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

er technology 85

Combination of gene therapy with therapeutic cloning 85

Expansion of transduced HSCs in vivo 86

The future of hematopoietic stem cell gene therapy 86

Routes of administration for gene therapy 86

Direct injection of naked DNA 87

Intramuscular injection 87

Intravenous DNA injection 87

Intraarterial delivery 87

Companies with gene delivery devices/ technologies 88

Targeted gene therapy 89

Targeted integration 89

Bacteriophage integrase system for site-specific gene delivery 90

Controlled-release delivery of DNA 90

Controlled gene therapy 91

Controlled delivery of genetic material 91

Controlled induction of gene expression 91

Drug-inducible systems for control of gene expression 92

Timed activation of gene therapy by a circuit based on signaling network 92

Small molecules for post-transcriptional regulation of gene expression 92

Engineered zinc finger DNA binding proteins for gene correction 93

Light Activated Gene Therapy 93

Spatial control of gene expression via local hyperthermia 93

Companies with regulated /targeted gene therapy 94

Gene marking 95

Germline gene therapy 95

Potential applications of human germline genome modification 95

Pros and cons of human germline genome modification 96

Role of gene transfer in antibody therapy 97

Genetically engineered vaccines 97

DNA vaccines 98

DNA inoculation technology 98

Methods for enhancing the potency of DNA vaccines 99

Advantages of DNA vaccines 99

Vaccine vectors 99

Challenges and limitations of genetically engineered vaccines 100

Vaccines based on reverse genetics 101

Technologies for gene suppression 101

Antisense oligonucleotides 101

Transcription factor decoys 102

Aptamers 103

Ribozymes 103

Peptide nucleic acid
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