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CRISPR

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...

CRISPR/Cas9 Genome Editing

CRISPR/Cas9 Genome Editing

The CRISPR-Cas system serves as a bacterial defense mechanism against invading genetic elements such as viruses and plasmids, forming the foundation for its adaptation as a powerful genome-editing tool. Originally discovered in prokaryotes, this system has been repurposed to revolutionize genetic engineering across a wide range of organisms, including plants, animals, and humans. The core component, Cas9, is an endonuclease derived from Streptococcus pyogenes, capable of introducing...

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Related Experiment Video

Updated: Aug 6, 2025

Efficient Genome Editing of Mice by CRISPR Electroporation of Zygotes

Efficient Genome Editing of Mice by CRISPR Electroporation of Zygotes

Published on: December 16, 2022

Foetal genome editing.

Sourav K Bose¹, Kara Kennedy, William H Peranteau

¹Center for Fetal Research, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.

Current Opinion in Obstetrics & Gynecology

|March 16, 2023

Summary

This summary is machine-generated.

In-utero gene editing shows promise for treating genetic disorders before birth. Preclinical studies demonstrate safety and feasibility, paving the way for potential one-shot therapies for monogenic diseases.

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Area of Science:

Genetics
Molecular Biology
Developmental Biology

Background:

Modern gene editing, sequencing, and prenatal diagnostics offer new avenues for treating genetic diseases.
Monogenic diseases causing early-life morbidity or mortality are targets for novel therapeutic strategies.

Approach:

This review highlights preclinical studies on in-utero gene editing for monogenic diseases.
It examines advances in cell and enzyme replacement therapies as precedents for in-utero approaches.

Key Points:

Significant progress in postnatal gene editing and in-utero therapies provides a foundation for 'one-shot' treatments.
Preclinical data show benefits of in-utero editing for liver, pulmonary, and multisystemic diseases.
Proof-of-concept studies confirm safety and feasibility across various organs and diseases.

Conclusions:

In-utero gene editing demonstrates potential for treating genetic disorders prenatally.
Clinical translation requires further vector and editing approach optimization for efficiency and safety.