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European Biopharmaceutical Review

Upcycling Genomics

Gene therapies have taken centre stage in the medical field and interest is growing rapidly. For the first time, people can hope for a true cure of heritable disabilities by accessing a patient’s genome, as opposed to being treated only symptomatically, and any acquired pathologies with genetic elements are part of this development. HIV, for example, ‘hides’ in blood cells by integrating into the host’s genome, making it nearly impossible to remove from the system without genetic means. Asthma, arthritis, hypertension and many forms of cancers are in part caused by environmental factors that throw our genetic or epi-genetic makeup out of whack.

Of course, the application of genetic tools to patients is a controversial issue, and the backlash of early trials gone wrong in the 1990s severely dampened the whole, a setback that would be felt for over 10 years in the research community. With the number of clinical studies slumping and little growth for a decade, the concurrent rise of improved gene tools, paired with a much deeper understanding of the mechanisms underlying gene delivery, prepared a veritable rebirth of this field.

Following the rise of CRISPR/Cas9 technologies, the number of therapeutic strategies has become as plentiful as the range of human afflictions. This new shift towards gene therapy brings a measurable need for the optimisation of gene delivery strategies to enable practitioners to better transport their candidate constructs in a more targeted therapeutic context. While systemic applications favour the development of adeno-associated virus vector systems, highly efficient lentiviral vectors stand out as the system of choice for ex vivo therapies of hematopoietic cells (HCs) and hematopoietic stem cells (HSCs).

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Dr Franziska C Ruf is Senior Key Accounts Manager at SIRION Biotech with strong experience in molecular and cellular biology and her immunologic and hematopoietic background. Franziska worked as Project Coordinator and a Post-Doctoral Researcher in the Department of Hematology and Oncology at Klinikum rechts der Isar, Technical University Munich, Germany, where she also received her PhD in the Laboratory of Stem Cell Physiology.
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Dr Franziska C Ruf
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