gene therapy Flashcards
what is somatic cell therapy
gene therapy by inserting functional alleles into body cells
what is germ line therapy
gene therapy by inserting functional alleles into gametes or zygotes
what is the function of gene therapy
insert functional allele of a gene into cells which only contain mutant or non functioning versions of that allele
what happens when the inserted allele by gene therapy is expressed
the individual who receive the therapeutic gene will produce a functioning protein and no longer have the symptoms of a genetic disorder
what genetic disorder is helped by somatic cell gene therapy
cystic fibrosis
describe cystic fibrosis
sticky mucus build up in lungs.
inherited two faulty recessive alleles of a particular gene
how is inheritance of genes different between somatic and germ line gene therapy
somatic cant be inherited
germline is inherited by offspring
is somatic gene therapy long term or short term
short
is germline therapy short or long term
long
can somatic gene therapy affect alot of cells or just certain cells
only certain cells get the allele
can germline therapy affect alot of cells or just specific cells
alot of cells get the allele,
what is the difference between the cells affected by germline and somatic
somatic- body cells
germline- zygotes, gametes
can germline therapy affect specific tissue
no, somatic does
is gene therapy a form of genetic engineering
yes
how are liposomes used in somatic gene therapy
dna lengths/ alleles are packaged in small spheres of lipid bilayer to a small sphere with can carry dna - this is a liposome
how are liposomes transported into the body
liposomes places in inhaler, sprayed into nose, pass through plasma membrane of respiratory cells.
they can also pass through nuclear envelope and insert functioning allele into the host genome, the host will express the functioning protein
what is another common vector using in gene therapy
virus
how does a virus make a good vector
they directly hyjack host dna, reproduce rapidly, until cell bursts.
why do viruses need to be genetically modified
modify so it encases functioning allele
modified so it doesnt cause disease
how do viruses spread normally in humans
hyjack host cells and genetic material, reproduce rapidly until cell bursts and they spread and infect other cells
what are the issues with using viruses as a vector in gene delivery
- even though not virulent, they may still provoke immune response
- patient may become immune to virus
-virus may insert the allele into patient genome in location which disrupts the expression of other genes
sometimes a new gene can be inserted into a chromosome can affect the functioning of other genes in the chromosome…
why
nearby gene can be switched on or off
new gene can disable to functioning gene if inserted into it
can offspring inherit the alleles from an altered genome affected by germ line therapy
yes
what are the severe dangers of gene disrupting expressions of other genes
increase cancer risk
