# Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)-Cas Unleashed: Transforming Gene Editing With Breakthroughs, Applications, and Ethical Dilemmas

**Authors:** Balasundari Ramesh, Adegbenro O Fakoya

PMC · DOI: 10.7759/cureus.95908 · 2025-11-01

## TL;DR

CRISPR-Cas technology allows precise gene editing and has promising applications in treating genetic diseases, but raises ethical concerns.

## Contribution

The paper provides an overview of CRISPR enzymes, their applications, and ethical implications.

## Key findings

- CRISPR-Cas9 has been approved for treating sickle cell disease.
- CRISPR holds promise for addressing various genetic disorders and diseases.
- Ethical dilemmas accompany the rapid advancement of CRISPR technology.

## Abstract

The most significant breakthrough in gene editing is the advent of the clustered regularly interspaced short palindromic repeats (CRISPR)-Cas system. This innovative technology enables scientists to insert or delete genes using specific enzymes, facilitating modifications to genomes that can influence an organism's phenotype. The Cas9 enzyme is the most widely used within the CRISPR framework and has already received approval for treating sickle cell disease, with many other applications likely to follow. As this rapidly evolving field continues to advance, it holds great promise for addressing genetic disorders and diseases. This article will explore the various enzymes available in the CRISPR system, the range of diseases and conditions that could be treated using this technology, alternative gene therapy methods, and the ethical considerations surrounding its use.

## Linked entities

- **Proteins:** cas9 (type II CRISPR RNA-guided endonuclease Cas9)
- **Diseases:** sickle cell disease (MONDO:0011382)

## Full-text entities

- **Diseases:** genetic disorders (MESH:D030342), sickle cell disease (MESH:D000755)

## Figures

1 figure with captions in the complete paper: https://tomesphere.com/paper/PMC12594536/full.md

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Source: https://tomesphere.com/paper/PMC12594536