TAILIEUCHUNG - Chapter 065. Gene Therapy in Clinical Medicine (Part 3)

Long-Term Expression in Genetic Disease: In Vivo Gene Transfer with Recombinant Adeno-Associated Viral (AAV) Vectors Recombinant AAV vectors have emerged as attractive gene delivery vehicles for genetic disease. Engineered from a small replication-defective DNA virus, they are devoid of viral coding sequences and trigger very little immune response in experimental animals. They are capable of transducing nondividing target cells, and the donated DNA is stabilized primarily in an episomal form, thus minimizing risks associated with insertional mutagenesis. Because the vector has a tropism for certain long-lived cell types, such as skeletal muscle, the central nervous system (CNS), and hepatocytes, long-term. | Chapter 065. Gene Therapy in Clinical Medicine Part 3 Long-Term Expression in Genetic Disease In Vivo Gene Transfer with Recombinant Adeno-Associated Viral AAV Vectors Recombinant AAV vectors have emerged as attractive gene delivery vehicles for genetic disease. Engineered from a small replication-defective DNA virus they are devoid of viral coding sequences and trigger very little immune response in experimental animals. They are capable of transducing nondividing target cells and the donated DNA is stabilized primarily in an episomal form thus minimizing risks associated with insertional mutagenesis. Because the vector has a tropism for certain long-lived cell types such as skeletal muscle the central nervous system CNS and hepatocytes long-term expression can be achieved even in the absence of integration. Clinical trials using recombinant AAV vectors are now ongoing for muscular dystrophies ai-antitrypsin deficiency lipoprotein lipase deficiency hemophilia B and a form of congenital blindness called Leber s congenital amaurosis. Hemophilia is often considered a promising disease model for gene transfer as the gene product does not require precise regulation of expression and biologically active clotting factors can be synthesized in a variety of tissue types permitting latitude in choice of target tissue. Moreover raising circulating factor levels from 1 levels seen in those severely affected into the range of 5 greatly improves the phenotype of the disease. Preclinical studies with recombinant AAV vectors infused into skeletal muscle or liver have resulted in long-term 5 years expression of factor VIII or factor IX in the hemophilic dog model. Administration to skeletal muscle of an AAV vector expressing factor IX in patients with hemophilia was safe and resulted in long-term expression as measured by muscle biopsy but circulating levels never rose 1 for sustained periods and a large number of IM injections 80-100 was required to access a large muscle mass. .

• Y học thường thức
• Gene Therapy
• Clinical Medicine
• bệnh học và điều trị
• bài giảng bệnh học
• tài liệu y khoa
• Harrisons Internal Medicine
• Transgene Expression
• Uptake Mechanism
• Gene Delivery
• Polylipid Nanoparticle
• Mesenchymal stem cells
• Baculoviral vector
• Cancer gene therapy
• Green fluorescence protein
• BMC Cancer
• Genetically engineered mesenchymal stromal cells
• Cell therapy
• Suicide gene therapy
• Clinical trial
• Clostridium perfringens enterotoxin
• Colon cancer
• Suicide gene
• Bladder cancer
• Radical cystectomy
• Perioperative chemotherapy
• Perioperative targeted therapy
• Immune therapy
• trình bày báo cáo
• tài liệu báo cáo khoa học
• nghiên cứu y học
• báo cáo y học
• kiến thức y học
• Novel
• Gene
• Therapy
• Approaches
• Ming Wei
• David Good
• Dermal fibroblast
• Nucleofection method
• Green fluorescent protein
• Clinical gene therapy
• Fetal Calf Serum
• Adeno associated viral vector
• Chicken beta actin promoter
• Heparan sulfate proteoglycans
• Red fluorescent protein
• Gene transfer techniques
• Hepatocellular carcinoma
• Combined therapy
• VX2 tumor
• Tumor gene therapy
• Thymidine kinase
• Directly targeting
• Bifidobacterial recombinant thymidine kinase
• Targeting system
• Antitumor mechanisms
• Artificial microRNA
• DNA polymerase
• Caspase 3
• Human sodium iodide symporter
• Radioiodine gene therapy
• Adenine nucleotide translocase 2
• Short hairpin RNA
• Radiation induced immune response
• BMC Bioinformatics
• Patient derived xenograft model
• Gene expression
• Predictive cancer biomarker
• Targeted therapy
• Drug response prediction
• Viral insertion
• Viral integration
• Sequence mapping
• Genome viewer
• BMC Molecular and Cell Biology
• Retinal neovascularization
• Endothelial progenitor cells
• Endostatin transfected EPCs
• BMC Musculoskeletal Disorders
• Endothelial mesenchymal transition
• Mouse aortic endothelial cells
• Endothelial cell targeting property
• Colorectal cancer
• MEK inhibitor
• Cancer stem cell
• Gene expression signature
• báo cáo khoa học
• báo cáo hóa học
• công trình nghiên cứu về hóa học
• tài liệu về hóa học
• cách trình bày báo cáo