P01
Editing cardiomyopathies
Gene therapy approaches to potentially rescue catecholaminergic polymorphic ventricular tachycardia (CPVT)
Jürgen Maximilian Groher, Ludwig-Maximilians-Universitat München
P02
Editing cardiomyopathies
Prime editing strategies for modeling and treatment of dilated cardiomyopathy
Sofia Ottonello, Klinikum rechts der Isar der TUM
P03
Editing cardiomyopathies
Prime Editing for Correcting Familial Mutation Associated with Cardiomyopathy
Vijayanand Rajendran, Klinik und Poliklinik für Innere Medizin I, Klinikum rechts der Isar, Technical University Munich, Germany.
P04
Editing cardiomyopathies
Engineering miniature CRISPR-Cas proteins for gene editing in human cells
Jessica Kornherr, Klinikum rechts der Isar der TUM
P05
In vivo gene theraputics
Establishing a targeted gene delivery platform for monogenic kidney disorders
Carlos Cordero, Max Delbrück Center for Molecular Medicine
P06
In vivo gene theraputics
Safety Support in (CAR)-T Cell Therapy Development:
- A qualitative qPCR assay as alternative approach for Replication Competent Lentivirus (RCL) monitoring -
R Nelson, BioAgilytix
P07
In vivo gene theraputics
Long-lasting antibody delivery by a myotropic Adeno-Associated Virus (AAV) Capsid Variant: high serum levels, favourable glycosylation profile and protection from SARS-CoV-2 challenge
Jannik Wagner, Institute of Clinical and Molecular Virology, University Hospital Erlangen, Friedrich-Alexander University Erlangen-Nürnberg, Erlangen, Germany
P08
In vivo gene theraputics
Development of a New Therapeutic Approach by Genome Editing of Tenascin-C Expressed in Breast Tumor and Microenvironment
H Bareke, Translational Research Group in Pediatric Oncology, Haematopoietic Transplantation and Cell Therapy, Hospital La Paz Institute for Health Research, IdiPAZ, La Paz University Hospital, Madrid, Spain
P09
In vivo gene theraputics
DART-AAVs enable specific transduction of human CD8 T cells for in vivo gene transfer
Mar Guaza Lasheras, Molecular Biotechnology and Gene Therapy, Paul-Ehrlich-Institut
P10
In vivo gene theraputics
Evaluation of a novel mRNA trans-splicing dual AAV vector strategy to treat Usher syndrome 1B
David Mittas, Department of Pharmacy – Center for Drug Research, LMU Munich, 81377 Munich, Germany
P11
In vivo gene theraputics
Enhanced precision for in vivo gene delivery through bispecific AAV vectors towards targeting of HIV reservoir cells
Fabian John, Paul-Ehrlich-Institut
P12
In vivo gene theraputics
Next-generation sequencing-guided screening of AAV9 peptide display libraries yields novel neurotropic capsids
Youssef Fouani, Boehringer-Ingelheim Pharma GmbH & Co. KG
P13
In vivo gene theraputics
Pleiotropic effects of AAV.SERCA2a treatment for heart failure in DMD pigs
Ina Luksch, Technische Universität München
P14
In vivo gene theraputics
Characterization of novel recombinant adeno-associated Virus (rAAV)-based capsid variants in the murine brain
Eda Arslantas, Department of Pharmacy – Centre for Drug Research, LMU Munich, 81377 Munich, Germany.
P15
New preclinical and clinical cancer concepts
T cells expressing HBV-specific chimeric antigen receptors harboring a Fab fragment control HBV infection in mice
Zhe Xie, Institute of virology, Technische Universität Münch / Helmholtz Munichen
P16
Novel cell theraputics
Super-resolution microscopy reveals gene-transfer strategy-induced disparity of CAR expression affecting CAR-T cell function in an antigen density dependent manner
Leon Gehrke, University Hospital Wuerzburg
P17
Novel cell theraputics
mRNA technology for the development of CAR T cells targeting fibrotic diseases
Sandro Bräuning, Fraunhofer Institute for Cell Therapy and Immunology (IZI), Leipzig
P18
Novel cell theraputics
PSMA-specific natural killer cells for prostate cancer immunotherapy
Jose Francisco Villena Ossa, Medical Center - University of Freiburg
P19
Novel cell theraputics
Selective elimination of cancer cells in tissue culture by using the collateral activity of a novel CRISPR-Cas nuclease
Torsten Fauth, Akribion Genomics
P20
Novel cell theraputics
Generation and analysis of cardiac progenitors expressing the DTPA-R PET-reporter gene for in vivo tracking of the cells in regenerative cell therapies
Sepideh Seyfi, Department of Nuclear Medicine, Klinikum rechts der Isar, School of Medicine, Technical University of Munich, 81675 Munich, Germany
P21
Novel cell theraputics
Identification and modeling of patient-specific CD19 escape variants to CAR T cell therapy
Naile Koleci, 1.Department of Medicine III: Hematology/Oncology & TranslaTUM, Klinikum rechts der Isar of the Technical University of Munich, TUM School of Medicine and Health, 81675 Munich, Germany.
P22
Novel cell theraputics
Enhancing endosomal escape of siRNA through chemical electron transfer
Fengrong Zhang, Pharmaceutical Biotechnology, Department of Pharmacy, Ludwig-Maximilians-Universität (LMU), Munich, Germany.
P23
Novel cell theraputics
Base editing for monogenic kidney disease
Nora Diederich, Max Delbrück Center for Molecular Medicine
P24
Novel cell theraputics
CAR-T cells: a new strategy to fight cytomegalovirus infections in the immunocompromised host
Sabrina Kübel, Virology, University Hospital Erlangen, Erlangen, 91054, Germany
P25
Novel cell theraputics
Gene editing of MHC class I and II enhances allogeneic regulatory T cell therapy
Dimitrios Wagner, Charité - Universitätsmedizin Berlin
P26
Novel cell theraputics
Generation of enhanced CD19-specific CAR-T cells through identification and editing of exhaustion-associated genes
Dinu Antony, Institute for Transfusion Medicine and Gene Therapy, Medical Center - University of Freiburg, 79106 Freiburg, Germany.
P27
Novel concepts in editing
Investigating the epigenetic regulation of the β-globin switch to identify novel curative opportunities for β-hemoglobinopathies via targeted epigenome editing
Melissa Whitehead, University Medical Center Freiburg
P28
Novel concepts in editing
CRISPR Prime Editing in Primary Human T Cell Subsets
Karl Petri, University Hospital Wuerzburg
P29
Novel concepts in editing
Lipid-nanoparticle co-delivery of rAAV genomes and mRNA for AAV integrase Rep78 replicates wildtype AAV genome integration as assessed by targeted sequencing
Marco Radukic, Bielefeld University, Faculty of Technology, Cellular and Molecular Biotechnolgy, Bielefeld, Germany
P30
Novel concepts in editing
CRISPR associated substrate-linked directed evolution (CaSLiDE) for evolving highly efficient and specific miniature CRISPR-Cas systems
Duran Sürün, Medical Faculty and University Hospital Carl Gustav Carus, UCC Section Medical Systems Biology, Dresden, TU Dresden 01307, Germany
P31
Novel concepts in editing
Surveying sequence specificity of designer site-specific recombinases
Jenna Hoersten, Dresden University
P32
Novel concepts in editing
A CRISPR-Cas9 double-hit strategy mitigates on-target aberrations and chromosomal translocations
Parinaz Zarghamian, Institute for Transfusion Medicine and Gene Therapy, Medical Center — University of Freiburg, Freiburg, Germany
P33
Novel concepts in editing
Lipo-xenopeptide polyplexes for CRISPR Cas9 based gene editing at ultra-low dose
Eric Weidinger, Ludwig-Maximilians-Universitat München
P34
Novel concepts in editing
Base editing of hematopoietic stem cells restores immune function in a mouse model of familial hemophagocytic lymphohistiocytosis
Lei Lei, Institute for Transfusion Medicine and Gene Therapy, Medical Center – University of Freiburg
P35
Novel concepts in editing
Intein-mediated cis- and trans-splicing enables tighter control of Sleeping Beauty transposase activity
Nicolas Sandoval Villegas, Paul-Ehrlich-Institut
P36
Other
Enhancement of in vitro AAV-mediated gene transfer for bone regeneration using a cationic poloxamer
Ahmed Musoski, Klinikum rechts der Isar der TUM
P37
Other
Universal CAST-Seq: Off-target detection of CRISPR nucleases and base editors
Masako Monika Kaufmann, Institute for Transfusion Medicine and Gene Therapy, Medical Center—University of Freiburg, Freiburg, Germany
P38
Other
Targeting choroidal endothelial cells: Development of novel AAV variants for improved transduction
Yi Lu, Department of Ophthalmology, University Hospital, LMU Munich
P39
Other
Next round in chemical evolution of xenopeptides for pDNA and mRNA transfer: The spacer effect
Melina Grau, Ludwig-Maximilians-Universitat München
P40
Other
Novel AAV variants for improved transduction of retinal microglia
Alice Reschigna, Department of Ophthalmology, University Hospital, LMU Munich, 80336 Munich, Germany
P41
Other
Next-generation transfection reagent for large scale therapeutic lentiviral vector production
L Dos Santos Silva, Polyplus Transfection
P42
Other
pPLUS® AAV-Helper, novel engineered pHelper plasmid to improve yield and quality of several AAV serotypes in suspension cell culture systems.
E Mauro, Polyplus Transfection
P43
Other
Transcriptome analysis supports specificity of multiplexed epigenome editing in primary T cells
Maria Silvia Roman Azcona, Institute for Transfusion Medicine and Gene Therapy
P44
Other
Epigenome Editing Results in Successful Transient Reactivation of Gamma Globin Gene Expression and Offers an Innovative Therapeutic Option for Î’-Hemoglobinopathies
Ali Faiq, Institute for Transfusion Medicine and Gene Therapy, Freiburg Im Breisgau, Germany
P45
Targeting delivery of RNA theraputics
siRNA CAPSULON Technology: Redefined siRNA Transfection
Irma Ochigava, CapCoBIO GmbH
P46
Targeting delivery of RNA theraputics
Evaluation of the Efficacy of Bcl-2-siRNA Loaded NK Cell-Based Exosomes in Xenograft Breast Cancer Model
H Bareke, Translational Research Group in Pediatric Oncology, Haematopoietic Transplantation and Cell Therapy, Hospital La Paz Institute for Health Research, IdiPAZ, La Paz University Hospital, Madrid, Spain
P47
Targeting delivery of RNA theraputics
Modeling delivery and efficiency of CRISPR systems in heart organoids
Sinem Sürmeli, Department of Medicine I: Cardiology, Klinikum rechts der Isar, Technical University of Munich, Germany
P48
Targeting delivery of RNA theraputics
Amphiphilic balance: fine-tuning of double pH-responsive mRNA carriers towards higher potency
Tobias Burghardt, Ludwig-Maximilians-Universitat München
P49
Targeting delivery of RNA theraputics
Optimizing miR-146a mimics: Unleashing precision in Graft-versus-Host Disease treatment
Xavier Segarra Visent, Universitätsklinikum Tübingen
P50
Targeting delivery of RNA theraputics
RNAi in macrophages mediated by efficient nanoparticles for siRNA delivery reveal complex effects on polarization markers upon knockdown of STAT3/STAT6
Achim Aigner, University of Leipzig
P51
Targeting delivery of RNA theraputics
Fine-tuning immunity – ex vivo siRNA treatment to enhance efficiency of cell-based therapies
Anastasia Kremer, Universitätsklinikum Tübingen
P52
Virotherapy
Control of bocavirus vector production by CRISPRa-VPR
Sergio D Moreno-Velasquez, University of Heidelberg, Department of Infectious Diseases, Section Viral Vector Technologies, Bioquant
P53
Virotherapy
rAAV displaying an anti-EGFR affibody transduces neuroendocrine tumor cell lines and organoids and mediates efficient enzyme prodrug tumor cell killing
Claire Rothschild-Gronau, Cellular and Molecular Biotechnology, Bielefeld University
P54
Virotherapy
RNA-switches facilitate drug-inducible expression of therapeutic proteins by oncolytic viruses
Laura Kayser, Clinical Cooperation Unit Virotherapy, German Cancer Research Center (DKFZ), Im Neuenheimer Feld 280, 69120 Heidelberg, Germany
P55
Virotherapy
Measurement of endothelial force in vivo