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Every year the “Highly Cited Researchers” list provided by Clarivate Analytics recognizes the most influential researchers with highly-cited papers that rank in the top 1% by citation in different scientific fields. This year 39 researchers in the list are working in Austria. Among them, CeMM PI Christoph Bock, who has been included in the cross-field category, highlighting the interdisciplinary nature of his work.

HCA|Organoid is a new EU research project that combines single-cell profiling and organoid technology to validate organoids as faithful models of human biology. The project seeks to kickstart the development of an open access “Organoid Cell Atlas”. By creating well-characterized in vitro models of human organs, this resource will enable future discovery-driven and translational research on rare genetic diseases, complex multifactorial diseases, and on cancer. Toward this goal, Europe’s leading organoid researchers as well as experts in single-cell sequencing, single-cell imaging, and computational data integration have teamed up. The HCA|Organoid project is one of six pilot actions funded by the EU Horizon 2020 Framework Program that will constitute European contributions to the “Human Cell Atlas” – an ambitious global initiative striving to advance biomedical research and therapy using single-cell technologies. The HCA|Organoid consortium comprises eight partners and will receive EUR 5 million in EU funding.

Researchers at CeMM, the Research Center for Molecular Medicine of the Austrian Academy of Sciences, have developed knowledge-primed neural networks (KPNNs), a new method that combines the power of deep learning with the interpretability of biological network models. KPNNs learn multiple layers of protein signaling and gene regulation from single-cell RNA-seq data, thereby providing a much-needed boost in our ability to convert massive single-cell atlas data into biological insights. These findings have now been published in the renowned scientific journal Genome Biology.

The human body is often seen as a “machine” that consists of specialized components: Bones and soft tissue provide structure, organs contribute physiological functions, and immune cells protect against pathogens. In reality, many cell types and organs may play more than one role. Researchers at CeMM, the Research Center for Molecular Medicine of the Austrian Academy of Sciences, have now discovered a striking example of multi-tasking cells. In a paper published in the scientific journal Nature, CeMM researchers analyzed the epigenetic and transcriptional regulation in structural cells, including epithelium, endothelium, and fibroblasts. They found widespread activity of immune genes, suggesting that structural cells are deeply involved in the body’s response to pathogens. Moreover, the study uncovered an “epigenetic potential” that pre-programs structural cells to engage in the immune response against pathogens. These findings highlight an underappreciated part of the immune system and open up an exciting area for research and future therapies.

Researchers from Stefan Kubicek's and Christoph Bock’s groups, at the CeMM Research Center for Molecular Medicine of the Austrian Academy of Sciences in Vienna, have developed a method to accurately assess the effect of specific drugs in isolated pancreatic tissue by using a refined single-cell RNA sequencing method. Their study published in Genome Biology describes the technique that they have developed to overcome the problem of contaminating RNA molecules in single-cell transcriptomics, which allowed for accurate results of dynamic drug responses in pancreatic cells. These findings will support the development of targeted drug therapies for the treatment of Type 1 diabetes in the future.

As a leading biomedical research institute, CeMM, the Research Center for Molecular Medicine of the Austrian Academy of Sciences, is committed to advancing our molecular understanding of the COVID-19 pandemic and the causative pathogen, SARS-CoV-2. Biomedical research on the virus and the disease will contribute substantially to informed policy decisions and, ultimately, the development of new treatments.

Researchers at the CeMM Research Center for Molecular Medicine of the Austrian Academy of Sciences, the Ludwig Boltzmann Institute for Rare and Undiagnosed Diseases in Vienna, the University Medical Center of Regensburg, and the National Institute of Hematology and Infectious Diseases and the Semmelweis University in Budapest have studied the response to targeted leukemia therapy in unprecedented detail, using single-cell sequencing and epigenetic analysis. The paper published in the journal Nature Communications uncovers a precise molecular program in patients with chronic lymphocytic leukemia (CLL) who start treatment with the targeted cancer drug ibrutinib. While this program was shared by all patients, the speed of its execution differed widely. These results will help develop personalized strategies for managing CLL as a chronic disease, which is particularly relevant for CLL as a disease of the elderly.

Langerhans cell histiocytosis (LCH) is a rare disease affecting primarily young children. While LCH may heal by itself without treatment in some patients, others require intensive chemotherapy and suffer from long-term consequences, or may even succumb to the disease. The reasons for these differences in disease severity are poorly understood. In a new study published in Cancer Discovery, researchers from the St. Anna Children’s Cancer Research Institute (CCRI) and the CeMM Research Center for Molecular Medicine of the Austrian Academy of Sciences revealed important insights into the cellular heterogeneity and molecular mechanisms underlying LCH.

Targeted drugs are a cornerstone of personalized medicine, yet come with important drawbacks. They restrain disease progression – but rarely eradicate the cancer. Patients therefore need to take the drugs essentially forever, exposing them to severe side effects and incurring high costs for the healthcare system. Combination therapies could potentially overcome these limitations. A study by scientists from CeMM, the Research Center for Molecular Medicine of the Austrian Academy of Sciences in collaboration with the Medical University of Vienna shows how epigenetic analysis and automated microscopy help prioritize new drug combinations for leukemia therapy. The results were published in Nature Chemical Biology. (DOI: 10.1038/s41589-018-0205-2) on 28 January 2019.

Glioblastoma is a brain cancer with devastating prognosis. A new collaborative study by scientists from CeMM, MedUni Vienna and the Austrian Brain Tumor Registry network demonstrates how epigenetic analysis of tumor samples collected in routine clinical practice could be used to better classify and treat the disease. The results were published in Nature Medicine.