Related Experiment Video
Updated: May 20, 2026
06:43
Intravenous and Intra-amniotic In Utero Transplantation in the Murine Model
Published on: October 9, 2018
The case for intrauterine gene therapy
Citra N Mattar1, Simon N Waddington, Arijit Biswas
1Experimental Fetal Medicine Group, Department of Obstetrics and Gynaecology, Yong Loo Lin School of Medicine, National University of Singapore, Singapore.
Summary
Intrauterine gene therapy offers a promising approach to correct genetic disorders before birth, potentially preventing irreversible damage. Research in animal models shows promise, with ongoing studies in primates to assess long-term safety and effectiveness.
Area of Science:
- Genetics
- Developmental Biology
- Gene Therapy
Background:
- Single-gene disorders can lead to severe perinatal complications.
- Early intervention is crucial to prevent irreversible fetal organ damage.
Purpose of the Study:
- To review candidate diseases for intrauterine gene therapy.
- To discuss the current evidence and potential clinical applications of fetal gene therapy.
Main Methods:
- Review of existing literature on intrauterine gene therapy.
- Discussion of proof-of-cure in animal models.
- Assessment of ongoing research in non-human primate models.
Main Results:
- Successful correction of genetic defects demonstrated in animal models.
- Potential for preventing irreversible organ damage and enabling wider biodistribution, including to the brain.
- Ongoing investigations into long-term efficacy, durability, and safety.
Conclusions:
- Intrauterine gene therapy holds significant promise for treating genetic disorders prenatally.
- Further research is needed to evaluate long-term outcomes and maternal safety.
- Clinical applications are being explored for various candidate diseases.
Related Concept Videos
00:59Gene 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...
00:59Gene 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...
01:21Intrauterine Drug Delivery Systems
Controlled-release systems for intravaginal and intrauterine drug delivery have been developed primarily for the administration of contraceptive steroid hormones. These delivery routes circumvent first-pass hepatic metabolism, thereby enhancing bioavailability and allowing for reduced systemic dosages compared to oral administration. Such approaches contribute to improved therapeutic efficacy and patient compliance, particularly in long-term contraceptive regimens.Intravaginal Drug Delivery...
01:16In-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.
01:59CRISPR
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...
01:29Microorganisms 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.