Precision Medicine, CRISPR, and Genome Engineering Moving from Association to Biology and Therapeutics
116 located in known gene clusters can now be characterized easily as CRISPR has been shown to be able to cleave large chromosom ...
117 References Antunes MS, Smith JJ, Jantz D, Medford J. Targeted DNA excision in Arabidopsis by a re- engineered homing endonu ...
118 Wang S, Zhang S, Wang W, Xiong X, Meng F, Cui X. Efficient targeted mutagenesis in potato by the CRISPR/Cas9 system. Plant ...
119 Endo M, Mikami M, Toki S. Multi-gene knockout utilizing off-target mutations of the CRISPR/Cas9 system in rice. Plant Cell ...
120 Kim S, Kim D, Cho SW, Kim J, Kim JS. Highly efficient RNA-guided genome editing in human cells via delivery of purified Ca ...
Part III The Future of CRISPR ...
© Springer International Publishing AG 2017 123 S.H. Tsang (ed.), Precision Medicine, CRISPR, and Genome Engineering, Advances i ...
124 applications of CRISPR screens in cancer, infectious diseases and inborn genetic disorders (Fig. 7.1b and Table 7.1). We hig ...
Table 7.1 CRISPR pooled screens by disease area and therapeutic implications Disease category Disease Screening phenotype Therap ...
Disease category Disease Screening phenotype Therapeutic implications Non-Hodgkin’s lymphoma Apilimod cytotoxicity [ 91 ]; induc ...
Disease category Disease Screening phenotype Therapeutic implications Sarcoma Lethal/anti-proliferation phenotype [^44 ]; myogen ...
Disease category Disease Screening phenotype Therapeutic implications Synthetic bacterial toxins Sensitivity to diphtheria and c ...
Disease category Disease Screening phenotype Therapeutic implications Dengue virus (DENV) and hepatitis C virus (HCV) infection ...
130 genomic region, and a part of a palindromic repeat that forms the secondary struc- ture for Cas9 docking [ 4 ]. Directed by ...
131 sgRNAs that are not in the original library. Validation of individual sgRNAs should also include analysis of indels (e.g. Su ...
132 a single type of domain (e.g. dCas9-VP64, which contains four tandem repeats of the VP16 domain) [ 10 ]. In addition to gene ...
133 NGG-poor regions. Since Cpf1 processes its own repeat array through its ribonucle- ase activity, it may be easier to multipl ...
134 For rare tumors, combining CRISPR screens with patient-derived in vitro models can be helpful for correlating functional gen ...
135 For loss-of-function CRISPRn screens, it can be more challenging to translate screen hits into drug targets/strategies. For ...
136 7.3.2 CRISPR Screens in Infectious Disease Pathogenic organisms such as bacteria, parasites, and viruses present a major pro ...
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