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7 Apr 2011
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LECTURE 24 โ€“ April 7th, 2011
GENE THERAPY
๎€Viable method to treat individuals with specific disease or disorder
๎€Sequencing of human genome โ€“ rschers thought will accelerate gene therapy
development
oNew information that came about in subsequent years and decades really
pinpointed or emphasized hat genes are not separate entities
oInteractions involved in cell and tissues are complex
oComplexity of interactions affect gene therapy methods
oProcedure can be harmful to patient
oCaused rschers to reevaluate gene therapy as method to treat disease
Somatic gene therapy
๎€Better than just treating symptoms
๎€Germ line gene therapy
oAlters DNA of gamete or of the embryo โ€“ so all cells contain alteration
๎€Can be passed down to offspring
๎€Not yet possible in humans
oCan create transgenic animals, like flies
๎€Other ytpe is somatic gene therapy
oNonheritable
oCorrects disease phenotype in somatic cells affected by disorder or disease
oTwo types
๎€In vivo
๎€Ex vivo
Differ in terms of invasiveness
๎€In vivo โ€“ functional gene and DNA vector being injected into say, the brain
oOr into artery to lead to liver
oVERY invasive
๎€Ex vivo โ€“ less invasive
oCells altered โ€“ removed, therapeutic genes added, reinfused and returned
into patient intravenously
oMost commonly performed because less invasive
First human trials occurred two decades ago
๎€Two girls
๎€SCID caused by mutation in ADA gene
๎€When have lack of ADA functional gene expression โ€“ unable to mount proper
immune response
๎€At cellular level, effect
oADA deficiency causes deoxy ATP to build up in cells
oLack of ADA blocks pathway important
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