Almost all human diseases are the result of inappropriate protein production or due to some structural disorder that impacts protein performance.
Biotechnology-based
drugs
Introduction
Almost
all human diseases are the result of inappropriate protein production or due
to some structural disorder that impacts protein performance. Traditional small molecule drugs are
designed to interact with protein molecules that support or cause diseases. Protein drugs seek to replace the
defec-tive protein in cases where missing or defective protein is the cause of
the disease. Enzyme replacement therapy
is a common example of protein drugs that seek to replace inherently defective
enzymes. Many severe and debilitat-ing diseases (e.g., diabetes, hemophilia,
and cystic fibrosis) and several chronic diseases (i.e., hypertension, ischemic
heart disease, asthma, Parkinson’s disease, motor neuron disease, and multiple
sclerosis) remain inadequately treated by conventional small molecular weight
and protein drugs.
Compared
to conventional small molecular weight and protein drugs, nucleic acid medicines are designed to suppress or generate
endogenous proteins by acting on or
with the transcription and translation mechanisms of formation of proteins from
the genetic code. These medicines can be administered to patients by
conventional routes, such as direct injection, inhalation, or intravenous
injection. Several different approaches are used for turning nucleic acids into
therapeutics. Among them, antisense oli-gonucleotides (ODNs), RNA interference
(RNAi) technologies, plasmid DNA and virus-based gene therapy approaches are most widely studied. Antisense ODNs and
small interfering RNAs (siRNAs) aim at inhibit-ing aberrant protein production,
whereas gene therapy aims at using the patient’s somatic cells to produce
therapeutic proteins needed for treating genetic or acquired diseases. The
promise of these nucleic acid drugs is to allow either the production of
therapeutic proteins that may be difficult to administer exogenously or the
inhibition of abnormal protein production.
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