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22 Cards in this Set
- Front
- Back
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What type of cells does a retrovirus commonly infect?
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dividing cells
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It is the vector of choice when stable expression of corrective gene is necessary
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retrovirus
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What is a retrovirus made from?
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RNA virus
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What type of cells does an adenovirus commonly infect?
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variety of human cell types including non dividing cells
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What is an adenovirus made from?
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human virus
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Adv. of adenovirus
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they are large = capable of accepting large gene inserts
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Disadv. of adenovirus
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1) inserted DNA does not integrate w/ chromosomal DNA and remain as an episomal vector
2) transient |
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What virus vector is used in CFTR?
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Adenovirus
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They are a group of single stranded DNA viruss requiring a helper virus such as adenovirus or herpes simplex virus to infect
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adeno-associated virus
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Adv. of adeno-associated virus
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1) long term gene expression possibility because they integrate into chromosomal DNA
2) safe because 96% of the parental AAV genome has been deleted and the viral gene construct mainly contain the gene of interest. |
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They are non-integrating, can carry large inserts, very infectious to epithelial and brain cells, natural target is host
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herpex simplex virus
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Major applications in disease mgt of herpex simplex virus?
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Neurological diseases (ex. Parkinsons, CNS tumors)
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Disadv. of herpex simplex virus
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1) non integrating so long term expression of transferred genes is not possible
2) like adenovirus, can induce an immune response |
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In liposomes, the DNA to be transferred is packaged - in vitro/in vivo/ex vivo? and used directly by transferring the DNA to a suitable target tissue in vitro/in vivo/ex vivo?
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in vitro, in vivo
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lipid coating in liposomes allows the DNA to survive
in vitro/in vivo/ex vivo |
in vivo
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Cationic liposomes have become popular vehicles for gene transfer in in vitro/in vivo/ex vivo gene therapy?
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in vivo
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What is the target limit size of DNA to be transferred to a liposome?
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no limit
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Disadv of liposome
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1) low efficiency
2) introduced DNA is not designed to integrate into DNA chromosome 3) expression of inserted gene is transient |
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It is caused by a single gene
mutation that leads to a deficiency of adenosine deaminase (ADA) |
SCIDS (Severe Combined Immune Deficiency Syndrome)- T cells nonfunctional due to reduced ADA
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What vector system is used in SCIDS for ADA gene delivery (ex vivo)
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retrovirus (for dividing cells) ADA gene was detected up to 2 years after gene admin was stopped
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What test - in vitro/in vivo/ex vivo was used that revealed populations of immunologically competent cells carrying introduced ADA gene after retrovirus therapy?
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in vitro
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ADA-PEGylate to?
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increase t1/2
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