Sci Rep 7: 4159.Ohmori T, Nagao Y, Mizukami H, Sakata A, Muramatsu SI, Ozawa K, Tominaga SI, Hanazono Y, Nishimura S, Nureki O et al (2017) CRISPR/ Cas9-mediated genome editing via postnatal administration of AAV vector cures haemophilia B mice. Sci Rep 7: 4159...
(D) Frequency of indels at on-target (m-H530R) and off-target (m-OT1-m-OT10 and m-WT) sites in heart genomic DNA from the +/H530R mouse injected with AAV9-Cas9/sgRNA-m3 at P4 or P42. Data represent mean ± SD (n = 6). Most prevalent on-target indels of PRKAG2 H530R...
but the phenotype could be masked under optimal culture conditions. Supporting this idea, we found thatOTUD5KO hESCs showed reduced growth in the E8 condition when the cells were passaged without the Rho-associated protein kinase (ROCK) inhibitor Y-27632 (Fig.4i), a compound normally...
MDA-MB-231-LUC cells (2 × 106) were subcutaneously injected into the right inguinal mammary fat pad of the mice to establish a Breast cancer orthotopic transplantation model [55]. The bioluminescent signal of MDA-MB-231 cells was analyzed using the CRi Maestro in vivo imaging system (C...
In comparison with the normal wild‐type mice ( Fam83h W/W), the F2 homozygous KO ( Fam83h Ko/Ko) had sparse, scruffy coats with small body size and decreased general activity. Also, they had the natural reproductive ability and natural lifespan. In addition, delay in opening the eyes ...
PSEN2N141I causes an increase in the Aβ42/40 ratio in the brains of transgenic mice; additionally, this enhanced Aβ42 production was observed in neural cell lines upon induced overexpression of mutant PSEN2 N141I protein [83] and in iPSC derived from PSEN2 N141I mutant patients [93]. ...
(7/648) with acquired megabase LOH induced by CRISPR-Cas9. In-depth analysis by SNP-array reveals the presence of copy-neutral LOH. This leads to 11p15.5 partial uniparental disomy, comprising two Chr11p15.5 imprinting centers (H19/IGF2:IG-DMR/IC1andKCNQ1OT1:TSS-DMR/IC2) and impactingH...
the TS9 and many of the TS11 off-target sites. At two of the SpCas9 off-target sites (OT9-4 and OT9-8), the use of the attenuated SpCas9MT2-SaCas9WTnuclease dramatically reduces the off-target activity compared to the independent wild-type SpCas9 or the SpCas9WT–SaCas9WTnuclease,...
Here we report defective electrophysiological properties in iPSC-derived BFCNs that are specifically associated with the PSEN2 N141Ifamilial mutation. Interestingly, although some of the previous studies attribute this impairment in neuronal activity to the build-up of plaques in the brain of AD mice...
GUIDE_seq-OT#1 CTGTGGAGG reverse primer to amplify EMX1-1- GCTTTTATACCATCTT 190. GUIDE_seq-OT#1 GGGGTTACAG forward primer to amplify EMX1-1- CAATGTGCTTCAACCC 191. GUIDE_seq-OT#2 ATCACGGC reverse primer to amplify EMX1-1- CCATGAATTTGTGATG 192. GUID...