Results 71 to 80 of about 361,542 (380)

Transcription Activator-Like Effector Nuclease (TALEN)-Mediated CLYBL Targeting Enables Enhanced Transgene Expression and One-Step Generation of Dual Reporter Human Induced Pluripotent Stem Cell (iPSC) and Neural Stem Cell (NSC) Lines

PLoS ONE, 2015
Targeted genome engineering to robustly express transgenes is an essential methodology for stem cell-based research and therapy. Although designer nucleases have been used to drastically enhance gene editing efficiency, targeted addition and stable ...
Trevor Cerbini, Ray Funahashi, Yongquan Luo, Chengyu Liu, Kyeyoon Park, M. Rao, Nasir Malik, J. Zou   +7 more
semanticscholar   +1 more source

Visual Recovery Reflects Cortical MeCP2 Sensitivity in Rett Syndrome

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective Rett syndrome (RTT) is a devastating neurodevelopmental disorder with developmental regression affecting motor, sensory, and cognitive functions. Sensory disruptions contribute to the complex behavioral and cognitive difficulties and represent an important target for therapeutic interventions.
Alex Joseph Simon, Nathalie Picard, Valeria d'Andrea, Enchi Chang, Joseph Leffler, Eleonora Centofante, Matthew Taylor, Francesca Bardi, Francesca Cavicchiolo, Takao K. Hensch, Stefano Panzeri, Chinfei Chen, Michela Fagiolini   +12 more
wiley   +1 more source

Random and targeted transgene insertion in C. elegans using a modified Mosl transposon

Nature Methods, 2014
We have generated a recombinant Mos1 transposon that can insert up to 45-kb transgenes into the Caenorhabditis elegans genome. The minimal Mos1 transposon (miniMos) is 550 bp long and inserts DNA into the genome at high frequency (∼60% of injected ...
Christian Frøkjær-Jensen, M. Davis, M. Sarov, Jon Taylor, S. Flibotte, Matthew L LaBella, Andrei Pozniakovsky, D. Moerman, Erik M. Jorgensen   +8 more
semanticscholar   +1 more source

Targeting a therapeutic LIF transgene to muscle via the immune system ameliorates muscular dystrophy. [PDF]

, 2019
Many potentially therapeutic molecules have been identified for treating Duchenne muscular dystrophy. However, targeting those molecules only to sites of active pathology is an obstacle to their clinical use.
Bertoni, Carmen, Flores, Ivan, Ramos, Julian, Tidball, James G, Wang, Ying, Wehling-Henricks, Michelle, Welc, Steven S   +6 more
core  

The role of the Cucumber mosaic virus 2b protein in viral movement and symptom induction [PDF]

, 2009
The Cucumber mosaic virus (CMV) 2b protein is a counter-defense factor and symptom determinant. Conserved domains in the 2b protein sequence were mutated in the 2b gene of strain Fny-CMV.
Chen H.-Y., Fiona C. Robertson, Ham B.-K., Heiko Ziebell, John A. Walsh, John P. Carr, Ki Hyun Ryu, Lot H., Mathew Lewsey, Monique Surette, Palukaitis P., Peter Palukaitis, Roossinck M. J., Sun Hee Choi, Tina Payne, Tomas Canto   +15 more
core   +2 more sources

Lipid Nanoparticles for the Delivery of CRISPR/Cas9 Machinery to Enable Site‐Specific Integration of CFTR and Mutation‐Agnostic Disease Rescue

Advanced Functional Materials, EarlyView.
Lipid nanoparticles (LNPs) are optimized to co‐deliver Cas9‐encoding messenger RNA (mRNA), a single guide RNA (sgRNA) targeting the endogenous cystic fibrosis transmembrane conductance regulator (CFTR) gene, and homologous linear double‐stranded donor DNA (ldsDNA) templates encoding CFTR.
Ruth A. Foley, Paul G. Ayoub, Vrishti Sinha, Colin Juett, Alicia Sanoyca, Emily C. Duggan, Lindsay E. Lathrop, Priyanka Bhatt, Kevin Coote, Beate Illek, Brigitte N. Gomperts, Donald B. Kohn, Steven J. Jonas   +12 more
wiley   +1 more source

Transgene Expression and Bt Protein Content in Transgenic Bt Maize (MON810) under Optimal and Stressful Environmental Conditions

PLoS ONE, 2015
Bt protein content in transgenic insect resistant (Bt) maize may vary between tissues within plants and between plants growing under different environmental conditions.
Miluse Trtikova, O. Wikmark, N. Zemp, A. Widmer, A. Hilbeck   +4 more
semanticscholar   +1 more source

MicroR159 regulation of most conserved targets in Arabidopsis has negligible phenotypic effects [PDF]

, 2016
BACKGROUND A current challenge of microRNA (miRNA) research is the identification of biologically relevant miRNA:target gene relationships. In plants, high miRNA:target gene complementarity has enabled accurate target predictions, and slicing of target ...
Allen, Robert S, Alonso-Peral, Maria M, Gubler, Frank, Li, Junyan, Millar, Anthony A, White, Rosemary G   +5 more
core   +1 more source

Modular Platform for Rapidly Investigating Long‐Distance Propagation of Human Neural Network Activity

Advanced Healthcare Materials, EarlyView.
This study presents the first human neural organoid culture model capable of rapidly exhibiting long‐distance neural network propagation, thus delivering a system to experimentally investigate large‐scale communication during normal and diseased states.
Megh Dipak Patel, Sailee Sham Lavekar, Ronak Jaisalmeria, Suki Oji, Jazmine Jayasi, Caroline Cvetkovic, Robert Krencik   +6 more
wiley   +1 more source

TG6050, an oncolytic vaccinia virus encoding interleukin-12 and anti-CTLA-4 antibody, favors tumor regression via profound immune remodeling of the tumor microenvironment

Journal for ImmunoTherapy of Cancer
Background TG6050 was designed as an improved oncolytic vector, combining the intrinsic properties of vaccinia virus to selectively replicate in tumors with the tumor-restricted expression of recombinant immune effectors to modify the tumor immune ...
Laetitia Fend, Eric Quemeneur, Jean-Baptiste Marchand, Nathalie Silvestre, Johann Foloppe, Christelle Remy, Patricia Kleinpeter, Jules Deforges, Fadi Azar, Christelle Demeusoit, Clémentine Spring-Giusti   +10 more
doaj   +1 more source