Gene therapy: a double-edged sword with great powers

Area of Science:

• Biotechnology
• Genetics
• Medical Science

Background:

• Gene therapy involves transferring genetic material to treat diseases.
• Significant advancements have led to over two dozen approved gene therapies globally.
• Concerns regarding efficacy and safety have historically accompanied gene therapy development.

Purpose of the Study:

• To review various gene therapy strategies.
• To discuss the clinical outcomes of approved gene therapies.
• To analyze the safety considerations of gene therapy.

Main Methods:

• Exploration of gene delivery methods (ex vivo vs. in vivo).
• Comparison of gene addition versus genome editing techniques.
• Differentiation between inherited and acquired disease applications.
• Analysis of somatic versus germline gene therapy approaches.

Main Results:

• Approved gene therapies demonstrate clinical success.
• Somatic gene therapy has proven efficient and safe for clinical use.
• Germline gene therapy currently presents significant efficiency and safety challenges.

Conclusions:

• Technological improvements support the clinical viability of somatic gene therapy.
• Germline gene therapy is not recommended for clinical practice due to unresolved issues.
• Ongoing research is crucial for advancing gene therapy safety and efficacy.