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Gene Therapy - Technologies, Markets and Companies
Date:11/30/2011

Biological nanoparticle technology 67

Dendrimers 67

Cochleates 67

Calcium phosphate nanoparticles as nonviral vectors 68

Gelatin nanoparticles for gene delivery 68

Lipid nanoparticles for nucleic acid delivery 69

Nanoparticles as nonviral vectors for gene therapy 69

Nanoparticles with virus-like function as gene therapy vectors 70

Nanobiolistics for nucleic acid delivery 70

Nonionic polymeric micelles for oral gene delivery 70

Silica nanoparticles as a nonviral vector for gene delivery 71

Receptor-mediated endocytosis 71

Artificial viral vectors 72

Directed evolution of AAV to create efficient gene delivery vectors 73

Bacterial ghosts as DNA delivery systems 73

Bacteria plus nanoparticles for gene delivery into cells 73

Chromosome-based vectors for gene therapy 75

Mammalian artificial chromosomes (MACs) 76

Artificial Chromosome Expression (ACE) 76

Human artificial chromosomes (HACs) 76

?C31 integrase system 77

Companies using nonviral vectors 77

Concluding remarks about vectors 78

Cell-mediated gene therapy 79

Fibroblasts 79

Skeletal muscle cells 80

Vascular smooth muscle cells 81

Keratinocytes 81

Hepatocytes 81

Lymphocytes 82

Regulating protein delivery by genetically encoded lymphocytes 82

Implantation of microencapulated genetically modified cells 82

Stem cell gene therapy 83

Therapeutic applications for hematopoietic stem cell gene transfer 83

Improving delivery of genes to stem cells 83

Lentiviral vectors for gene transfer to marrow stem cells 84

Use of mesenchymal stem cells for gene therapy 84

Microporation for transfection of MSCs 84

In utero gene therapy using stem cells 84

Gene delivery to stem cells by artificial chromosome expression 85

Linker based sperm-mediated gene transf
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