摘要
基于单克隆抗体(mAbs)的治疗是当前治疗免疫紊乱、肿瘤和感染性疾病的最成功方案之一。衍生于腺相关病毒(AAV)的载体对于递送编码mAbs的基因是非常有吸引力的,因为它们允许长期表达,从而减少给药次数。他们也能穿透生物膜障碍如穿透血脑屏障来转换免疫特异性器官里面的细胞。近期AAV相关的动物实验研究证实 了AAV传递足够数目的抗体以对慢性传染性疾病进行有效的免疫保护几个月甚至几年。根据治疗及其进展,治疗成功地用于预防或治疗途径。本文讨论了AAV应用于mAb和免疫黏附素传递的优势和局限性,以及载体设计和抗体工程的近期的研究进展。载体设计的优化能够提高mAbs的动力学及表达水平,而蛋白质工程能够加强转基因产物性质。此外,本文提供了对对包括阿尔茨海默病,肌萎缩性侧索硬化和癌症的慢性疾病的临床前研究以及传染病的详尽综述。
关键词: 单克隆抗体,重组腺相关病毒,抗体传导,慢性感染性疾病,载体设计
Current Gene Therapy
Title:Antibody Delivery Mediated by Recombinant Adeno-associated Virus for the Treatment of Various Chronic and Infectious Diseases
Volume: 16 Issue: 6
关键词: 单克隆抗体,重组腺相关病毒,抗体传导,慢性感染性疾病,载体设计
摘要: Monoclonal antibodies (mAbs) based-therapies are currently one of the most successful strategies to treat immune disorders, cancer and infectious diseases. Vectors derived from adenoassociated virus (AAV) are very attractive to deliver the genes coding the mAbs because they allow long-term expression thus, reducing the number of administrations. They can also penetrate biological barriers such as the blood-brain-barrier to transduce cells localized in immunoprivileged organs. Recent animal studies with AAV have demonstrated the capacity of AAV to deliver sufficient quantity of antibodies to confer an efficient immunoprotection against chronic and infectious diseases for several months to years. The treatment was successfully applied either for prophylaxis or therapeutic use, depending on the disease and its progression. In this review, we discuss the advantages and the limitations of AAV for mAb and immunoadhesin delivery. Recent advances in vector design and antibody engineering are also presented. Optimization of the vector design can improve the kinetic and the level of mAbs expression whereas protein engineering can enhance transgene product properties. Furthermore, an exhaustive review of pre-clinical studies for chronic diseases including Alzheimer disease, amyotrophic lateral sclerosis and cancer is presented as well as for infectious diseases.
Export Options
About this article
Cite this article as:
Antibody Delivery Mediated by Recombinant Adeno-associated Virus for the Treatment of Various Chronic and Infectious Diseases, Current Gene Therapy 2016; 16 (6) . https://dx.doi.org/10.2174/1566523217666170102111251
DOI https://dx.doi.org/10.2174/1566523217666170102111251 |
Print ISSN 1566-5232 |
Publisher Name Bentham Science Publisher |
Online ISSN 1875-5631 |
Call for Papers in Thematic Issues
Programmed Cell Death Genes in Oncology: Pioneering Therapeutic and Diagnostic Frontiers (BMS-CGT-2024-HT-45)
Programmed Cell Death (PCD) is recognized as a pivotal biological mechanism with far-reaching effects in the realm of cancer therapy. This complex process encompasses a variety of cell death modalities, including apoptosis, autophagic cell death, pyroptosis, and ferroptosis, each of which contributes to the intricate landscape of cancer development and ...read more
Related Journals
- Author Guidelines
- Graphical Abstracts
- Fabricating and Stating False Information
- Research Misconduct
- Post Publication Discussions and Corrections
- Publishing Ethics and Rectitude
- Increase Visibility of Your Article
- Archiving Policies
- Peer Review Workflow
- Order Your Article Before Print
- Promote Your Article
- Manuscript Transfer Facility
- Editorial Policies
- Allegations from Whistleblowers
- Announcements
Related Articles
-
The Trigeminal System in Birds and Nociception
Central Nervous System Agents in Medicinal Chemistry Daidzein and its Effects on Brain
Current Medicinal Chemistry ACKNOWLEDGEMENTS TO THE REVIEWERS
Current Neuropharmacology Consequences of Aberrant Insulin Regulation in the Brain: Can Treating Diabetes be Effective for Alzheimers Disease
Current Neuropharmacology Bioactive Compounds Containing Benzoxadiazole, Benzothiadiazole, Benzotriazole
Current Bioactive Compounds Advanced Assessment of the Endogenous Hormone Level as a Potential Biomarker of the Urogenital Tract Cancer
Combinatorial Chemistry & High Throughput Screening Rhein-Huprine Derivatives Reduce Cognitive Impairment, Synaptic Failure and Amyloid Pathology in AβPPswe/PS-1 Mice of Different Ages
Current Alzheimer Research The Paths to Neurodegeneration in Genetic Parkinson's Disease
CNS & Neurological Disorders - Drug Targets Recent Developments of Structure Based β-Secretase Inhibitors for Alzheimers Disease
Current Topics in Medicinal Chemistry Patent Selections:
Recent Patents on CNS Drug Discovery (Discontinued) Targeting Protein-Protein Interactions: A Promising Avenue of Anti-HIV Drug Discovery
Current Medicinal Chemistry Proteasome Regulators: Activators and Inhibitors
Current Medicinal Chemistry Commentary (Research Highlights: TRPV-ing up Pain for a Long Life)
CNS & Neurological Disorders - Drug Targets Botulinum A Toxin Intravesical Injections for Painful Bladder Syndrome: Impact Upon Pain, Psychological Functioning and Quality of Life
Current Drug Delivery Inhibition of Ceramide Metabolism Key Enzymes and its Implication in Cell Physiology and Pathology
Current Enzyme Inhibition Structural and Physicochemical Profiling of Morphine and Related Compounds of Therapeutic Interest
Mini-Reviews in Medicinal Chemistry Phytochemical and Biological Activities of an Anticancer Plant Medicine: Brucea javanica
Anti-Cancer Agents in Medicinal Chemistry HTLV-1 Associated Neurological Disorders
Current Topics in Medicinal Chemistry Oxidative Stress in the Pathogenesis of Alzheimer’s Disease and Cerebrovascular Disease with Therapeutic Implications
CNS & Neurological Disorders - Drug Targets Transposons for Gene Therapy!
Current Gene Therapy