Oldal 350 [350]
OCR
JUDIT SÁNDOR 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. + 348 *
