Precision Medicine, CRISPR, and Genome Engineering Moving from Association to Biology and Therapeutics
14 1.5.1 Modifications and Implementation of Adeno-associated Viral Vectors AAV vectors have a long history of use in the field ...
15 Additionally, much effort has been put into understanding the antigenic epitopes of the AAV capsid by many experimental and c ...
16 serotypes used in clinical gene therapy applications (AAV5, AAV8, and AAV9) [ 162 ]. Table 1.1 summarizes some of the importa ...
17 variable region antibodies with T-cell activation domains. Unlike TCRs which rec- ognize HLA-presenting peptides and are ther ...
18 The crossover of the CRISPR multiplex editing techniques to CAR-T therapy is a new and exciting area of active investigation. ...
19 Cas9 methods were used to remove the premature stop codon in the DMD gene leading to Duchenne Muscular Dystrophy and resulted ...
20 1.7 Conclusion Genetic medicine has allowed for patient-specific treatment of disease. Progress in modification of patient-sp ...
21 Rouet P, Smih F, Jasin M. Introduction of double-strand breaks into the genome of mouse cells by expression of a rare-cutti ...
22 Li Y, et al. Gene therapy in patient-specific stem cell lines and a preclinical model of retinitis pigmentosa with membrane ...
23 Cronin J, Zhang X-Y, Reiser J. Altering the tropism of lentiviral vectors through pseudotyp- ing. Curr Gene Ther. 2005;5:387 ...
24 Jaffe HA, et al. Adenovirus–mediated in vivo gene transfer and expression in normal rat liver. Nat Genet. 1992;1:372–8. Rose ...
25 Bauerschmitz GJ, et al. Treatment of ovarian cancer with a tropism modified oncolytic adeno- virus. Cancer Res. 2002;62:1266 ...
26 Chew WL, et al. A multifunctional AAV-CRISPR-Cas9 and its host response. Nat Methods. 2016;13:868–74. Tabebordbar M, et al. ...
27 Imai C, et al. Chimeric receptors with 4-1BB signaling capacity provoke potent cytotoxicity against acute lymphoblastic leuk ...
© Springer International Publishing AG 2017 29 S.H. Tsang (ed.), Precision Medicine, CRISPR, and Genome Engineering, Advances in ...
30 crRNA CRISPR targeting RNA DMD Duchenne’s muscular dystrophy Dmd Dystrophin Fah Fumarylacetoacetate hydrolase FIX Coagulation ...
31 expressing the vascular endothelial growth factor (VEGF) inhibitor sFLT-1 in patients with age-related macular degeneration ...
32 Fig. 2.1 Cellular mechanisms of DNA repair following DNA double-strand break. When a double-strand DNA break occurs, one of t ...
33 desired genetic loci and fused with Fo kI nuclease domains to yield custom nucleases [ 15 , 16 ]. This advance opened the doo ...
34 2.2.1 A AV Adeno-associated virus (AAV) is the most clinically successful in vivo gene therapy vector to date. AAVs are a fam ...
«
1
2
3
4
5
6
7
8
9
»
Free download pdf