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CRISPR-Cas9 genome editing techniques have many potential applications. The use of the CRISPR-Cas9-gRNA complex for genome editing [10] was the AAAS's choice for Breakthrough of the Year in 2015. [11] Many bioethical concerns have been raised about the prospect of using CRISPR for germline editing, especially in human embryos. [12]
In the specific context of genome-wide CRISPR screens, producing and transducing the lentiviral particles is relatively laborious and time-consuming, taking about two weeks in total. [44] Additionally, because the DNA integrates into the host genome, lentiviral delivery leads to long-term expression of Cas9, potentially leading to off-target ...
The formation of the IGI was initially announced in March 2014 as the "Innovative Genomics Initiative", a partnership between UC Berkeley and UCSF researchers and biopharmaceutical industry partners with the aim of enhancing and genome-editing technology and applying it to drug development and global health, with funding support from the Li Ka ...
In March 2017 Intellia and Regeneron, partners in co-developing a CRISPR-based treatment for transthyretin amyloidosis, presented data from a gene editing experiment in mice. [16] [17] By that time, University of California had lost a challenge to Broad's CRISPR patents, putting Intellia at a disadvantage relative to Editas. [16]
Designing an appropriate gRNA is an important element of genome editing with the CRISPR/Cas system. A gRNA can and at times does have unintended interactions ("off-targets") with other locations of the genome of interest.
A paper demonstrated that genome wide activation could be used to determine which proteins are involved in mediated resistance to a specific drug. [7] Another paper used genome wide activation of long, noncoding RNAs and observed that increasing the expression of certain long noncoding RNAs conferred resistance to the drug vemurafenib. [ 16 ]
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