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

C. Pearson - Repeat instability as the basis for human diseases and as a potential target for therapy

Studies in the function of genes and their mutations.

Medicine

Science & Medicine

Science

Health & Fitness

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

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

T. Hortobagyi - Pathology and current molecular classification of ALS/FTD

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

A. Klein - Identification of modifier genes in lysosomal storage disorders

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

S. Oliver - Making, mending, and extending the yeast metabolic model

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

C. Pearson - Repeat instability as the basis for human diseases and as a potential target for therapy

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