17:00-19:00

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Parallel 2a

Gene editing: Technology development

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Chairs: Julian Grunewald, TUM, Munich / Alessia Cavazza, UCL London

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INV08: Miguel Esteban, Guangzhou Institutes of Biomedicine and Health, Chinese Academy of Science

Identification of mammalian progenitor cells at tissue and body scale

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INV09: Angelo Lombardo, SR Tiget, Milan

Exploiting targeted epigenome editing for therapeutic applications

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OR10: Rajeev Rai, UCL London

CRISPR/Cas9 based disease modelling and functional correction of Interleukin 7 Receptor Severe Combined Immunodeficiency

 

OR11: Eleonora Pedrazzoli, University of Trento

CoCas9: a compact nuclease from the human microbiome for efficient and precise editing

 

OR12: Lucrezia della Volpe, SR Tiget, Milan

Inhibition of p38-MAPK counteracts DNA damage induced by ex vivo expansion of hematopoietic stem and progenitor cells for efficient genetic engineering

 

OR13: Julia Klermund, University of Freiburg

An in vivo CAST-Seq workflow identifies and quantifies off-target activity as well as chromosomal translocations in organs edited in vivo with CRISPR-Cas nucleases or nickases

 

P425: Maria Silvia Roman Azcona, University of Freiburg

Contemporary modulation of two major immune checkpoints expression in CAR T cells via hit-and-run epigenome editing

 

P371: Sébastien Levesque, Université Laval Quebec

MTOR as a selectable genomic harbor for CRISPR-engineered CAR-T cell therapy

17:00-19:00

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Parallel 2c

Lentiviral & integrative vectors

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Chairs: Gloria Gonzalez-Aseguinolaza, CIMA, Pamplona / Ivana Barbaric, University of Sheffield

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INV06: Eric Kremer, University of Montpellier

Why are some adenovirus vectors poorly immunogenic?

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INV07: HIldegund Ertl, The Wistar Institute, Philadelphia

Immunotoxicity of high dose AAV vector gene transfer: can we develop protocols that allow for repeated dosing of lower and less toxic doses?

​

OR06: Greg Gojanovich, AskBio

Using a systems biology approach to unravel the Immunogenicity of AAV8 empty capsids in healthy volunteers.

 

OR07: Kei Kishimoto, Selecta BioSciences

ImmTOR tolerogenic nanoparticles combined with Treg-selective IL-2 mutein induces massive expansion of antigen-specific regulatory T cells and synergistically inhibit formation of anti-AAV antibodies to high vector doses

 

OR08: Wolfgang Miesbach, University Hospital Frankfurt

Sustained factor IX activity levels and bleeding protection following etranacogene dezaparvovec administration in people with haemophilia B without and with adeno-associated virus serotype 5 neutralizing antibodies

 

OR09: Helena Costa Verdera, SR Tiget, Milan

Investigating the role of innate immune signaling in cell toxicity upon AAV-mediated gene transfer in hiPSC-derived CNS models

 

P423: Ana Cavallo, AstraZeneca

FaDe-Cas9: a fast degrading SpCas9 with reduced cellular immunity

17:00-19:00

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Parallel 2b

Lentiviral & integrative vectors

 

Chairs: Claire Booth, UCL London / Anne Galy, Genethon, Evry

INV04: Frank Staal, Leiden University Medical Center

Developing stem cell based gene therapy for RAG1 and RAG2 deficient SCID.

 

INV05: Rosa Bacchetta, Stanford School of Medicine

Current Advances in the Gene therapy for IPEX syndrome 

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OR01: Francesca Ferrua, SR Tiget, Milan

Safety and clinical benefit of lentiviral haematopoietic stem and progenitor cell gene therapy in 23 patients with Wiskott-Aldrich Syndrome with up to 10.5 years of follow-up

 

OR02: Sameer Bahal, UCL, London

Experience of genome editing patient haematopoietic stem cells to treat X-linked Agammaglobulinemia

 

OR03: Stuart Ellison, University of Manchester

Enhanced transduction and immunophenotyping demonstrates preclinical safety and efficacy of haematopoietic stem cell gene therapy for Mucopolysaccharidosis II (MPSII) using an IDS.ApoEII brain targeted therapy

 

OR04: Valentina Poletti, University of Padova

Preclinical development of an ex vivo gene therapy for Mucopolysaccharidosis type II

 

OR05: Ludovica Santi, SR Tiget, Milan

Skeletal damage and cross-correction in MPSIH HSPC-gene therapy

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17:00-19:00

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Parallel 2c

Lentiviral & integrative vectors

​

Chairs: Gloria Gonzalez-Aseguinolaza, CIMA, Pamplona / Ivana Barbaric, University of Sheffield

​

INV06: Eric Kremer, University of Montpellier

Why are some adenovirus vectors poorly immunogenic?

​

INV07: HIldegund Ertl, The Wistar Institute, Philadelphia

Immunotoxicity of high dose AAV vector gene transfer: can we develop protocols that allow for repeated dosing of lower and less toxic doses?

​

OR06: Greg Gojanovich, AskBio

Using a systems biology approach to unravel the Immunogenicity of AAV8 empty capsids in healthy volunteers.

 

OR07: Kei Kishimoto, Selecta BioSciences

ImmTOR tolerogenic nanoparticles combined with Treg-selective IL-2 mutein induces massive expansion of antigen-specific regulatory T cells and synergistically inhibit formation of anti-AAV antibodies to high vector doses

 

OR08: Wolfgang Miesbach, University Hospital Frankfurt

Sustained factor IX activity levels and bleeding protection following etranacogene dezaparvovec administration in people with haemophilia B without and with adeno-associated virus serotype 5 neutralizing antibodies

 

OR09: Helena Costa Verdera, SR Tiget, Milan

Investigating the role of innate immune signaling in cell toxicity upon AAV-mediated gene transfer in hiPSC-derived CNS models

 

P423: Ana Cavallo, AstraZeneca

FaDe-Cas9: a fast degrading SpCas9 with reduced cellular immunity