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

tes mellitus 121

Methods of gene therapy of diabetes mellitus 122

Viral vector-mediated gene transfer in diabetes 122

Gene delivery with ultrasonic microbubble destruction technology 123

Genetically engineered cells for diabetes mellitus 123

Genetically altered liver cells 124

Genetically modified stem cells 124

Genetically engineered dendritic cells 124

Insertion of gene encoding for IL-4 124

Leptin gene therapy 125

Concluding remarks about cell and gene therapy of diabetes 125

Gene therapy of growth-hormone deficiency 126

Gastrointestinal disorders 126

Introduction 126

Methods of gene transfer to the gastrointestinal tract 127

Direct delivery of genes 127

Naked plasmid DNA into the submucosa 127

Viral vectors 127

Receptor-mediated endocytosis 128

Indications for gastrointestinal gene therapy 128

Gene therapy for inflammatory disorders of the bowel 128

Gene transfer to the salivary glands 129

Potential clinical applications of salivary gene therapy 130

Hematology 130

Hemophilias 130

Gene therapy of hemophilia 131

Hemophilia A 131

Hemophilia B 132

Concluding remarks about gene therapy of hemophilias 133

Hemoglobinopathies 133

Stem cell-based gene therapy and RNAi for sickle cell disease 133

Gene therapy for ?-thalassemia 134

Gene therapy of Fanconi's anemia 135

Acquired hematopoietic disorders 136

Chronic acquired anemias 136

Neutropenia 137

Thrombocytopenia 138

Concluding remarks about gene therapy of hemoglobinopathies 138

Companies involved in gene thery of hematological disorders 139

In utero/fetal gene therapy 139

Fetal gene transfer techniques 139

Animal models of fetal gene therapy 140

Potential applications of fetal gene therapy 140

Fetal gene therapy for cystic
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