GENE THERAPY Flashcards
what is gene therapy?
replacing ‘bad’ genes with ‘good’ (replace genetic defect)
what are the 2 approaches of gene therapy?
Somatic gene therapy
Germline gene therapy
what does Somatic gene therapy influence?
the individual
what does Germline gene therapy influence?
future generations
what is a transgene?
ageneorgeneticmaterial that has been transferred naturally, or by any of a number ofgenetic engineeringtechniques from one organism to another
what is transgenesis?
introduction of a transgene has the potential to change thephenotypeof an organism
what does a transegene contain?
a segment ofDNAcontaining a gene sequence that has been isolated from one organism and is introduced into a different organism
how are transgenes delivered?
in vivo (within the body) ex vivo (outside the body)
what does gene therapy do?
replace a defective gene
what does gene therapy treat?
recessive and dominant gene disorders
how does a transgene work?
Insert the gene required, into a cell,
to alter the genetic make-up/phenotype
what are episomes?
Transgenes that do not integrate
how are tans genes delivered into the cell?
delivery vehicle or delivery vector
what factors affect which transgene delivery vehicle is used?
Efficiency of delivery Cell-cycle state of cell Cell-type Immunogenicity Size of transgene
what is the most used transgene delivery vehicle?
viruses
