Antibodies & Gene Therapies - focus: RNA/DNA therapeutic delivery Flashcards
Focus: delivery of genetic therapeutics (11 cards)
What is an idiotype?
Antigen binding site
Where is the idiotype located?
On the ends of the variable region
What is the effector region?
Cellular binding site
What is serum sickness?
Sickness caused by the immune system developing antibodies against foreign antibodies used therapeutically
What does it mean for an antibody to be humanized?
The variable region is part-animal, part-human
What is an epitope?
Part of the antigen the antibody is specific to
How can cancer cells develop resistance to antibodies? (related to epitopes)
Mutate the antigen’s epitope
How do antibody-drug conjugates treat cells specifically?
The antibodies bind to their antigen leading to endocytosis. Once in the endosome, the environment leads to cleavage of the drug’s linker, leading to release
Why are lentiviruses used to introduce RNA to cells?
They can penetrate the nucleus and dividing cells
How can retroviral vectors be used therapeutically?
Pathogenic sequences are removed and replaced with genes/treatments of interest, cells are treated with the virus, RNA is converted to DNA and integrated into the genome, and the cells express the new gene
How is CRISPR/Cas9 given to cells?
RNA encoding for sgRNA, Cas9 proteins, new KI gene is added into lentiviral vectors and transduced
