11Pres - Human Gene Therapy •  •  •  • ...

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Human Gene Therapy Different types of gene therapy Gene addition versus gene replacement Use of viral vectors for gene addition First human gene therapy: severe combined immunodeficiency (SCID) Other attempts at somatic human gene therapy Induced Pluripotent Stem Cells Genetic modification of non-human mammals Germ line gene therapy for humans?
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In a typical gene therapy, a viral vector is used to insert a foreign gene (“transgene”) into the genome of the targeted cell.
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August Weismann (1834-1914) was a descriptive and experimental embryologist until he lost his eye sight and became a theoretical biologist. He is best known for his concept of the germ line.
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The Germ Line Concept The germ line is a continuous lineage of cells that are capable of forming eggs or sperm. Germ line cells are potentially immortal, connecting individuals of successive generations in an uninterrupted chain. The other cells – collectively called soma - will die, having served as temporary caretakers of the germ line.
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Human Gene Therapy Different types of gene therapy Gene addition versus gene replacement Use of viral vectors for gene addition First human gene therapy: severe combined immunodeficiency (SCID) Other attempts at somatic human gene therapy Induced Pluripotent Stem Cells Genetic modification of non-human mammals Germ line gene therapy for humans?
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Gene Addition vs. Gene Replacement
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Method for generating transgenic mice by microinjecting the cloned transgene into one pronucleus of a fertilized egg. From Kalthoff (2001)
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Supersized mouse (left) raised from an egg injected with a transgene for rat growth hormone. The mouse grew to twice the size of his uninjected litter mate (right).
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After Cummings (2006) Crossing Over Crossing over during meiosis produces chromosomes with new combinations of genetic alleles in gametes. Human chromosome 9 is shown. B, O: Genetic alleles for blood antigens B and O (no antigen). N: Mutant allele (dominant) of gene controlling nail-patella syndrome. n: Wild-type allele (recessive) of same gene.
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Homologous Recombination Traced with a Selectable Marker
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Embryonic stem (ES) cells are exposed to an engineered transgene, which replaces part of the resident target gene by homologous recombination. The neo R gene is used as a selectable marker to isolate transformed ES cells. In the strategy shown, a resident gene is inactivated (“knocked out”). Similarly, an additional gene can be “knocked in”, or a mutant gene can be replaced with its wild- type allele. From Kalthoff (2001) Gene replacement by homologous recombination
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Futuristic cell replacement therapy using isogenic, genetically repaired stem cells.
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