Related Experiment Video
Updated: Aug 6, 2026
07:39
Mouse In Vivo Placental Targeted CRISPR Manipulation
Published on: April 14, 2023
In utero gene editing and precision medicine
Olivia L Katz1, Kara Kennedy1, Natalie F Berger1
1Center for Fetal Research, The Children's Hospital of Philadelphia, Philadelphia, PA, USA; Division of General, Thoracic, and Fetal Surgery, The Children's Hospital of Philadelphia, Philadelphia, PA, USA.
Seminars in Pediatric Surgery
|July 25, 2026
Summary
Advances in prenatal genetic testing and gene editing offer new hope for treating genetic diseases before birth. Regulatory support may accelerate the development of these precision medicine therapies.
Area of Science:
- Genetics
- Genomic Medicine
- Biotechnology
Background:
- Preconception and prenatal genetic testing have advanced significantly.
- Targeted gene therapy technologies are rapidly evolving and gaining regulatory acceptance.
- This creates opportunities for antenatal treatment of genetic diseases.
Purpose of the Study:
- To review recent advances in in utero gene editing and precision medicine.
- To discuss the regulatory landscape facilitating clinical translation.
- To highlight the role of prenatal genetic diagnosis in identifying therapy candidates.
Main Methods:
- Review of recent scientific literature and regulatory guidance.
- Analysis of advancements in gene editing and precision medicine.
- Examination of prenatal diagnostic techniques.
Main Results:
- Significant progress in in utero gene editing and precision medicine.
- Favorable regulatory guidance from the FDA for gene editing therapeutics.
- Established role of prenatal genetic diagnosis in identifying candidates for antenatal therapies.
Conclusions:
- In utero gene editing and precision medicine hold promise for treating genetic diseases antenatally.
- The current regulatory environment is conducive to developing these novel therapeutics.
- Prenatal genetic diagnosis is crucial for patient selection for these advanced treatments.
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