Target Discovery and Validation Reviews and Protocols

Target Discovery and Validation 11
High-throughput screens of small-molecule inhibitors have generated many
target-specific drugs that could potentially be used for the treatment of patients
with cancer (see Chapters 1 and 2, Volume 2). Also, molecules such as antisense
oligonucleotides, siRNAs, and antibody inhibitors used to validate gene targets
are themselves being developed as drugs (see Chapters 7, 10, 12, Volume 2).
However, new developed drugs should be given to the right patients because the
target might be active or present in only certain patients. There is also a growing
list of genetic polymorphisms in drug metabolizing enzymes and transport
proteins that have been shown to influence drug response. Drug concentrations
in plasma can vary more than 600-fold between two individuals of the same
weight on the same drug dosage (see Chapter 17, Volume 2). Therefore, molecular
diagnostics need to be developed and integrated with drug development
and clinical trial design.
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