The future of human gene therapy

Area of Science:

• Biotechnology
• Molecular Biology
• Genetics

Background:

• Human gene therapy (HGT) involves transferring nucleic acids to somatic cells for therapeutic effects, aiming to correct genetic defects or overexpress therapeutic proteins.
• Historically, HGT faced high expectations but encountered challenges in demonstrating clinical efficacy and addressing safety concerns, highlighted by the "Gelsinger Case."
• A significant disparity between theoretical potential and practical application has been observed due to limitations in existing technologies.

Purpose of the Study:

• To review the current state and future prospects of human gene therapy.
• To identify key technological advancements and challenges in HGT.
• To highlight promising therapeutic areas and necessary future developments for successful HGT.

Main Methods:

• Review of existing literature and clinical trial outcomes in human gene therapy.
• Analysis of technological advancements in gene delivery systems (viral and non-viral vectors).
• Evaluation of molecular understanding of diseases and manufacturing processes for HGT products.

Main Results:

• Significant progress has been made in enabling technologies, disease understanding, and vector manufacturing.
• Hemophilias (monogenic) and cardiovascular diseases (multigenic) are identified as the most promising areas for current HGT applications.
• Further developments in gene delivery vectors and expression systems are required for diseases like cancer.

Conclusions:

• Human gene therapy holds significant therapeutic potential, but practical challenges in delivery, targeting, and regulation must be overcome.
• Tailored approaches, including modified viral vectors, engineered non-viral vectors, and cell-based delivery, are crucial for specific clinical indications.
• Continued technical advances and increased knowledge are essential to realize the full potential of HGT in treating a wide range of diseases.