Related Experiment Video
Updated: Dec 30, 2025

08:23
CIRCLE-Seq for Interrogation of Off-Target Gene Editing
Published on: November 1, 2024
1.3K
Therapeutic Germline Editing: Sense and Sensibility
1Warren Alpert Medical School, Brown University, Providence, RI, USA.
Trends in Genetics : TIG
|January 27, 2020
Summary
Heritable human genome editing is not clinically ready due to significant scientific, technical, regulatory, and societal hurdles. Key challenges include improving editing efficiency, specificity, and uniformity, alongside legal barriers.
Area of Science:
- Genetics
- Bioethics
- Genomic Medicine
Background:
- Heritable genome editing offers potential therapeutic benefits but faces substantial obstacles.
- Current scientific understanding and technical capabilities are still developing.
Purpose of the Study:
- To outline the major challenges impeding clinical application of heritable human genome editing.
- To highlight the scientific, technical, legal, and societal factors requiring resolution.
Main Methods:
- Review of current scientific literature on genome editing technologies.
- Analysis of existing legal and regulatory frameworks.
- Discussion of ethical and societal implications.
Main Results:
- Significant technical challenges remain in achieving efficient, specific, and uniform genome editing.
- Statutory and regulatory landscapes present considerable roadblocks.
- Societal acceptance and ethical considerations are paramount.
Conclusions:
- Clinical translation of heritable genome editing requires substantial advancements in science and technology.
- Addressing legal, regulatory, and societal concerns is crucial for responsible development.
Related Concept Videos
CRISPR
57.3K
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...
57.3K
What is Genetic Engineering?
79.4K
Overview
79.4K
Homologous Recombination
62.1K
The basic reaction of homologous recombination (HR) involves two chromatids that contain DNA sequences sharing a significant stretch of identity. One of these sequences uses a strand from another as a template to synthesize DNA in an enzyme-catalyzed reaction. The final product is a novel amalgamation of the two substrates. To ensure an accurate recombination of sequences, HR is restricted to the S and G2 phases of the cell cycle. At these stages, the DNA has been replicated already and the...
62.1K
Gene Therapy
27.2K
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...
27.2K
CRISPR/Cas9 Genome Editing
1.5K
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...
1.5K
In-vitro Mutagenesis
15.9K
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.
15.9K

