Gene therapy: principles, challenges and use in clinical practice.

Area of Science:

• Medical Science
• Biotechnology
• Genetics

Background:

• Gene therapy is an emerging medical field with licensed treatments for immune deficiency, spinal muscular atrophy, hemophilia, and cancers.
• Numerous gene therapy products are undergoing preclinical and clinical trials, indicating rapid advancements.

Purpose of the Study:

• To review the fundamental principles of gene therapy.
• To discuss the challenges, risks, benefits, and adverse effects associated with gene therapy.
• To illustrate these aspects using examples of hemophilia and spinal muscular atrophy.

Main Methods:

• Overview of current gene therapy approaches, including gene addition/augmentation and in situ gene editing.
• Emphasis on the necessity of long-term patient observation for safety, efficacy, predictability, and durability.
• Discussion of safety concerns like immune reactions and insertional mutagenesis-related cancer risk.

Main Results:

• Gene addition and augmentation are currently the most common gene therapy methods.
• Gene editing techniques are emerging as more precise approaches.
• Long-term monitoring is essential due to potential safety concerns, including immune responses and cancer risk.

Conclusions:

• Gene therapy holds significant potential for treating genetic diseases.
• Further clarification of issues is needed to ensure safe, effective, and accessible gene therapy products.
• Addressing ethical, regulatory, and reimbursement challenges is vital for widespread adoption.