This session will describe two technologies to manipulate genes such as CFTR for potential therapeutic purposes as well as for more fundamental mechanistic studies about cell biology. One of these technologies is genome editing by the CRISPR/Cas9 system, in which targeted alterations are made in the genome of particular cells. Various applications of the technology, from mutation correction in primary cells to creation of mutations to study a mutation's effects on cellular processes or as a therapeutic tool will be discussed. RNA editing, by which one can correct mutations in messenger RNA without modifying the genomic DNA from which it was transcribed will also be discussed.
Explain the principles behind genome editing by the CRISPR/Cas9 System.
Describe the uses of genome editing in primary cells for therapeutic strategies & in cell lines as tools to probe mechanistic questions.
Discuss the process of targeted RNA editing & its potential as a therapeutic strategy for CF.