Biologics Flashcards
What are the four types of aggregates?
- Soluble
- Insoluble
- Reversible
- Irreversible
What are the the properties of soluble aggregates?
Small aggregation number
Cannot visually detect
Cannot be filtered out with 0.2um filter
What are the properties of insoluble aggregates?
Large aggregation number
Solution opalescent/turbid
Can generally be filtered out
How do irreversible aggregates form?
can be non-covalent or covalent though disulphide bonds
Why are dextrose diluents not a good idea?
Can react with lysines on the protein surface to form Schiff bases relatively quickly at ambient temperatures. Can occur in vivo, especially in patients with uncontrolled diabetes.
Define gene therapy
The deliberate introduction of genetic material into human somatic cells for therapautic, prophylactic or diagnostic purposes.
What is gene replacement?
Replacing faulty genes with normal genes
Corrects inherited genetic errors
Adding back or restoring a missing function
Monogenic diseases e.g. cystic firbrosis
What is the advantage of RNA viruses as vectors?
Intergrate into host DNA for transgene expression - long term expression of therapeutic gene.
What are the disadvantages of RNA virues as gene vectors?
potential to disrupt cell cycle regulation
Cancer
Ex-vivo
what are the disadvantages of DNA viruses as vectors?
Short term expression (no integration)
Repeated administration
Immune reaction
Define genetic modification
The alteration of genetic material of an organism by means that could not occur naturall through mating/recombination
What additional regulations apply to non-viral vectors?
NONE
What additional regulations apply to viral vectors?
Genetically modified organisms regulations 2014 (contained use) HSE.
GMO (deliberate release) regulations 2002, DEFRA.
Protection of human health and the environment excludes product and or patient safety
