Gene_therapy

Gene_therapy - Gene along the Way to Gene Therapy Steps...

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7. Systemic Response X X Gene Gene ? 1. Delivery 2. Binding 3. Internalization 4. Escape 5. Nuclear Transport 6. Expression Gene Therapy
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What are the desirable properties of a gene therapy vector?
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AAV2 as a gene therapy vector Adeno-associated virus Dependovirus, family Parvoviridae Icosahedral capsid (22 nm), non-enveloped ssDNA genome, 4.7 kb (+ or - polarity) Require helper virus (adenovirus or herpes) for Latent infection in absence of helper Non-pathogenic Integration or stable episome for long-term infection Broad range of infectivity of cell/tissue types
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AAV life cycle
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Does AAV2 kill cancer cells? AAV2 infection counteracts deregulation of cell cycle controls in HPV-infected and cancerous cell lines (Alam et al. 2006, J Virol 80:4927-4939).
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Course of AAV infection 1. Attachment to receptor 2. Endocytosis 3. Endosome 4. Escape 5. Uncoating 6. Entry into nucleus 7. Synthesis of 2 nd strand 8. Integration or episome Santos-Coura and Nady, 2008, Genetics and Molecular Biology 31:1-11
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Gene_therapy - Gene along the Way to Gene Therapy Steps...

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