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.

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.

G. Petris - CRISPR-Cas9 and beyond: delivery, specificity and therapy

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

A. Inga - p53-directed translational control can shape and expand the universe of p53 target genes

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

B. L. Troisi - TBPH involvement in Myotonic Dystrophy 1

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

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