TAILIEUCHUNG - Chapter 065. Gene Therapy in Clinical Medicine (Part 2)

Indications in gene therapy clinical trials. The chart divides clinical gene transfer studies by disease classification. A majority of trials have addressed cancer, with monogenic disorders and cardiovascular diseases the next largest categories. (Reproduced with permission from J Gene Med. New Jersey, Wiley, 2006.) Gene Transfer for Genetic Disease Gene transfer strategies for genetic disease generally involve gene addition therapy. This approach most commonly involves transfer of the missing gene to a physiologically relevant target cell. . | Chapter 065. Gene Therapy in Clinical Medicine Part 2 Figure 65-1 Q Cancer diseases 67 n 797 Q Vascular diseases n .1O6 Q Monogenic diseases 8-8 n 1O2 Q infectious diseases rj-73 2 Olher diseases rr-4D Q Gena marking r 50 O Healthy volunteers 1 6 n l9 Source Feud AS. Ktipar OL írcunvcld E. H uitr SL. -Lento DL JL Lorcilto h Prirreiplts rrt3f iTth Editioni http vir .4o Copïrtflht OTM HtQr W-HlH ComporUCi. int All rtfltltf . Indications in gene therapy clinical trials. The chart divides clinical gene transfer studies by disease classification. A majority of trials have addressed cancer with monogenic disorders and cardiovascular diseases the next largest categories. Reproduced with permission from J Gene Med. New Jersey Wiley 2006. Gene Transfer for Genetic Disease Gene transfer strategies for genetic disease generally involve gene addition therapy. This approach most commonly involves transfer of the missing gene to a physiologically relevant target cell. However other strategies are possible including supplying a gene that achieves a similar biologic effect through an alternative pathway . factor VIIa for hemophilia A supplying an antisense oligonucleotide to splice out a mutant exon if the sequence is not critical to the function of the protein as has been done with the dystrophin gene in Duchenne muscular dystrophy or downregulating a harmful response through an siRNA. Two distinct strategies are used to achieve long-term gene expression one is to transduce stem cells with an integrating vector so that all progeny cells will carry the donated gene the other is to transduce long-lived cells such as skeletal muscle or neural cells. In the case of long-lived cells integration into the target cell genome is unnecessary provided the donated DNA can be stabilized in an episomal form. Immunodeficiency Disorders Proof of Principle Early attempts to provide gene replacement into hematopoietic stem cells HSCs were stymied by

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• Gene Therapy
• Clinical Medicine
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• 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
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