petercelec@gmail - uniba.skgmail.com 2016 institute of molecular biomedicine peter celec gene...
TRANSCRIPT
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2016 Institute of Molecular Biomedicine
www.imbm.sk
Peter Celec
Gene Therapy
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Definition
Principles
Classification
Vectors
Clinics
Issues
Gene therapy
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Definition
All approaches for the treatment of diseases that use
nucleic acids and genetic engineering
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Principles of gene therapy
Gene substitution
Killing of target cells
Mutation repair
Targeted inhibition of gene expression
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Principles of gene therapy
Gene substitution
Normal phenotypeDiseased cells
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Principles of gene therapy
Killing of target cells
Target cells
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Principles of gene therapy
Killing of target cells
Prodrug
Target cells
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Principles of gene therapy
Killing of target cells by activation of the immune system
Foreign antigen
Cytokine gene
Target cells
Target cells
Immune cells
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Principles of gene therapy
Mutation repair
Diseased cells Normal phenotype
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Principles of gene therapy
Targeted inhibition of gene expression
Diseased cellsInhibition
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Nobel Prize in Physiology or Medicine 2006
Andrew Z. Fire Craig C. Mello
1/2 of the prize 1/2 of the prize
USA USA
Stanford University School of
Medicine
Stanford, CA, USA
University of Massachusetts
Medical School
Worcester, MA, USA
b. 1959 b. 1960
"for their discovery of RNA
interference - gene
silencing by double-
stranded RNA"
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Principles of gene therapy
RNA interference
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Classification of gene therapy
According to the used nucleic acid
– DNA based
– RNA based
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Classification of gene therapy
According to the target cell type
– Somatic
– Germline
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Classification of gene therapy
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Classification of gene therapy
According to the approach
– In vivo
– Ex vivo
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Classification of gene therapy
According to the approach
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Vectors
Viral
Non-viral
Bacterial
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Vectors
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Viral vectors
Retrovirus
Adenovirus
Adeno-associated virus
Herpesvirus
Alfavirus
...
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Non-viral vectors
Naked or coated DNA
Various physico-chemical
transport systems
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Non-viral vectors
Plasmids PCR products
Injection of naked DNA
Elektroporation
Gene gun
Lipoplexes
Polyplexes
Oligodeoxynucleotides
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Bacterial vectors
Bactofection
– Listeria
– Escherichia
– Salmonella
Alternative gene therapy
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Bactofection
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Alternative gene therapy
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Transkingdom RNAi
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Transkingdom RNAi
lysteriolysin
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Bacterial vectors and cancer
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Targeting organelles
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Clinics
1989/1990 – first clinical study
Severe combined immunodeficiency (SCID)
Retroviral vector
Integrating into the chromosome
Ex vivo
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Clinics
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Clinics
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Clinics
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Antioxidative gene therapy
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Antioxidative gene therapy
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DNA vaccination
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DNA vaccination - bacteria
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Bactofection
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Bactofection
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Alternative gene therapy
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Alternative gene therapy
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CRISPR/Cas9
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CRISPR/Cas9
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CRISPR/Cas9
http://zlab.mit.edu/
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Gene therapy issues
Ethics
Gene transfer
– Safety
– Efficiency
– Specificity
– Regulation of expression
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Topics
Sleeping Beauty transposon system
AAV induced immune response
Zinc finger endonucleases
SCID GT trial
Ethics – doping, embryonal GT
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miRNA for targeting
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iPS cells
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GT restores vision
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GT restores hearing
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€ 1.1 million
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