Author: Frank Dullweber, Böhringer Ingelheim / April 2016
Gene editing technology CRISPR-Cas
Modern biological research deals with approaches for modifying genetic information (genotype), i.e. the genetic makeup of a cell - and therefore of an organism. It is this information that determines the characteristics of that cell. In other words the change of the genotype can lead to an observable change of a cell or whole organism (phenotype).
Human diseases are based on different causes - from a clearly identified (mono) genetic disorder to influences of the environment as well as life style or a mixture of both. In pharmaceutical drug development it is becoming more and more important to understand the molecular basis of a disease in order to be able to modify this disease in an efficient manner. Using the vocabulary introduced in the first paragraph, we could say that the researcher who observes a specific phenotype would like to understand the underlying genotype to find the best medication for a patient.
A technique to study such relationships is gene editing, It can be seen as the molecular scissors to insert, delete or replace genomic information in the genome of a cell. The CRISPR-Cas technology is a relatively new, simple technology in the field of gene editing and was selected by the Science Magazine as the "2015 Breakthrough of the Year". This technique was initially described by Jennifer Doudna and Emmanuelle Charpentier. Several scientists call it the biggest biotechnological advance since the polymerase chain reaction (PCR). Its inventor Kary Mullis was honored with the Nobel Prize in Chemistry in the year 1993.
The CRISPR-Cas technology is of high interest in the scientific community. Therefore my goal was to identify the most important authors in this area by more objective indicators and guide colleagues to identify scientific conferences with speakers of high scientific expertise.