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

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 inserted. The...
Gene Therapy00:59

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 inserted. The...
Pharmacogenomics: Identification of New Drug Targets01:29

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...
In-vitro Mutagenesis01:16

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.
Teratogenicity01:07

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...
Genomic Imprinting and Inheritance02:30

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

Next generation AAV-F capsid gene therapy rescues disease pathology in a model of pyruvate dehydrogenase complex deficiency.

Molecular therapy. Advances·2026
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

Metabolic expenditure, neurodevelopment, and weight gain into early childhood after fetal growth restriction.

Scientific reports·2026
Same author

AAV9-mediated targeting of natural antisense transcript as a novel treatment for Dravet syndrome.

Molecular therapy. Nucleic acids·2026
Same author

Placental apelin expression is low in early-onset fetal growth restriction but there is no association between maternal circulating apelin and pregnancy outcome.

Placenta·2026

Related Experiment Video

Updated: May 21, 2026

A Novel Strategy Combining Array-CGH, Whole-exome Sequencing and In Utero Electroporation in Rodents to Identify Causative Genes for Brain Malformations
08:22

A Novel Strategy Combining Array-CGH, Whole-exome Sequencing and In Utero Electroporation in Rodents to Identify Causative Genes for Brain Malformations

Published on: December 1, 2017

Candidate diseases for prenatal gene therapy.

Anna L David1, Simon N Waddington

  • 1Prenatal Cell and Gene Therapy Group, EGA Institute for Women's Health, University College London, London, UK. a.david@ucl.ac.uk

Methods in Molecular Biology (Clifton, N.J.)
|June 1, 2012
PubMed
Summary

Prenatal gene therapy offers a promising approach to correct genetic defects early in fetal development, potentially treating serious congenital disorders before irreversible damage occurs. This method may enhance treatment efficacy and immune tolerance compared to postnatal gene therapy.

More Related Videos

FISH for Pre-implantation Genetic Diagnosis
07:34

FISH for Pre-implantation Genetic Diagnosis

Published on: February 23, 2011

Semiconductor Sequencing for Preimplantation Genetic Testing for Aneuploidy
09:03

Semiconductor Sequencing for Preimplantation Genetic Testing for Aneuploidy

Published on: August 25, 2019

Related Experiment Videos

Last Updated: May 21, 2026

A Novel Strategy Combining Array-CGH, Whole-exome Sequencing and In Utero Electroporation in Rodents to Identify Causative Genes for Brain Malformations
08:22

A Novel Strategy Combining Array-CGH, Whole-exome Sequencing and In Utero Electroporation in Rodents to Identify Causative Genes for Brain Malformations

Published on: December 1, 2017

FISH for Pre-implantation Genetic Diagnosis
07:34

FISH for Pre-implantation Genetic Diagnosis

Published on: February 23, 2011

Semiconductor Sequencing for Preimplantation Genetic Testing for Aneuploidy
09:03

Semiconductor Sequencing for Preimplantation Genetic Testing for Aneuploidy

Published on: August 25, 2019

Area of Science:

  • Medical Genetics
  • Developmental Biology
  • Gene Therapy

Background:

  • Prenatal gene therapy involves delivering therapeutic genes to fetal cells and tissues during early gestation.
  • This approach aims to correct genetic defects before significant, irreparable damage occurs.
  • Advantages include targeting a larger stem cell population and achieving a higher vector-to-target cell ratio due to smaller fetal size.

Purpose of the Study:

  • To explore the potential and applications of prenatal gene therapy for various congenital and obstetric conditions.
  • To discuss the criteria for selecting candidate diseases for prenatal gene therapy.
  • To outline how prenatal gene delivery might be implemented for specific disorders.

Main Methods:

  • Review of existing research and committee guidelines (e.g., NIH Recombinant DNA Advisory Committee) on prenatal gene therapy.
  • Identification of candidate diseases based on severity (morbidity/mortality) and lack of effective postnatal treatments.
  • Discussion of diagnostic methods (antenatal screening, ultrasound) for identifying treatable fetal conditions.

Main Results:

  • Prenatal gene therapy may offer advantages over postnatal treatments, including improved immune tolerance and efficacy for severe genetic disorders.
  • Candidate diseases include inherited conditions like thalassaemia and lysosomal storage disorders, as well as obstetric issues like fetal growth restriction.
  • Successful application requires prenatal diagnosis and a clear benefit of in-utero gene delivery over alternative therapies.

Conclusions:

  • Prenatal gene therapy presents a viable strategy for treating life-threatening fetal conditions and potentially non-lethal ones if postnatal treatment is less effective.
  • Early intervention can prevent irreversible damage and may facilitate better long-term outcomes.
  • Further research and careful consideration of disease-specific factors are crucial for advancing prenatal gene therapy.