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Updated: Feb 5, 2026
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In utero and ex vivo Electroporation for Gene Expression in Mouse Retinal Ganglion Cells
Published on: September 24, 2009
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Future AAVenues for In Utero Gene Therapy
1The Department of Surgery and the Center for Maternal-Fetal Precision Medicine, University of California, San Francisco, San Francisco, CA, USA.
Cell Stem Cell
|September 8, 2018
Summary
Prenatal gene therapy using viral vectors shows promise. A study demonstrated improved neurologic function after in utero viral vector injection, suggesting fetal gene therapy is nearing clinical use.
Area of Science:
- Reproductive biology
- Neurology
- Genetics
Background:
- Fetal gene therapy is an emerging field.
- Viral vectors are key delivery tools for gene therapy.
Purpose of the Study:
- To evaluate the potential of in utero gene therapy.
- To assess the safety and efficacy of prenatal viral vector delivery.
Main Methods:
- Prenatal intracranial injection of a viral vector in a fetal model.
- Assessment of neurologic function post-injection.
Main Results:
- Improved neurologic function was observed following prenatal viral vector administration.
- The study supports the feasibility of in utero gene therapy.
Conclusions:
- Prenatal gene therapy via viral vectors is a promising approach.
- This research indicates that fetal gene therapy may soon be applicable in clinical settings.
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