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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 Β-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

Tilman Ziegler, Technische Universität München

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