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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...
CRISPR01:59

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 Short...
What is Genetic Engineering?00:49

What is Genetic Engineering?

Overview
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...
Microorganisms in Medicine and Therapeutics01:29

Microorganisms in Medicine and Therapeutics

Microorganisms play a fundamental role in vaccine development, gene therapy, and therapeutic production. Their biological properties are harnessed to advance medicine and public health. Beyond immunization, microorganisms contribute to gut health, antibiotic synthesis, and genetic disease treatment.Live Attenuated and Inactivated VaccinesLive attenuated vaccines, such as the measles, mumps, and rubella (MMR) vaccine, utilize weakened forms of pathogens to closely resemble natural infections.

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Related Experiment Video

Updated: Jun 23, 2026

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

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Published on: October 31, 2012

Fetal gene therapy: opportunities and risks.

Anna M Wagner1, Andreina Schoeberlein, Daniel Surbek

  • 1Dept. of Obstetrics and Gynecology, and Department of Research, Inselspital, Bern University Hospital and University of Bern, Switzerland.

Advanced Drug Delivery Reviews
|May 12, 2009
PubMed
Summary

Prenatal gene therapy using genetically modified hematopoietic stem cells (HSC) offers a promising approach to correct fetal genetic diseases. Advances in vector technology enhance HSC transduction, potentially overcoming immune barriers in the immature fetal system.

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Area of Science:

  • Reproductive Biology
  • Genetics
  • Immunology

Background:

  • Prenatal diagnosis of genetic diseases has advanced significantly.
  • In-utero transplantation of allogeneic hematopoietic stem cells (HSC) shows promise but faces engraftment challenges in non-immunocompromised fetuses.
  • Current limitations restrict HSC transplantation to fetuses with severe immunodeficiency.

Purpose of the Study:

  • To explore gene therapy with genetically modified autologous HSC as a novel approach for treating fetal genetic disorders.
  • To investigate recent advancements in vector constructs and transduction protocols for efficient gene transfer.
  • To assess the potential of the fetal immune system's naiveté for successful prenatal gene therapy.

Main Methods:

  • Utilizing genetically modified autologous HSC to bypass HLA barriers.
  • Employing new vector constructs and transduction protocols to improve the transduction of quiescent hematopoietic cells.
  • Leveraging the immunologic naiveté of the fetal hematopoietic system.

Main Results:

  • Gene therapy with autologous HSC circumvents allogeneic barriers, offering a promising treatment avenue.
  • Improved transduction strategies enhance the potential for successful prenatal gene transfer.
  • The immature fetal immune system may be more receptive to transgene expression, reducing immune reactions.

Conclusions:

  • Prenatal gene therapy using modified autologous HSC presents a viable strategy for correcting genetic deficits in utero.
  • Advancements in gene transfer technology are crucial for the success of fetal gene therapy.
  • Ethical considerations, particularly safety, are paramount before initiating human clinical trials for fetal gene therapy.