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Gene Therapy - Technologies, Markets and Companies

mic 36

Microinjection 36

Particle bombardment 37

Ultrasound-mediated transfection 39

Molecular vibration 39

Application of pulsed magnetic field and superparamagnetic nanoparticles 39

Gene transfection using laser irradiation 40

Photochemical transfection 40

Chemical methods of gene transfer 41

Gene repair and replacement 41

Gene repair by single-stranded oligonucleotides 41

History and current status of chimeraplasty 42

mRNA gene therapy 42

Spliceosome mediated RNA trans-splicing 42

Vectors for gene therapy 43

Basic considerations 43

Use of genes as pharmaceuticals 44

The ideal vector for gene therapy 44

Viral vectors 45

Adenovirus vectors 46

Adeno-associated virus vectors 48

Alphavirus vectors 50

Baculovirus vectors 50

Foamy virus vectors 51

Herpes simplex virus vectors 51

Lentiviral vectors 53

Multicistronic retroviral vectors 54

Retroviral vectors 55

Oncogenic potential of retroviral vectors 56

Future prospects of viral vectors 57

Companies using viral vectors 57

Nonviral vectors for gene therapy 59

Anionic lipid-DNA complexes 59

Cationic lipid-DNA complexes 60

Effects of shape of DNA molecules on delivery with nonviral vectors 60

Electrostatic modifications of surface to improve gene delivery 60

Liposomes for gene therapy 61

Liposome-nucleic acid complexes 62

Liposome-HVJ complex 63

Transposons DNA vectors 63

Polycation-DNA complexes (polyplexes) 64

Plasmid DNA vector for treatment of chronic inflammatory disease 65

Polymer molecules 65

Synthetic biology and DNA vectors 65

Synthetic peptide complexes 66

Future prospects of nonviral vs viral vectors 66

Nanobiotechnology for gene therapy 66

Antisense nanoparticles for gene regulation 67

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