Pros of gene therapy
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Pros of Gene Therapy: A Comprehensive Overview
Therapeutic Benefits and Safety Record
Gene therapy has shown remarkable therapeutic benefits and an excellent safety record in recent clinical trials. These advancements have demonstrated the potential to deliver cures for otherwise terminal or severely disabling conditions. Improved vector designs have enabled the safe delivery of therapeutic genes to specific cells, significantly enhancing the efficacy and safety of gene therapy1.
Treatment of Various Diseases
Gene therapy has emerged as a promising treatment option for a variety of human diseases, including blindness, neuromuscular diseases, hemophilia, immunodeficiencies, and cancer. The development of safer and more efficient gene transfer vectors has been crucial in achieving these clinical benefits2. For instance, the use of lentiviral vectors has improved the efficiency of gene transfer to non-dividing cells, leading to clinical benefits in patients with immunodeficiencies and metabolic disorders2.
Advances in Cancer Treatment
Gene therapy offers a potentially beneficial approach to cancer treatment, particularly over traditional chemotherapy, which often lacks selectivity and can cause non-specific toxicity. Innovative genetic approaches are being developed to enhance targeting and expression in a tumor-selective manner, making gene therapy a promising first-line therapy for neoplastic diseases3. Gene-directed enzyme prodrug therapy (GDEPT) is one such approach that has shown effectiveness in both tissue culture and animal models, with ongoing clinical trials demonstrating its potential10.
Gene Editing Technologies
Emerging genome editing technologies, such as CRISPR, offer precise methods for gene addition, ablation, and correction. These technologies are expected to play a major role in the future of gene therapy, providing a precise scalpel for genetic modifications2. The ability to perform genome editing ex vivo or in vivo opens new possibilities for treating a wide range of genetic disorders2.
Regeneration and Immune Response
Gene therapy also leverages the engagement of stem cells to regenerate tissues and the effective exploitation of powerful immune responses to fight cancer. These strategies contribute to the revitalization of gene therapy, making it a versatile tool in modern medicine1.
FDA Approvals and Clinical Successes
The FDA has approved several gene therapy products, including chimeric antigen receptor (CAR) T cells for treating B cell malignancies and adeno-associated virus (AAV) vectors for treating congenital blindness. These approvals mark significant milestones in the field and highlight the clinical successes of gene therapy2.
Potential for Routine Clinical Use
With continuous advancements, gene therapy is poised to become a routine practice in clinical settings. The development of efficient and safe gene delivery vehicles, both viral and non-viral, is crucial for the widespread application of gene therapy5 8. The ability to target gene expression in a tissue- and organ-specific manner further enhances the potential for routine use in treating various diseases3.
Conclusion
Gene therapy has made significant strides in recent years, offering promising treatment options for a range of diseases. The therapeutic benefits, improved safety record, and advancements in gene editing technologies underscore the potential of gene therapy to become a standard treatment modality. As research continues to address the remaining challenges, gene therapy is set to revolutionize the treatment of genetic and acquired diseases, providing hope for many patients worldwide.
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Most relevant research papers on this topic
Gene therapy returns to centre stage
Gene therapy shows promise for treating terminal or severely disabling conditions, with improved vector designs, gene editing, stem cell engagement, and immune responses enhancing its potential for 'cures'.
Rigorous Journal
Highly CitedGene therapy comes of age
Gene therapy has become a promising treatment option for various human diseases, with safer and more efficient vectors leading to clinical benefits in various conditions.
Highly CitedGene Therapies for Cancer: Strategies, Challenges and Successes
Gene therapy shows promise for cancer treatment, with recent advances in safer and more effective vectors and delivery technologies, poised to become a routine first-line therapy for neoplastic diseases.
Highly CitedGene Therapy for Human Genetic Disease?
Gene therapy may improve some human genetic diseases in the future, but for now, it should be researched and only applied when it is proven beneficial and ethical criteria are developed.
Highly CitedGene therapy - promises, problems and prospects
Gene therapy has not lived up to many of its promises, but by 2010, it may become as routine as heart transplants, despite challenges in designing efficient delivery vehicles.
Highly CitedImprovements in Gene Therapy
Gene therapy has improved efficiency and reduced immunogenicity, but adenoviral vectors still face challenges in clinical trials due to immune system reactions.
Entering the Modern Era of Gene Therapy.
Gene therapies show promise in clinical studies and show promise for treating complex diseases, with early successes in single-gene disorders and complex acquired diseases.
Highly CitedGene therapy and DNA delivery systems.
Gene therapy shows promise for treating incurable diseases, but requires safe and efficient DNA delivery vehicles to overcome challenges in transporting large molecules into cells without degradation.
Highly CitedGene therapy and its applications
Gene therapy can treat abnormal genes in cells, potentially becoming the medicine of the next generation if overcome limitations like immunogenicity, mutagenicity, and high costs.
Gene directed enzyme/prodrug therapy of cancer: Historical appraisal and future prospectives
Gene-directed enzyme prodrug therapy (GDEPT) shows potential in selectively eradicating cancer cells while sparing normal tissue, with promising results in tissue culture and animal models.
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