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failed to inform Jesse about the earlier patients’ side effects or about the fact that
two lab monkeys were killed by the high doses of adenoviruses.
In the field of genetic based therapy, we reached the latest stage of progress a
few years ago, but this might be one of the most significant milestones so far. In
fact, having a vast knowledge of the genetic background of certain human diseases,
of stem cell research and of cell reprogramming is not enough if we cannot apply
these technologies to cure people or eliminate certain biological threats. Without
clinical application, these remain only interesting scientific achievements to be
published; however, clinical applicability is crucial for mankind.
This is the area in which gene editing provides opportunities, by correcting
the gene segments responsible for a predisposition to our diseases. Although it is
similar in many ways to gene therapy, gene editing opens new horizons. The most
well-known gene editing technique is CRISPR'’. The term CRISPR was first used
by the Spaniard Francisco Mojica in 2000 and it is an acronym that refers to the
organization of short, repeated DNA sequences found in the genomes of bacteria.
Although several journals rejected his publication as not interesting or required
more laboratory proof, finally in 2005, he and his colleagues managed to publish
his paper.“ CRISPR is based on the molecular defense system in bacteria. It was
known that the CRISPR defense system is found in many bacteria, but only much
later was it discovered that it can be used as genetic scissors.
To use a more illustrative metaphor, gene editing works a bit like Microsoft’s
“replace text” feature. After writing a long text, it is not uncommon that we change
our minds and decide to replace an expression with another that fits better. The
replace text feature can be very useful in these cases. It searches through the
text for the words to replace and it replaces them with one click. In any case gene
editing, unlike “gene manipulation” or “gene engineering,” is a friendly term that
is followed by international curiosity and hope rather than fear.
8 CRISPR is the abbreviation of the term Clustered Regularly Interspaced Short Palindromic
Repeats. The discovery of the type II prokaryotic CRISPR “immune system” has allowed for
the development for an RNA-guided genome editing tool that is simple to use.
4 Francisco J.M. Mojica — César Diez-Villasefior — Jesüs Garcia-Martinez — Elena Soria,
Intervening Sequences of Regularly Spaced Prokaryotic Repeats Derive from Foreign
Genetic Elements, Journal of Molecular Evolution 60 (2005), 174-182.
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