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

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

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

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