Studies in the function of genes and their mutations.
Molecular Genetics
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3.3(3 reviews)
11 episodes
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Podcast Overview
Studies in the function of genes and their mutations.
Language
🇺🇲
Publishing Since
3/10/2010
4 verified contact emails on file for Molecular Genetics
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Recent Episodes
September 7, 2021
G. Petris - CRISPR-Cas9 and beyond: delivery, specificity and therapy
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.
May 23, 2016
T. Hortobagyi - Pathology and current molecular classification of ALS/FTD
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
April 20, 2016
A. Klein - Identification of modifier genes in lysosomal storage disorders
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
11 total episodes available
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