Fig. 2 Cas9/RecA-mediated in vivo gene correction of Pde6b.

Slides:

Advertisements

Similar presentations

Fig. 5 Correlation of RNA expression and protein abundance.

Advertisements

Fig. 4 Altitudinal distributions of ice thickness change (m year−1) for the 650 glaciers. Altitudinal distributions of ice thickness change (m year−1)

Fig. 2 Global production, use, and fate of polymer resins, synthetic fibers, and additives (1950 to 2015; in million metric tons). Global production, use,

Fig. 5 Thermal conductivity of n-type ZrCoBi-based half-Heuslers.

Fig. 1 Map of water stress and shale plays.

Fig. 1 Examples of experimental stimuli and behavioral performance.

Fig. 1 NP-free Ch-CNC droplets.

Fig. 4 Morphogenesis in the Ch-CNC host droplets and NP assemblies.

Fig. 6 Comparison of properties of water models.

Fig. 2 Reference-fixing experiment, results.

Fig. 1 Experimental apparatus used to train and test free-flying bees on their capacity to learn addition and subtraction. Experimental apparatus used.

Fig. 2 TRXSS data. TRXSS data. TRXSS data covering delay times from 10 ns to 10 ms for wild-type DmCry (A), for the DmCry(H378A) mutant (B), and for XlPho.

Fig. 3 Rotation experiment, setup.

Fig. 1 Product lifetime distributions for the eight industrial use sectors plotted as log-normal probability distribution functions (PDF). Product lifetime.

Fig. 5 EGLN2-mediated HIF1A down-regulation promotes ferroptosis.

Fig. 1 Bioinspired design of AAD for promoting wound contraction.

Fig. 2 Contribution of SQSTM1 to the autophagic degradation of ARNTL.

Fig. 1 Distribution of total and fake news shares.

Fig. 1 Schematic illustrating the combined use of bioorthogonal ligation (i.e., the reaction between Tz and TCO) and disulfide cleavage–driven by DTT to.

Fig. 6 RUNX/CBFB interaction inhibitor, Ro5-3335, significantly decreases mouse neurofibroma growth in vivo. RUNX/CBFB interaction inhibitor, Ro5-3335,

Fig. 3 Photon number statistics resulting from Fock state |l, S − l〉 interference. Photon number statistics resulting from Fock state |l, S − l〉 interference.

Fig. 2 2D QWs of different propagation lengths.

Fig. 3 Magnetic and transport properties of ACoO3 (A = Ca, Sr).

Electronic structure of the oligomer (n = 8) at the UB3LYP/6-31G

Fig. 6 WPS imaging of different chemical components in living cells.

Fig. 4 Cdc42 enhances the cellular uptake of EVs.

Fig. 4 DFT ωB97x/def2-TZVPP atomic charges on the sulfur atom of substituted thioaldehyde and AIMNet prediction with a different number of iterative passes.

Fig. 3 ET dynamics on the control and treatment watersheds during the pretreatment and treatment periods. ET dynamics on the control and treatment watersheds.

Fig. 1 Histograms of the number of first messages received by men and women in each of our four cities. Histograms of the number of first messages received.

Fig. 5 Schematic phase diagrams of Ising spin systems and Mott transition systems. Schematic phase diagrams of Ising spin systems and Mott transition systems.

Fig. 3 Transcriptional changes in dKO mice.

Fig. 4 OER performance of ACoO3 (A = Ca, Sr) in alkaline solutions with different pH. OER performance of ACoO3 (A = Ca, Sr) in alkaline solutions with.

Fig. 1 Average contribution (million metric tons) of seafood-producing sectors, 2009–2014. Average contribution (million metric tons) of seafood-producing.

Fig. 4 Evolution of fraction of sickled RBCs under hypoxia.

Fig. 1 Exon 44–deleted DMD patient iPSC-derived cardiomyocytes express dystrophin after CRISPR-Cas9–mediated genome editing. Exon 44–deleted DMD patient.

Fig. 2 Results of the learning and testing phases.

by Pan Tao, Xiaorong Wu, and Venigalla Rao

Fig. 2 Schematic drawings of Göbekli Tepe skulls.

Fig. 2 NH3, NOx, SO2, and NMVOC emission changes triggered by the JJJ clean air policy. NH3, NOx, SO2, and NMVOC emission changes triggered by the JJJ.

Fig. 1 Size fractions of MPPs in different fertilizers.

Fig. 2 Viperin promotes methionine oxidation of KSHV helicase.

Fig. 2 Top 10 countries, ecoregions, conservation hotspots, and KBAs with the largest area of restoration hotspots. Top 10 countries, ecoregions, conservation.

Fig. 5 Comparison of the liquid products generated from photocatalytic CO2 reduction reactions (CO2RR) and CO reduction reactions (CORR) on two catalysts.

Schematic of the proposed brain-controlled assistive hearing device

Fig. 1 Location of the Jirzankal Cemetery.

Fig. 4 CO2 emission changes triggered by the JJJ clean air policy.

Fig. 3 Electroporation-mediated RNAi.

Fig. 3 Directional rolling of an NP on a dsDNA fragment with flexibility gradient. Directional rolling of an NP on a dsDNA fragment with flexibility gradient.

Fig. 2 Simulations of possible doping positions and band structures.

Multiplexed four- and eight-channel devices for rapid processing

Fig. 2 Mean field results. Mean field results. (A) Solutions P(x) to Eq. 4 for a range of T and wc = (B) Modulus ∣pk∣ of order parameters versus.

Fig. 4 The relationship between the total mean absolute momentum disturbance 〈∣p∣〉zB (in units of ℏ/D) and fringe visibility V. The relationship between.

Experimenter gender and replicability in science

Fig. 1 Schematic depiction of a paradigm for rapid and guided discovery of materials through iterative combination of ML with HiTp experimentation. Schematic.

Fig. 4 No genetic divergence of HIV during cART.

Fig. 4 Spatial mapping of the distribution and intensity of industrial fishing catch. Spatial mapping of the distribution and intensity of industrial fishing.

Fig. 2 Relaxation to a fitness maximum does not generate a logarithmic fitness trajectory. Relaxation to a fitness maximum does not generate a logarithmic.

Fig. 3 Performance of the generative model G, with and without stack-augmented memory. Performance of the generative model G, with and without stack-augmented.

Fig. 1 Schematic structure of a fluorescently labeled eGLP1-conjugated MALAT1 ASO and internalization of fluorescent eGLP1 and eGLP1-MALAT1-ASO. Schematic.

Fig. 4 Behavior of resistance peak near density nm = 5.

Fig. 2 Comparison between the different reflective metasurface proposals when θi = 0° and θr = 70°. Comparison between the different reflective metasurface.

Fig. 4 Systemic AAV9 delivery of gene editing components to ΔEx44 mice rescues dystrophin expression. Systemic AAV9 delivery of gene editing components.

Fig. 2 Speaker-independent speech separation with ODAN.

Fig. 3 Calculated electronic structure of ZrCoBi.

Fig. 3 Correction of Dmd exon 44 deletion in mice by intramuscular AAV9 delivery of gene editing components. Correction of Dmd exon 44 deletion in mice.

Fig. 4 MxB intermolecular assembly interfaces and their role in the MxB assembly and HIV-1 inhibition. MxB intermolecular assembly interfaces and their.

Fig. 6 Schematic illustration of the decreased conformational stability in the N92I mutant and its activation mechanism. Schematic illustration of the.

Mitochondrial import of CerS1 from smooth ER mediates lethal mitophagy

Fig. 5 Enhancers and super-enhancers identified in various brain cell populations. Enhancers and super-enhancers identified in various brain cell populations.

Fig. 5 CD19-tPSMA(N9del) CAR T cell numbers in mouse and human.

Presentation transcript:

Fig. 2 Cas9/RecA-mediated in vivo gene correction of Pde6b. Cas9/RecA-mediated in vivo gene correction of Pde6b. (A) Schematic of Pde6b gene correction by Cas9/RecA in rd1 (top) and wild-type (bottom) mice. The Pde6b point mutation (C to A) is marked in red, and the premature stop codon of the Pde6b rd1 mutation is labeled by a red arrowhead. The truncated rd1 protein is in red, and the wild-type sequence loss in the rd1 mutant is in blue. (B) Experimental design for Cas9/RecA-mediated HDR gene correction of Pde6b rd1 mutant mice. Plasmids required for Cas9/RecA were introduced into mouse retinae by retinal electroporation. (C) Schematic of genomic DNA and cDNA sequencing of Cas9/RecA-treated cells. EGFP+ cells from treated rd1 mice were collected using glass capillaries, with DNA extracted and enriched by Dde I restricted enzyme digestion for genome sequencing or cDNA collected through Smart-seq2 methods. (D) Representative direct genomic sequencing results from Cas9/RecA-mediated HDR gene correction. The Cas9-treated group (top) did not detect any gene correction. The Cas9/RecA-treated group (bottom) showed A to C conversion (red dashed frame). (E) Representative sequencing result of Pde6b cDNA from the Cas9/RecA-treated group. A was converted to C by Cas9/RecA-mediated gene correction. (F) Representative Western blot results from Cas9/RecA-mediated gene correction, and quantification of levels of PDE6B protein. Western blot for PDE6B shows the recovery of PDE6B expression. Actin was used as a loading control. Two retinae were mixed together for each Western blot sample, and three independent experiments were done. 1/10 wild type, loading 1/10 volume wild type and 9/10 sample buffer to make up the volume; Cas9, rd1 mice treated with Cas9, sgRNA, and donor; Cas9/RecA, rd1 mice treated with Cas9, sgRNA, donor, and RecA. Yuan Cai et al. Sci Adv 2019;5:eaav3335 Copyright © 2019 The Authors, some rights reserved; exclusive licensee American Association for the Advancement of Science. No claim to original U.S. Government Works. Distributed under a Creative Commons Attribution NonCommercial License 4.0 (CC BY-NC).