DD_2 Flashcards
(18 cards)
What are enzyme replacement therapies (ERT) used for?
To treat deficiencies in enzymes, such as Factor VIII in hemophilia and enzymes in lysosomal storage diseases.
What are examples of enzyme replacement therapies?
Factor VIII for hemophilia, glucocerebrosidase for Gaucher’s disease, and alpha-galactosidase for Fabry disease.
What is personalized medicine?
A medical approach that tailors treatments to individual genetic, phenotypic, and molecular profiles.
What are companion diagnostics?
Tests used to determine which patients will benefit from a specific drug, such as HER2 testing for Herceptin.
What are monoclonal antibodies (mAbs)?
Engineered antibodies designed to target specific proteins, used in cancer and autoimmune treatments.
What is an antibody-drug conjugate (ADC)?
A targeted therapy where an antibody delivers a cytotoxic drug directly to cancer cells.
What are the four key pharmacokinetic processes (ADME)?
Absorption, Distribution, Metabolism, and Excretion.
Why is drug metabolism important?
Metabolism detoxifies drugs and determines their half-life and activity in the body.
What is the difference between pharmacokinetics (PK) and pharmacodynamics (PD)?
PK studies how the drug moves through the body, while PD studies the drug’s effects on the body.
What is bioavailability?
The proportion of a drug that enters systemic circulation and is available to exert its effect.
How does the microbiome affect drug metabolism?
Gut bacteria can metabolize drugs, impact efficacy, and produce active metabolites.
What is a fecal microbiota transplant (FMT)?
A procedure where fecal bacteria from a healthy donor are transplanted to treat microbiome-related diseases.
What is lead optimization in drug development?
Modifying chemical compounds to improve potency, selectivity, and pharmacokinetics.
What are bioisosteres?
Molecular replacements that improve drug properties without altering biological activity.
What is Lipinski’s Rule of 5?
A guideline for predicting oral drug absorption based on molecular weight, lipophilicity, and hydrogen bonding.
What is logP?
A measure of a drug’s lipophilicity, affecting its ability to cross membranes and dissolve in fats.
What are immune checkpoint inhibitors?
Drugs that block inhibitory immune signals, such as PD-1 and CTLA-4, to enhance the immune response against cancer.
What are CAR-T cell therapies?
A personalized cancer treatment where T cells are engineered to recognize and attack tumors.
