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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]
Targeted gene knockout using CRISPR/Cas9 requires the use of a delivery system to introduce the sgRNA and Cas9 into the cell. Although a number of different delivery systems are potentially available for CRISPR, [37] [38] genome-wide loss-of-function screens are predominantly carried out using third generation lentiviral vectors.
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]
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 ...
CRISPR-Cas12a was found by searching a published database of bacterial genetic sequences for promising bits of DNA. Its identification through bioinformatics as a CRISPR system protein, its naming, and a hidden Markov model (HMM) for its detection were provided in 2012 in a release of the TIGRFAMs database of protein families.
For a given candidate gRNA, these tools report its list of potential off-targets in the genome thereby allowing the designer to evaluate its suitability prior to embarking on any experiments. Scientists have also begun exploring the mechanics of the CRISPR/Cas system and what governs how good, or active, a gRNA is at directing the Cas nuclease ...