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HBIGS International Summer School on "The era of human gene therapy - prospects and challenges", 15-18 Sep 2024

Scope:
Over half a decade ago, the idea of gene therapy has been conceptualized, i.e., the treatment of a human disorder with an inherent or acquired genetic component by fixing the genetic problem at its source. Historically, gene therapy was defined as the addition or replacement of a critical gene in a given cell type using a delivery vehicle or a so-called vector, such as a recombinant virus. Nowadays, following the advent of powerful technologies for gene and genome editing such as CRISPR, a broader definition of gene therapy comprises any ex vivo or in vivo attempt to treat conditions with a genetic cause by over-expressing, adding, suppressing or editing (gene, genome or epigenome) targeted nucleic acids in a patient’s cells.
The enormous power and far-ranging potential of human gene therapy has already been illustrated in thousands of successful clinical trials and is best exemplified by the FDA and/or EMA approval of numerous gene and cell therapy drugs. A representative example that is also administered at Heidelberg University Hospital is the gene therapeutic Zolgensma, which is a recombinant Adeno-associated virus (AAV) expressing a gene (SMN1) in children at the age of 0-2 that is critically involved in spinal muscular atrophy. Most recently approved additional examples include another AAV-based drug, Elevidys, for the treatment of Duchenne muscular dystrophy, or the CRISPR-based drug Casgevy that is directed against sickle cell disease.
While these and abundant further examples showcase the tremendous promise of human gene therapy as a leading medicine of the future, concurrently increasing findings of severe toxicity or even fatality in treated patients also drastically highlight the need to return to the bench and design the next wave of gene therapeutics combining high efficacy with maximum safety. Clearly, this ambitious and clinically utmost relevant task requires a joint, international and interdisciplinary effort merging a wide array of expertise including but not limited to molecular and cell biology, virology, biochemistry, immunology, synthetic biology, engineering, bioinformatics, structural biology, optogenetics, modeling, AI and machine learning.
Towards this aim, a pivotal step is to specifically train and educate a new generation of young scientists, and to foster their interaction by establishing the required world-wide networks and infrastructure. The proposed HBIGS Summer school addresses this need by bringing together international leaders in relevant research fields and by enabling the participating PhD students to directly and actively interact with these experts as well as with each other. Importantly, the invited speakers not only comprise leaders in biological sciences, such as the organizers who have collaborated and contributed to gene therapy technologies for over a decade, but also experts in clinical translation, regulatory affairs and ethics. Moreover, the presentation of their data in talks or posters in the scientific sessions as well as discussions in the evenings and during the social events will provide the students with plenty of opportunities to shape, expand and refine their work. This promises the ability of the Summer school to instruct and motivate a next generation of key opinion leaders and to fully harness the vast potential of gene therapy.

Organizers:
Dirk Grimm, Dept. of Infectious Diseases/BioQuant
Dominik Niopek, Institute for Pharmacy and Molecular Biotechnology (IPMB)

Venue: Boppard, Germany.

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HBIGS International Winter School on "Molecular Mechanisms in Mitosis", 4-7 Feb 2019

Scope:
The HBIGS Winter School is aimed to PhD students interested in the molecular mechanisms governing mitosis. We will explore fundamental open questions in the field, including the roles of the signalling cascades in the segregation of subcellular components, the mechanisms and temporal control of molecular machines during mitosis, how asymmetry of stem cell divisions is modulated, and how misregulation of these processes lead to loss of cellular hierarchy and chromosome instability, which are hallmarks of several diseases, including cancer.
The goal of the Winter School is to bring together an interactive community with a shared interest in cell division to explore the latest developments and emerging methods in the field. We will select a small group of PhD students to foster productive interaction and discussion between scientists at different career stages. Thus, attendees are expected to participate in the discussions, and present their own research via posters or short talks.
Applicants should provide a motivation letter explaining their research interests, and why they think the Winter School can benefit them. Selected applicants will not be charged any fee for attending the school.

Scientific Coordinators:
Sergio P. Acebrón, Centre for Organismal Studies (COS)
Gislene Pereira, Centre for Organismal Studies (COS)

Venue: Heidelberg, Germany.

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HBIGS International Summer School on "Infectious Diseases", 5-7 Oct 2011

Organizers: Co-organized by HBIGS and the University of Montpellier

Venue: Sète, France.

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HBIGS International Summer School on "Regulatory RNAs in physiology, development and disease", 4-7 Sep 2011

Scope:
Over the past decade, we have seen a dramatic shift in our understanding of the role of ribonucleic acid (RNA) in the flow of genetic information in higher organisms. Based on data from small- and large-scale transcriptome analyses, we now know that a large portion of eukaryotic genomes is transcribed into RNAs that actually do not encode proteins, but rather exhibit vital and highly diverse regulatory functions. These include roles in chromosome segregation and stability, as well as fine-tuning of gene expression at the epigenetic, transcriptional and post-transcriptional levels. Unsurprisingly, this unexpected radical departure from the central dogma of molecular biology has sparked tremendous interest across all biological research areas and has already led to a flurry of exciting findings on how these different classes of regulatory RNAs are formed, how they act and to what extent they impact development, physiology and disease in eukaryotes. Concurrently, these new insights into the biology and function of non-coding RNAs have rapidly paved ways for a wealth of novel options for biotechnological exploitation, particularly for implementation of potent and safe therapeutical modalities based on deliberate RNA-mediated gene regulation in humans. Considering this unprecedented impact on biology and enormous potential for future medicine, we believe that a Summer School focused on the emerging role of regulatory RNAs in physiology, development and disease is both timely as well as of interest to a majority of the HBIGS students. In detail, we favor a broad focus spanning several relevant model organisms (plants, invertebrates, vertebrates as well as viruses), RNA classes (e.g., piRNAs, long non-coding RNAs, cellular or viral miRNAs, or ectopic shRNAs) and research areas (physiology, development, pathology as well as therapy). The panel of contacted speakers and the tentative program reflect this essential diversity.

Organizers:
Alexis Maizel, Center for Organismal Studies (COS)
Dirk Grimm, Dept. of Infectious Diseases/BioQuant

Venue: Boppard, Germany.

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HBIGS International Summer School on "Cell Cycle Regulation", 18-21 May 2009

Scope:
The concept of the Summer School is to invite internationally renowned scientists working in distinct areas of cell cycle regulation. Participants are PhD students and lecturers on selective topics of cell cycle regulation (Cyclin-Dependent Kinases (CDKs), Mitotic Kinases, Centromere Biology, Centrosome Biology, Spindle Assembly Pathways, Regulation of DNA Replication, Chromosome Segregation /APC, Spindle Assembly Checkpoint, DNA Damage Checkpoints, Mass spec of Mitotic Regulators). Each invited scientist was invited to bring along one PhD student from his/her laboratory.

Organizers:
Elmar Schiebel, Center for Molecular Biology (ZMBH)
Olver Gruss, Center for Molecular Biology (ZMBH)
Gislene Pereira, Centre for Organismal Studies (COS)

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