genetic therapies

Question 1

gene therapy

Answer 1

the transfer of DNA or RNA into the cells of an organism to treat disease or to mark cell populations with a disease
- only somatic gene therapy in human at the moment (cant be passed on)

Question 2

criteria for genetic disorders amenable to gene therapy

Answer 2

1. life-threatening condition without effective treatment
2. cause due to single gene (identifiable)
3. regulation of gene doesn’t need to be precise to return to healthy function
4. technical problems associated with gene delivery and expression must be resolvable

• intensely regulated due to the risk of manipulating human genome

Question 3

gene delivery strategies

Answer 3

ex vivo - cells removed from patient, manipulated in lab and then returned

in vivo - requires targeting to correct cells

Question 4

the two major approaches to getting genes into cells

Answer 4

physical e.g. microinjection or lipofection

viral e.g. retroviruses or adenoviruses

Question 5

the consequence of replacing a gene

Answer 5

that gene can have more than one function (not just function its replacing) e.g. may also drive the proliferation of cells, prevent apoptosis and lead to a cancer

Question 6

challenges that remain with gene therapy

Answer 6

• immune response
• potential for misuse
• lack of permanence
• problems with viral vectors
• rarity of true single gene disorders

Question 7

mutation correction in vivo

Answer 7

required for gene therapy of gain-of-function mutations

Question 8

two approaches to mutation correction in vivo

Answer 8

repair at DNA level

repair at RNA level

Question 9

what is the potential use of target inhibition of gene expression

Answer 9

for cancers, infectious diseases, some immune disorders

Question 10

recombinant pharmaceuticals

Answer 10

idea of producing drugs by expressing biological molecules in cloning e.g. microorganism, transgenic livestock, mammalian cell lines

• need large amounts of protein generated
• reduce risks of pathogen contamination
• avoid immunogenicity of closely related, but not identical proteins

Question 11

examples of recombinant proteins in medicine

Answer 11

insulin, growth hormone, interferon etc

Question 12

genetically engineered antibodies (recombinant pharmaceutical)

Answer 12

antibodies designed specifically to recognize disease-causing proteins/antigens
- can target cancers, autoimmune disorders