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A Real-time Potency Assay for Chimeric Antigen Receptor T Cells Targeting Solid and Hematological Cancer Cells
Published on: November 12, 2019
Advanced gene therapy system for the treatment of solid tumour: A review
Yuhan Ma1,2,3, Juan Liao4, Hongxia Cheng1,2,3
1Engineering Research Center of Nano-Geomaterials of Ministry of Education, China University of Geosciences, Wuhan, 430074, China.
Abstract:
In contrast to conventional therapies that require repeated dosing, gene therapy can treat diseases by correcting defective genes after a single transfection and achieving cascade amplification, and has been widely studied in clinical settings. However, nucleic acid drugs are prone to catabolism and inactivation. A variety of nucleic acid drug vectors have been developed to protect the target gene against nuclease degradation and increase the transformation efficiency and safety of gene therapy. In addition, gene therapy is often combined with chemotherapy, phototherapy, magnetic therapy, ultrasound, and other therapeutic modalities to improve the therapeutic effect. This review systematically introduces ribonucleic acid (RNA) interference technology, antisense oligonucleotides, and clustered regularly interspaced short palindromic repeat/CRISPR-associated nuclease 9 (CRISPR/Cas9) genome editing. It also introduces the commonly used nucleic acid drug vectors, including viral vectors (adenovirus, retrovirus, etc.), organic vectors (lipids, polymers, etc.), and inorganic vectors (MOFs, carbon nanotubes, mesoporous silica, etc.). Then, we describe the combined gene therapy modalities and the pathways of action and report the recent applications in solid tumors of the combined gene therapy. Finally, the challenges of gene therapy in solid tumor treatment are introduced, and the prospect of application in this field is presented.
Insights
Gene therapy offers a single-dose treatment by correcting genes, but nucleic acid drugs need protection. This review explores gene therapy technologies, vectors, and combination strategies for treating solid tumors.
Area of Science:
- Biomedical Engineering
- Molecular Biology
- Oncology
Background:
- Conventional therapies require repeated dosing, unlike gene therapy's potential for single-dose curative treatments.
- Nucleic acid drugs face challenges like degradation and inactivation, necessitating effective delivery vectors.
- Gene therapy is increasingly combined with other modalities to enhance therapeutic outcomes.
Purpose of the Study:
- To systematically review gene therapy technologies, including RNA interference, antisense oligonucleotides, and CRISPR/Cas9.
- To introduce various nucleic acid drug vectors (viral, organic, inorganic) for improved gene delivery.
- To explore combined gene therapy modalities and their applications in solid tumors.
Main Methods:
- Literature review of gene therapy technologies and delivery systems.
- Analysis of combined gene therapy approaches and their mechanisms.
- Summary of recent advancements and challenges in solid tumor treatment.
Main Results:
- Gene therapy, utilizing technologies like CRISPR/Cas9, offers a promising alternative to conventional treatments.
- Diverse vectors, including viral, organic, and inorganic types, are crucial for protecting nucleic acids and enhancing delivery.
- Combination therapies show potential for improved efficacy in solid tumor treatment.
Conclusions:
- Gene therapy, supported by advanced vectors and combination strategies, presents a significant advancement in treating genetic diseases and solid tumors.
- Overcoming challenges in delivery and safety is key to realizing the full potential of gene therapy.
- Further research into combined modalities and novel vectors will drive future applications in oncology.
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