It’s All in the Genes

The secret behind human life has engaged scientists and thinkers for centuries. Just how do our bodies work? How do we inherit our unique attributes? And how do these factors affect our health? Every human adult consists of around 100 trillion cells, each aware of its precise role and function. How they all work together is determined by our personal “instruction manual”: genetic information in the cell’s nucleus, represented by the characteristic DNA double helix model.

In 1953, Francis Crick and James Watson visualized DNA’s unmistakable staircase structure using cardboard cutouts of the molecule’s chemical components. Their single-page paper in the scientific journal "Nature", which featured a sketch by Crick’s wife Odile, shook the scientific community to its core: Crick and Watson’s proposed twisted ladder structure “immediately suggests a copying mechanism for the genetic material.”

Researchers soon realized that the same code could be used to describe every living organism on earth, from humble bacteria to the mighty blue whale. Their combined understanding laid the groundwork for modern genetics and an industrial revolution of biotechnology driven by an innovation Crick and Watson would have dismissed as science fiction: genome editing.

In layman’s terms, genome editing allows research scientists to alter gene sequences by adding, removing, or swapping specific segments of a gene, e.g. to fix a sequence associated with a serious condition or disease.

In this field, few technologies have shown as much potential, scope, and promise as the new CRISPR process. It makes it easier, and faster, to find, delete, or replace gene segments using RNA-protein complexes to pinpoint specific DNA sequences in a cell before cutting them and allowing the cell to paste in new DNA information. Paired with an enzyme designed to “cut” the code (Cas9), this dynamic duo can detect errors in a very specific way to potentially stop diseases in their tracks.

Since their mainstream introduction in 2014, these new “gene scissors” have taken the scientific community by storm. Now used in thousands of labs around the world, they allow research scientists to observe, highlight, and modify gene sequences responsible for specific biological processes or even to screen for disease and cancer markers.

Be it lifelong autoimmune disorders, chronic conditions, or cancers – by giving us the potential power to tackle serious diseases like leukemia, cystic fibrosis, or multiple sclerosis, CRISPR defines an exciting new path for medical research and treatment. And Merck KGaA, Darmstadt, Germany is set to lead the way, supplying tools used from discovery right through to CRISPR licenses for therapeutic use.

An ongoing commitment to research and development in the field of genome editing is already paying off, with many valuable discoveries and insights. Fluorescent markers, for example, can make the invisible visible: scientists can use them to tag muscle proteins in cardiac cells to watch them work or follow messenger proteins for inflammation genes – all without disrupting the living cells under observation. Meanwhile, Merck KGaA, Darmstadt, Germany’s new Centinel technology prevents contamination in production, increasing patient safety while saving biopharmaceutical manufacturers millions of dollars.

Rapid innovations in genome editing and CRISPR have resulted in groundbreaking advancements in biological research and medicine. At the same time, the growing potential of using gene editing technologies in the human germline has led to scientific, legal, and societal concerns. As both user and supplier of gene editing technology, Merck KGaA, Darmstadt, Germany carries out and supports research with genome editing under careful consideration of ethical and legal standards. Under the guidance of our Bioethics Advisory Panel, Merck KGaA, Darmstadt, Germany has defined a clear operational approach, taking into account scientific and societal issues without blocking any promising therapeutic approaches for use in research and application. More on our approach can be found here.