Biotherapeutics Flashcards
Give examples of biological therapies.
antibodies
vaccine
ASO (anti sense oligos)
gene therapy
targeted drug therapy
-monoclonal antibodies
-polyclonal antibodies
bacillus Calmette-Guerin therapy
Describe nucleic acid vaccines.
nucleic acid coding for the antigen is injected
DNA plasmid: enters nucleus, translated to mRNA for expression of protein
or mRNA can be injected, more direct but less stable than DNA
What is antisense oligonucleotide?
short DNA analogue that hybridizes with the complementary mRNA in a sequence-specific manner
hybridization of ASO to the target mRNA can result in specific inhibition of gene expression resulting in reduced levels of translation of the target transcript
Describe gene therapy.
normal gene may be inserted into a non-specific location within the genome to replace a gene that is not functional
an abnormal gene could be swapped for a normal gene through homologous recombination
the abnormal gene could be repaired through selective reverse mutation
the regulation of a particular gene could be altered
Which organs are less suited for ex vivo gene therapy?
lung, brain, heart
-culture of the target cells or re-transplantation is not feasible
=do in vivo gene therapy
What is Luxturna?
first FDA-approved gene therapy
for retinal dystrophy (rare inherited eye disease) due to a mutation of the RPE65 gene
-as it progresses, patients experience gradual loss of
peripheral and central vision which can lead to blindness
supplies a normal RPE65 gene to retinal cells
-essentially adds a 3rd version of the gene, which is able to
code for a protein crucial to vision
True or false: bivalent vaccines have more total antigen than monovalent vaccines
false
same amount
What can occur once a cancer cell has been targeted by the antibody?
binding of Ab to the target antigen can inhibit signaling
drugs can be attached to the Ab and delivered specifically to the cancer cell, leading to cell death
binding of the Ab can elicit an immune response
What is an example of an ASO?
mipomersen
-used for FH
How are antibodies made?
immunizing a mouse with the desired antigen
isolate the antibodies from the mouse spleen specific to the antigen
fuse mouse antibody-forming cell with a cancer cell so that they will proliferate in culture
these fused cells are called hybridomas
test to ensure specificity and efficiency
Differentiate between polyclonal antibody and monoclonal antibody.
polyclonal:
-cheap to produce
-mixed population of antibodies
-may bind to different areas of the targeted molecule
-tolerant to small changes in protein structure
monoclonal:
-expensive to produce
-single antibody species
-will only bind single specific site
-may recognise a particular protein form
What are antibodies?
natural targeting agents
-their homing ability is combined with functional activity
-“mab”
How are monospecific antibodies made?
clones of a unique parent cell (B cell)
commonly used in research and as a therapeutic agent
What is herceptin?
targeted therapy for HER2 tumors
Which organisms are used to make most antibiotics?
bacteria or fungi
