell spatiotemporal dissection of the human maternal-fetal interface.

Nature·2026

Same author

Infections following minimally invasive repair of pectus excavatum in pediatric patients: a multi-institutional retrospective cohort study of the Western Pediatric Surgery Research Consortium.

Pediatric surgery international·2026

Same journal

Obstetric Care for Patients With Cognitive Disabilities.

Clinical obstetrics and gynecology·2026

Same journal

Dissecting the Americans With Disabilities Act (ADA) Requirements when Approaching Obstetric Patients With Disabilities: How to Ensure Your Spaces are Accessible for All.

Clinical obstetrics and gynecology·2026

Same journal

Clinician Comfort and Barriers to the Obstetric and Gynecologic Care of Patients With Disabilities.

Clinical obstetrics and gynecology·2026

Same journal

Obstetric Care for Patients With Visual Impairments: Preconception Counseling Through Postpartum Care.

Clinical obstetrics and gynecology·2026

Same journal

Menstrual Management and Contraception for Patients With Disabilities.

Clinical obstetrics and gynecology·2026

Same journal

Favoring Elective Hospitalization in Pregnancies With Vasa Previa.

Clinical obstetrics and gynecology·2026

See all related articles

Search research articles

Related Experiment Video

Updated: Oct 17, 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

Prenatal Gene Therapy.

Marisa E Schwab^1,2, Tippi C MacKenzie^1,2

¹Center for Maternal-Fetal Precision Medicine.

Clinical Obstetrics and Gynecology

|October 7, 2021

Summary

This summary is machine-generated.

Prenatal gene therapy offers a potential cure for monogenic diseases by treating them before birth. This approach, including in utero gene therapy, presents advantages over postnatal treatments and is being explored in preclinical studies.

More Related Videos

Pre-Implantation Genetic Testing for Aneuploidy on a Semiconductor Based Next-Generation Sequencing Platform

Pre-Implantation Genetic Testing for Aneuploidy on a Semiconductor Based Next-Generation Sequencing Platform

Published on: August 17, 2022

Mouse In Vivo Placental Targeted CRISPR Manipulation

Mouse In Vivo Placental Targeted CRISPR Manipulation

Published on: April 14, 2023

Related Experiment Videos

Last Updated: Oct 17, 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

Pre-Implantation Genetic Testing for Aneuploidy on a Semiconductor Based Next-Generation Sequencing Platform

Pre-Implantation Genetic Testing for Aneuploidy on a Semiconductor Based Next-Generation Sequencing Platform

Published on: August 17, 2022

Mouse In Vivo Placental Targeted CRISPR Manipulation

Mouse In Vivo Placental Targeted CRISPR Manipulation

Published on: April 14, 2023

Area of Science:

Medical Genetics
Developmental Biology
Translational Medicine

Background:

Monogenic diseases are inherited disorders caused by mutations in a single gene.
Current treatments often manage symptoms after disease onset.
Prenatal intervention holds promise for preventing disease progression.

Purpose of the Study:

To review the rationale and potential of in utero gene therapy.
To discuss clinical trials of postnatal gene therapy.
To examine preclinical in utero gene therapy studies and associated risks.

Main Methods:

Review of existing literature on gene therapy.
Analysis of clinical trial data for postnatal gene therapy.
Evaluation of preclinical data for in utero gene therapy models.

Main Results:

Prenatal gene therapy can potentially cure monogenic diseases before symptoms appear.
In utero gene therapy offers advantages like early treatment and immune tolerance induction.
Preclinical studies show promise, but risks to mother and fetus require careful consideration.

Conclusions:

In utero gene therapy is a promising strategy for treating genetic disorders prenatally.
Further research and careful risk assessment are crucial for clinical translation.
This approach could revolutionize the management of inherited diseases.