Current developments of gene therapy in human diseases.

Area of Science:

• Biotechnology
• Genetics
• Medical Science

Background:

• Gene therapy has emerged as a significant therapeutic strategy for various human diseases.
• Recent years have seen substantial advancements in gene therapy techniques and applications.

Purpose of the Study:

• To provide a comprehensive review of gene therapy developments.
• To explore diverse applications, delivery systems, and targeted approaches in gene therapy.
• To highlight recent clinical trials and the transformative potential of gene therapy.

Main Methods:

• Review of viral (e.g., adeno-associated virus; AAV) and nonviral gene delivery systems.
• Analysis of tissue-specific and cell-type targeting strategies.
• Examination of progress in genetic disorders including lysosomal storage diseases, neurodegenerative disorders, and cardiovascular diseases.
• Evaluation of recent clinical trials involving AAV and CRISPR-Cas9 systems.

Main Results:

• Gene therapy utilizes advanced delivery systems for treating a wide range of genetic disorders.
• Targeted gene delivery to specific tissues like the eye, liver, muscles, and CNS has shown significant progress.
• Clinical trials demonstrate successful outcomes, particularly with AAV vectors and CRISPR technology.

Conclusions:

• Gene therapy holds transformative potential for disease treatment, significantly improving patient quality of life.
• Ongoing research and clinical applications underscore the expanding role of gene therapy in modern medicine.
• This review offers critical insights into the current status and future prospects of gene therapy for researchers and clinicians.