Ethics of gene therapy

Area of Science:

• Genetics
• Bioethics
• Molecular Biology

Background:

• CRISPR-Cas, base editing, and prime editing enable precise genetic interventions.
• Approved therapies now exist for previously untreatable monogenic disorders.
• Heritable genome editing faces significant ethical debate.

Purpose of the Study:

• To analyze the ethical permissibility of heritable genome editing.
• To differentiate ethical considerations for various applications of heritable genome editing.
• To propose a framework for responsible germline gene editing.

Main Methods:

• Ethical analysis of heritable genome editing applications.
• Distinction between catastrophic monogenic disorders, polygenic risk reduction, and trait enhancement.
• Evaluation of evidence, risks, and benefits for each application.

Main Results:

• Heritable editing for catastrophic monogenic disorders is permissible when alternatives involve severe suffering or early death.
• Current scientific uncertainty precludes clinical application of polygenic interventions.
• Enhancement applications present weaker ethical cases due to positional benefits and social stratification risks.

Conclusions:

• Governance frameworks should permit evidence-supported heritable editing under safeguards and prohibit unsupported applications.
• Ethical considerations should focus on welfare rather than abstract principles.
• Responsible pursuit of heritable gene therapy, where evidence warrants, may be an ethical imperative.