Background:

CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) has revolutionized the field of gene editing, offering unparalleled precision and efficiency in genetic manipulation. Its application in pharmacology is transformative, promising novel therapeutic solutions for monogenic disorders, cancers, and infectious diseases. However, the integration of CRISPR-based therapies into clinical practice presents significant ethical and practical challenges. Ethical concerns such as germline editing, consent, and healthcare equity, coupled with technical hurdles like off-target effects and delivery mechanisms, demand careful scrutiny.

Aim:

This paper aims to explore the ethical and practical implications of CRISPR-based therapeutics in pharmacology. It evaluates the technology's potential to address unmet medical needs while identifying the challenges that hinder its equitable and responsible deployment.

Methods:

A comprehensive literature review was conducted, synthesizing data from peer-reviewed articles, clinical trial databases, and ethical frameworks. Case studies of CRISPR-based interventions were analyzed to assess therapeutic outcomes and associated challenges. Additionally, regulatory policies from leading global jurisdictions were reviewed to contextualize the governance of gene editing technologies.

Results:

CRISPR-based therapeutics have demonstrated promising outcomes in treating monogenic diseases such as sickle cell anemia and beta-thalassemia, as well as in advancing cancer immunotherapy. However, ethical dilemmas surrounding germline editing and societal inequities in treatment access persist. Practical challenges include the precise delivery of CRISPR components, risk of off-target effects, and high costs of therapy development. Current regulatory frameworks vary significantly across regions, revealing gaps in international governance and ethical oversight.

Conclusion:

CRISPR technology holds transformative potential in pharmacology, yet its integration into clinical practice requires a balance between innovation and responsibility. Addressing ethical concerns, advancing delivery systems, and establishing robust global regulatory frameworks are crucial to ensuring that CRISPR-based therapeutics are safe, effective, and accessible. Interdisciplinary collaboration and proactive policy-making are essential to maximizing the societal benefits of this groundbreaking technology.