Gene Engineering: Gene Therapy – Flashcards
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Genetic Engineering
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technology that allows one to manipulate genetic material to obtain certain desired functions
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examples of Genetic Engineering
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- Gene Therapy - cloning - transgenic organisms - genetically modified organisms
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Gene Therapy
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treatment of a disease due to a malfunction gene by replacing the gene to correct the abnormality
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2 types of gene therapy
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- germline - somatic
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Germline Gene Therapy
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alters the DNA of a gamete or fertilized ovum
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results of Germline Gene Therapy
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- all cells change - heritable - not in humans but creates transgenic organisms
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Somatic Gene Therapy
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corrects only the cells that an illness affects
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results of Somatic Gene Therapy
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- not heritable - completed in humans
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3 different approaches of Gene Therapy?
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- Ex vivo - In situ - In vivo
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Ex vivo Therapy
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cells can be altered outside the body and then infused Ex: ADA Deficieny
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In situ Therapy
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the functional gene and the vector are injected into a very localized and accessible body part ex: a single melanoma
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In vivo Therapy
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the gene and the vector are introduced directly into an internal portion of the body; most invasive ex: Canavan Disease
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3 Methods to Gene Therapy
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- physical - chemical - biological
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Physical Methods of Gene Therapy
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- electroporation - microinjection - particle bombardment
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Electroporation
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application of an electrical current encourage a cell to allow permeability of external DNA
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Microinjection
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introduction of DNA into a cell by micropipette
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Particle Bombardment
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use of heavy metals coated in DNA to breach the cell wall
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Chemical Methods of Gene Therapy
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Liposomes
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Liposomes
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- penetration of plasma membrane the DNA cannot cross alone - may not deliver a sufficient amount, causing only temporary gene expressions
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Biological Methods
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- use of a vector Ex: Virual genome
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Vector
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DNA molecule in a virus that is joined with a foreign segement of DNA so that new DNA is introduced into a cell
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Retrovirus
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- most famous: HIV - RNA - 8,000 base pairs
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Adenovirus
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- common cold - affected dividing and non dividing cells - 7,500 base pairs - double stranded DNA
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Adeno- Associated
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- 5,000 base pairs - single pairs - no human illness
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Herpes Simplex
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- afffects the nervous system - 20,000 base pairs - single stranded DNA
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Liposome
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- can fuse with any cell membrane - no max length - carries by DNA
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Naked DNA
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- "plasmid" - no max length - double stranded DNA
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Pioneer for Gene Therapy
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- Laura Cay Boren - ADA Deficiency - SCID - parents dont live past 1st birthday; she lived to her 19th
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Jesse Gelsinger
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- OTC - X linked recessive disorder - absence of an enzyme required for breakdown of A.A. in food
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Expectations and Limitations
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- high in the 90's - progress in the 80's reveled positive outcomes for genetic alterations - Human Genome Project did not provide new genes to study, but revealed complexity
