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Gene Therapy - Technologies, Markets and Companies

fibrosis 141

Fetal intestinal gene therapy 141

Hearing disorders 141

Potential of gene therapy 142

Vectors for gene therapy of hearing disorders 142

Auditory hair cell replacement and hearing improvement by gene therapy 143

Kidney diseases 143

End-stage renal disease 143

Methods of gene delivery to the kidney 144

Gene transfer into kidney by adenoviral vectors 144

Non-viral gene transfer to the kidneys 144

Gene transfer into the glomerulus by HVJ-liposome 145

Bone marrow stem cells for renal disease 145

Mesangial cell therapy 145

Liposome-mediated gene transfer into the tubules 146

Gene transfer to tubules with cationic polymer polyethylenimine 146

Gene therapy in animal experimental models of renal disease 146

Genetic manipulations of the embryonic kidney 147

Antisense intervention in glomerulonephritis 147

Gene therapy for renal fibrosis 147

Use of genetically engineered cells for uremia due to renal failure 148

Concluding remarks 148

Liver disorders 148

Techniques of gene delivery to liver 149

Direct injection of DNA into liver 149

Local gene delivery by isolated organ perfusion 150

Liposome-mediated direct gene transfer 150

Retroviral vector for gene transfer to liver 150

Adenoviral vectors for gene transfer to liver 150

Receptor-mediated approach 151

Cell therapy for liver disorders 151

Transplantation of genetically modified hepatocytes 151

Genetically modified hematopoietic stem cells 152

Gene therapy by ex vivo transduced liver progenitor cells 152

Gene therapy of genetic diseases affecting the liver 152

Crigler-Najjar syndrome 152

Hereditary tyrosinemia type I (HT1) 153

Hereditary tyrosinemia type 3 153

Gene therapy of acquired diseases affecting the liver 153

Cirrhosis of liver 153

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