Biotechnologies for Medicinal Chemists
Pp. 109-127 (19)
Robert E. Smith
Biotechnology is the use of technology to study biology and develop new
drugs. For example, genetic engineering tools were used to solve the human genome
and may lead to the production of new types of bacteria, algae and other life forms that
will make many of the products that are now made from fossil fuels. Pieces of DNA can
be cut from chromosomes using restriction endonucleases and other forms of genome
editing. Clustered, regularly interspaced, short palindromic repeats, abbreviated as
CRISPRs are especially useful. The pieces of DNA are inserted into vectors made from
viruses or artificial chromosomes to make recombinant DNA. They are inserted into
bacteria where enough copies of them are made to enable DNA sequencing.
Biotechnologies can be used to determine the genome and proteome of cells. Also,
instead of making prescription drugs from small molecules by chemical synthesis or
purification from natural sources, some of them can be made using biotechnologies.
That is, genetic engineering can be used to insert (recombine) the gene that codes for
them or the enzymes needed to biosynthesize them into vectors. The vectors can be
inserted into bacteria, yeasts, plants and animals. Also, larger therapeutic molecules can
be made. This can include antibodies, DNA, RNA and even whole cells. Antibiotics,
vitamins, vaccines and natural products can also be made. Not only genes, but also
smaller pieces of single-stranded DNA or RNA oligonucleotides (5-40 kDa) called
aptamers can be used therapeutically . Also, catalytic ribozymes, small interfering
RNA (siRNA), RNA aptamers, short hairpin RNA (shRNA), bifunctional shRNA and
microRNA can be used to knock down specific proteins . Finally, whole cells can be
used therapeutically. This includes immune cells, glial progenitor cells, embryonic stem
cells, vaccines and even fecal transplants.
Biotechnology, monoclonal antibodies, gene therapy, siRNA, cell
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