Precision Medicine, CRISPR, and Genome Engineering Moving from Association to Biology and Therapeutics
137 proteins function in a common pathway to sulfate CCR5 so that it can be recognized by HIV. Loss of either of these proteins ...
138 Mutagenesis of the enhancer phenocopies knock-out of BCL11A and results in re-activation of HbF. For therapeutic gene editin ...
139 7.4 Conclusion and Future Perspectives In order to develop new therapies for complex diseases, a key challenge is to identif ...
140 Wang T, Wei JJ, Sabatini DM, Lander ES. Genetic screens in human cells using the CRISPR- Cas9 system. Science. 2014;343:80– ...
141 Feng J, Fan G. The role of DNA methylation in the central nervous system and neuropsychi- atric disorders. Int Rev Neurobio ...
142 Hong AL, Tseng YY, Cowley GS, Jonas O, Cheah JH, Kynnap BD, Doshi MB, Oh C, Meyer SC, Church AJ, Gill S, Bielski CM, Keskul ...
143 Petersen LR, Jamieson DJ, Powers AM, Honein MA. Zika virus. N Engl J Med. 2016;374:1552–63. Simmonds P. Genetic diversity ...
14 4 Xie S, Duan J, Li B, Zhou P, Hon GC. Multiplexed engineering and analysis of combinatorial enhancer activity in single cel ...
145 Kiessling MK, Schuierer S, Stertz S, Beibel M, Bergling S, Knehr J, Carbone W, de Valliere C, Tchinda J, Bouwmeester T, Seu ...
© Springer International Publishing AG 2017 147 S.H. Tsang (ed.), Precision Medicine, CRISPR, and Genome Engineering, Advances i ...
148 with permanent healthy cells. As retinal degenerations are irreversible and the retina has little regenerative potential, pe ...
149 8.2 “Is There Anything You Can Do for Me?” Perhaps what we hear the most from our patients with inherited retinal disorders ...
150 8.3 Research Highlights Driven by a desire to improve patient care, applications of CRISPR is evolving at an accelerated rat ...
151 cells to be a “regenerative cure-all” has grown, exploitation of patients has also become a real concern [ 49 , 50 ]. We rep ...
152 of preclinical model library [ 67 ]. CRISPR-X harnesses the antibody maturation process which creates point mutations in imm ...
153 Li Y, et al. Gene therapy in patient-specific stem cell lines and a preclinical model of retini- tis pigmentosa with membra ...
154 Ghazi NG, et al. Treatment of retinitis pigmentosa due to MERTK mutations by ocular sub- retinal injection of adeno-associa ...
155 Daiger SP, Bowne SJ, Sullivan LS. Perspective on genes and mutations causing retinitis pig- mentosa. Arch Ophthalmol. 2007; ...
© Springer International Publishing AG 2017 157 S.H. Tsang (ed.), Precision Medicine, CRISPR, and Genome Engineering, Advances i ...
158 repair pathways to change and precisely edit the genome [ 1 – 3 ]. In the case of human health, CRISPR operates as both a to ...
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