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

103

Intracellular delivery of PNAs 103

Locked nucleic acid 104

Zorro-LNA 104

Gene silencing 104

Post-transcriptional gene silencing 105

Definitions and terminology of RNAi 105

RNAi mechanisms 105

Inhibition of gene expression by antigene RNA 106

RNAi gene therapy 107

microRNA gene therapy 107

Application of molecular diagnostic methods in gene therapy 107

Use of PCR to study biodistribution of gene therapy vector 108

PCR for verification of the transcription of DNA 108

In situ PCR for direct quantification of gene transfer into cells 108

Detection of retroviruses by reverse transcriptase (RT)-PCR 109

Confirmation of viral vector integration 109

Monitoring of gene expression 109

Monitoring of gene expression by green fluorescent protein 109

Monitoring in vivo gene expression by molecular imaging 110

Advantages of gene therapy compared with protein therapy 110

3. Clinical Applications of Gene Therapy 111

Introduction 111

Bone and joint disorders 111

Bone fractures 111

Gene therapy for intervertebral disc degeneration 112

Spinal fusion 112

Osteogenesis imperfecta 113

Rheumatoid arthritis 113

Local or systemic treatment 114

In vivo or ex vivo gene therapy of RA 114

Clinical trials 115

Gene therapy for osteoarthritis 116

Sports injuries 117

Repair of articular cartilage defects 117

Regeneration and replacement of bone by gene therapy 118

Bacterial infections 119

Antisense approach to bacterial infections 119

Dentistry 119

Tissue engineering in dental implant defects 119

Endocrine and metabolic disorders 120

Introduction 120

Gene therapy of obesity 120

Ad viral vector-mediated transfer of leptin gene 120

AAV vector-mediated delivery of GDNF for obesity 121

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