Jove
Visualize
Contact Us
JoVE
x logofacebook logolinkedin logoyoutube logo
ABOUT JoVE
OverviewLeadershipBlogJoVE Help Center
AUTHORS
Publishing ProcessEditorial BoardScope & PoliciesPeer ReviewFAQSubmit
LIBRARIANS
TestimonialsSubscriptionsAccessResourcesLibrary Advisory BoardFAQ
RESEARCH
JoVE JournalMethods CollectionsJoVE Encyclopedia of ExperimentsArchive
EDUCATION
JoVE CoreJoVE BusinessJoVE Science EducationJoVE Lab ManualFaculty Resource CenterFaculty Site
Terms & Conditions of Use
Privacy Policy
Policies

Related Concept Videos

Gene Therapy00:59

Gene Therapy

26.3K
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...
26.3K
In-vitro Mutagenesis01:16

In-vitro Mutagenesis

15.5K
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.
15.5K
CRISPR01:59

CRISPR

53.9K
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...
53.9K
What is Genetic Engineering?00:49

What is Genetic Engineering?

76.5K
Overview
76.5K

You might also read

Related Articles

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

Sort by
Same author

Plug-and-play assembly of biodegradable ionizable lipids for potent mRNA delivery and gene editing in vivo.

Proceedings of the National Academy of Sciences of the United States of America·2026
Same author

Genomic Contributors to Congenital Diaphragmatic Hernia: Results of Exome Sequencing in 560 Probands and Cross Reference of Findings in an Independent Cohort.

American journal of medical genetics. Part A·2026
Same author

Early Childhood Outcomes in Severe Congenital Diaphragmatic Hernia Treated with Fetoscopic Endoluminal Tracheal Occlusion (FETO) versus Non-FETO at a Single Center.

Fetal diagnosis and therapy·2026
Same author

Maternal Subcutaneous Seroma following Open Maternal Fetal Surgery for Closure of Fetal Open Neural Tube Defects.

Fetal diagnosis and therapy·2026
Same author

Non-synthetic vs synthetic local drug delivery agents for the treatment of periodontitis in adults: a systematic review and meta-analysis protocol.

JBI evidence synthesis·2025
Same author

Improved specificity and efficiency of in vivo adenine base editing therapies with hybrid guide RNAs.

Nature biomedical engineering·2025
Same journal

Hyocholic acids: Third bile acids for neonatal health.

Trends in molecular medicine·2026
Same journal

Clonal hematopoiesis in Alzheimer's brain: Protective, pathogenic, and context-dependent?

Trends in molecular medicine·2026
Same journal

Targeting amino acid metabolism in hepatocellular carcinoma.

Trends in molecular medicine·2026
Same journal

Turning perfusion into repair through ferroptosis blockade.

Trends in molecular medicine·2026
Same journal

CaMKK2: A tumor stress-integration node.

Trends in molecular medicine·2026
Same journal

Precision gene editing: From proof-of-concept to curative therapies.

Trends in molecular medicine·2026
See all related articles

Related Experiment Video

Updated: Nov 1, 2025

A Mouse Model of in Utero Transplantation
09:11

A Mouse Model of in Utero Transplantation

Published on: January 27, 2011

19.7K

InUtero Gene Therapy: Progress and Challenges.

Sourav K Bose1, Pallavi Menon1, William H Peranteau1

  • 1The Center for Fetal Research, Division of General, Thoracic and Fetal Surgery, The Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.

Trends in Molecular Medicine
|June 28, 2021
PubMed
Summary
This summary is machine-generated.

In utero gene therapy offers a promising approach to treating serious diseases before birth. Careful consideration of clinical, social, and institutional factors is crucial for its successful implementation.

Keywords:
CRISPRfetalgene editinggene therapyin uterolipid nanoparticle

More Related Videos

Intravenous and Intra-amniotic In Utero Transplantation in the Murine Model
06:43

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

Published on: October 9, 2018

9.8K
A Novel Surgical Approach for Intratracheal Administration of Bioactive Agents in a Fetal Mouse Model
10:12

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

Published on: October 31, 2012

16.7K

Related Experiment Videos

Last Updated: Nov 1, 2025

A Mouse Model of in Utero Transplantation
09:11

A Mouse Model of in Utero Transplantation

Published on: January 27, 2011

19.7K
Intravenous and Intra-amniotic In Utero Transplantation in the Murine Model
06:43

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

Published on: October 9, 2018

9.8K
A Novel Surgical Approach for Intratracheal Administration of Bioactive Agents in a Fetal Mouse Model
10:12

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

Published on: October 31, 2012

16.7K

Area of Science:

  • Perinatal medicine
  • Gene therapy
  • Developmental biology

Background:

  • In utero gene therapy presents a novel strategy for addressing lethal and morbid perinatal diseases.
  • The unique physiological characteristics of the fetus, including small size and immune tolerance, make it a suitable candidate for prenatal genetic interventions.
  • Successful implementation requires addressing multifaceted challenges beyond the purely scientific.

Purpose of the Study:

  • To explore the potential of in utero gene therapy for treating perinatal diseases.
  • To identify key factors influencing the feasibility and success of prenatal genetic treatments.
  • To highlight the importance of considering clinical, social, and institutional aspects.

Main Methods:

  • Review of existing literature on fetal development and gene therapy.
  • Analysis of physiological advantages of the fetal environment for gene therapy.
  • Discussion of clinical, social, and institutional considerations.

Main Results:

  • The fetus possesses unique characteristics, such as small size and immune tolerance, that are advantageous for gene therapy.
  • Dosing efficiency can be optimized in the fetal context.
  • Numerous non-biological factors significantly impact the potential for successful in utero gene therapy.

Conclusions:

  • In utero gene therapy holds significant promise for treating severe perinatal conditions before birth.
  • Realizing this potential necessitates a comprehensive approach that integrates scientific advancements with clinical, social, and institutional readiness.
  • Further research and strategic planning are essential to overcome barriers and translate this therapeutic approach into clinical practice.