Gianluca Petris, MRC Laboratory of Molecular Biology, UK speaks on "CRISPR-Cas9 and beyond: delivery, specificity and therapy". CRISPR-Cas nucleases can be considered the most powerful technologies for genome editing. However, several difficulties may affect the success of genome editing, in particular in vivo; these include achieving efficient RNA-guided nuclease delivery, appropriate targeted genome editing, absence of off-target activity and potential immune responses (in vivo) associated with long lasting nuclease presence in target cells.I will discuss the technologies I have developed to address these challenges including i) the engineering a lentiviral vector encoding a Self-Limiting Cas9 circuit for Enhanced Specificity (SLiCES), which increases genome editing precision by controlling Cas9 levels in a programmable fashion; ii) the development of a cellular production system to generate exosome-like vesicles (VEsiCas) carrying Cas9-guide RNA ribonucleoprotein complexes, which are efficiently delivered into target cells for gene editing applications; iii) examples of microhomology-based genome engineering with potential therapeutic applications where Cas12a can be exploited more successfully than Cas9 based on the different activity of these two “blades” for genetic surgery.

Tibor Hortobagyi - Department of Neuropathology, Institute of Pathology, University of Debrecen, Debrecen, HUNGARY speaks on “Pathology and current molecular classification of ALS/FTD”. This seminar has been recorded by ICGEB Trieste

Andrés Klein TIGEM - Telethon Institute of Genetics and Medicine, Pozzuoli (NA) - ITALY speaks on "Identification of modifier genes in lysosomal storage disorders". This seminar has been recorded by ICGEB Trieste

Steve OLIVER, Department of Biochemistry, University of Cambridge, Cambridge - UK speaks on "Making, mending, and extending the yeast metabolic model". This seminar has been recorded by ICGEB Trieste

Alberto Inga, Centre for Integrative Biology, CIBIO, University of Trento, Trento, ITALY speaks on "p53-directed translational control can shape and expand the universe of p53 target genes". This seminar has been recorded by ICGEB Trieste

Michael Breitenbach Department of Cell Biology, University of Salzburg, Salzburg, AUSTRIA speaks on "Yeast aging models".This seminar has been recorded by ICGEB Trieste

John F.X. Diffley Cancer Research UK, London Research Institute, Clare Hall Laboratories, South Mimms, UK speaks on "Investigating how DNA replication initiates in eukaryotes". This seminar has been recorded by ICGEB Trieste

Jean-Luc Souciet, Université de Strasbourg et CNRS, UMR 7156 Génétique Moléculaire, Génomique, Microbiologie, Génomique comparative des Levures Hémiascomycètes, Institut de Botanique, Strasbourg, FRANCE,speaks on "The mosaic genome of the yeast Pichia sorbitophila : genomic changes occuring after formation of a recent interspecies hybrid". This seminar has been recorded by ICGEB Trieste

Anna Tramontano, Department of Physics, Sapienza University of Rome, ITALY, speaks on "The surprising transcriptome". This seminar has been recorded by ICGEB

Beatriz Llamusí Troisi, Dpto. Genetica, Facultad de Biología Universidad de Valencia, Burjassot Valencia, SPAIN speaks on "TBPH involvement in Myotonic Dystrophy 1". This seminar has been recorded by ICGEB Trieste

Christopher Pearson, Department of Genetics, The Hospital for Sick Children, Toronto, CANADA speaks on "Repeat instability as the basis for human diseases and as a potential target for therapy". This seminar has been recorded by ICGEB Trieste