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Reportlinker Adds Gene Therapy - technologies, markets and companies

of DNA vaccines 98

Vaccine vectors 98

Challenges and limitations of genetically engineered vaccines 99

Vaccines based on reverse genetics 100

Technologies for gene suppression 100

Antisense oligonucleotides 100

Transcription factor decoys 101

Aptamers 101

Ribozymes 102

Peptide nucleic acid 102

Intracellular delivery of PNAs 102

Locked nucleic acid 103

Zorro-LNA 103

Gene silencing 103

Post-transcriptional gene silencing 103

Definitions and terminology of RNAi 104

RNAi mechanisms 104

Inhibition of gene expression by antigene RNA 105

RNAi gene therapy 106

Application of molecular diagnostic methods in gene therapy 106

Use of PCR to study biodistribution of gene therapy vector 106

PCR for verification of the transcription of DNA 107

In situ PCR for direct quantification of gene transfer into cells 107

Detection of retroviruses by reverse transcriptase (RT)-PCR 107

Confirmation of viral vector integration 107

Monitoring of gene expression 108

Monitoring of gene expression by green fluorescent protein 108

Monitoring in vivo gene expression by molecular imaging 108

Advantages of gene therapy compared with protein therapy 108

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 infecti

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