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European Biopharmaceutical Review

How Gene Editing May Pave the Way for Personalised Medicine

“It is heartbreaking to see my child go through this crisis. When my son, Eric*, was five years old, he had a sickle cell crisis and the doctors told me that he might have a 50/50 chance of surviving. My heart just broke in half,” said the mother of an eight-year-old boy with sickle cell disease (SCD) (1). SCD is an inherited disorder of the red blood cells, causing them to be crescent-shaped. The clinical hallmark of SCD is pain, caused by the clumping of misshaped red blood cells to block capillaries and deprive tissues and organs of oxygen. The pain is severe and pain crisis is the most common cause of hospitalisation for patients with SCD, with many frequently spending days, weeks, or even months in hospital (2). Without treatment, SCD can lead to complications such as organ damage, pulmonary hypertension, and heart failure (3).

Currently, the only curative treatment for SCD is haematopoietic stem cell transplantation (4). Yet, despite over 90% event-free survival when a matched donor is used, treatment-related complications limit its broad acceptability and applicability. Moreover, more than 80% of patients do not have a matched donor. However, there may soon be a better cure, offering real hope for Eric and thousands of others who are born with this debilitating disease. CRISPR gene editing can correct the genetic mutation that causes SCD, and scientists such as Dr Matthew Porteus at Stanford Medicine, US, are pioneering this development into potentially new personalised gene and cell therapies (5-6).

How Far We Have Come

Since the seminal paper on CRISPR-Cas9 gene editing was published in 2012, the technology has generated substantial attention for its ability to make precise, permanent changes to DNA in living organisms (7). By altering genetic sequences, CRISPR has the potential to provide novel therapies for patients suffering from diseases caused by single gene mutations. To realise the potential of CRISPR, pharmaceutical companies have been working to develop ever better research tools for scientists. By devising an unbiased bacterial screen, two Cas enzymes were engineered with very high fidelity: HiFi Cas9 and Cas12a (CPF1) Ultra (8-9). Both nucleases have improved specificity regarding the on-target cutting of DNA sequences with minimised off-target effects, without compromising the enzymatic activity.

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Rolf Turk is a Senior Staff Scientist in the molecular genetics research group at Integrated DNA Technologies (IDT). Rolf is originally from the Netherlands, where he obtained a Master’s degree in Science at the University of Amsterdam. He continued his training as a graduate student at the Center for Human and Clinical Genetics at the Leiden University Medical Center, The Netherlands. Rolf research revolved around the molecular mechanisms behind different forms of muscular dystrophy. After obtaining his PhD, he continued his research as a post-doctoral researcher at the University of Iowa, US. Since his employment at IDT in the fall of 2015, Rolf has contributed to the development of RNA-guided endonucleases and optimisation of delivery strategies. Additionally, he focuses on off-target nomination and validation methodologies.
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Rolf Turk
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