Britannica Sources used in this answer

How could gene editing be used to reduce the risk of inherited diseases?

Created from Britannica articles using AI. AI can make mistakes, so verify using Britannica articles.

Vaccine development — genetic engineering The generation of vaccines relies heavily on techniques in genetic engineering.

Gene editing offers a transformative approach to addressing inherited diseases by enabling precise modifications to an organism's DNA sequence. Rather than simply providing a functional copy of a gene, modern tools like CRISPR-Cas9 allow scientists to target specific "molecular mistakes" within the human genome and replace them with the correct genetic sequence.12

This process typically involves:
* Targeting: Using molecular guides to identify the precise location of a mutation among billions of base pairs.1
* Cleavage: Employing nucleases to create a double-stranded break at the site of the error.1
* Repair: Utilizing the cell’s natural repair mechanisms to replace the defective sequence with a healthy template.1

While somatic gene therapy aims to treat existing patients by modifying body cells, germline gene therapy seeks to make changes that are inheritable. However, the use of gene editing in humans remains a subject of intense ethical debate, particularly regarding safety, the potential for unintended mutations, and the moral implications of altering the human germline. Ethical principles demand that such powerful technologies be applied with extreme caution to prevent harm and ensure that medical interventions do not exacerbate social inequalities or violate human dignity.31