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Intravenous and Intra-amniotic In Utero Transplantation in the Murine Model
Published on: October 9, 2018
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Delivery technologies for in utero gene therapy
Rohan Palanki1, William H Peranteau2, Michael J Mitchell3
1Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA; Department of Bioengineering, University of Pennsylvania, Philadelphia, PA 19104, USA.
Advanced Drug Delivery Reviews
|November 12, 2020
Summary
In utero gene therapy offers a promising approach to correct genetic disorders before birth. Key challenges involve ensuring biocompatibility and efficient delivery of therapeutic genes within the womb.
Area of Science:
- Reproductive Biology
- Genetics
- Biotechnology
Background:
- Prenatal diagnostics and fetal interventions enable early treatment of congenital diseases.
- In utero gene therapy offers potential for widespread genetic disorder correction during gestation.
- Current limitations include biocompatibility and efficient intracellular transgene delivery for in utero gene therapy.
Purpose of the Study:
- To review delivery considerations for in utero gene therapy components.
- To highlight advancements in viral and non-viral delivery platforms for fetal gene therapy.
- To discuss gene editing delivery technologies and future directions for clinical translation.
Main Methods:
- Review of current literature on prenatal diagnostics and fetal interventions.
- Analysis of viral and non-viral delivery systems for in utero gene therapy.
- Exploration of gene editing technologies and delivery strategies for prenatal applications.
Main Results:
- Progress in prenatal imaging and genetic screening facilitates early intervention.
- Viral and non-viral vectors show promise for overcoming delivery challenges in fetal gene therapy.
- Specialized delivery technologies are emerging for in utero gene editing.
Conclusions:
- In utero gene therapy holds significant potential for treating genetic disorders prenatally.
- Overcoming delivery and biocompatibility challenges is crucial for clinical translation.
- Continued innovation in delivery modalities is essential for advancing fetal gene therapy.
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