Results 31 to 40 of about 43,663 (164)

CRISPR-Cas9 Gene Editing of Hematopoietic Stem Cells from Patients with Friedreich's Ataxia. [PDF]

, 2020
Friedreich's ataxia (FRDA) is an autosomal recessive neurodegenerative disorder caused by expansion of GAA repeats in intron 1 of the frataxin (FXN) gene, leading to significant decreased expression of frataxin, a mitochondrial iron-binding protein.
Cherqui, Stephanie, Haquang, Joseph H, Luebeck, Jens, Mali, Prashant, Rainaldi, Joseph N, Rocca, Celine J, Sharma, Jay, Shi, Yanmeng   +7 more
core  

Efficient In Vivo Genome Editing Using RNA-Guided Nucleases [PDF]

, 2014
Clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated (Cas) systems have evolved in bacteria and archaea as a defense mechanism to silence foreign nucleic acids of viruses and plasmids.
Fu, Yanfang, Hwang, Woong Y., Joung, J. Keith, Maeder, Morgan L., Peterson, Randall T., Reyon, Deepak, Sander, Jeffry D., Tsai, Shengdar Q., Yeh, J.-R. Joanna   +8 more
core   +1 more source

Distinct horizontal transfer mechanisms for type I and type V CRISPR-associated transposons

bioRxiv, 2023
CRISPR-associated transposons (CASTs) co-opt CRISPR-Cas proteins and Tn7-family transposons for RNA-guided vertical and horizontal transmission. CASTs encode minimal CRISPR arrays but can’t acquire new spacers.

semanticscholar   +1 more source

The physicist's guide to one of biotechnology's hottest new topics: CRISPR-Cas

, 2018
Clustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR-associated proteins (Cas) constitute a multi-functional, constantly evolving immune system in bacteria and archaea cells.
Bonomo, Melia E., Deem, Michael W.
core   +1 more source

BATCH-GE : batch analysis of next-generation sequencing data for genome editing assessment [PDF]

, 2016
Targeted mutagenesis by the CRISPR/Cas9 system is currently revolutionizing genetics. The ease of this technique has enabled genome engineering in-vitro and in a range of model organisms and has pushed experimental dimensions to unprecedented proportions.
Boel, Annekatrien, Callewaert, Bert, Coucke, Paul, De Paepe, Anne, De Rocker, Nina, Menten, Björn, Steyaert, Wouter, Willaert, Andy   +7 more
core   +2 more sources

Peroxidasin enables melanoma immune escape by inhibiting natural killer cell cytotoxicity

Molecular Oncology, EarlyView.
Peroxidasin (PXDN) is secreted by melanoma cells and binds the NK cell receptor NKG2D, thereby suppressing NK cell activation and cytotoxicity. PXDN depletion restores NKG2D signaling and enables effective NK cell–mediated melanoma killing. These findings identify PXDN as a previously unrecognized immune evasion factor and a potential target to improve
Hsu‐Min Sung, David Bickel, Lena C. M. Krause, Daria Ezeriņa, Christian Ickes, Julian Wojtachnia, Christine S. Gibhardt, Magdalena Shumanska, Khadija Wahni, Andrea Paluschkiwitz, Julia Malo Pueyo, Ekaterina Baranova, Wim Vranken, Hedwig Stanisz, Ioana Stejerean‐Todoran, Michael P. Schön, Joris Messens, Ivan Bogeski   +17 more
wiley   +1 more source

Bacteria herald a new era of gene editing. [PDF]

, 2013
The demonstration that nucleases guided by bacterial RNA can disrupt human genes represents a landmark in the rapidly developing field of genome ...
Segal, David J
core  

Efficient genome editing in zebrafish using a CRISPR-Cas system [PDF]

, 2013
In bacteria, foreign nucleic acids are silenced by clustered, regularly interspaced, short palindromic repeats (CRISPR)--CRISPR-associated (Cas) systems.
Fu, Yanfang, Hwang, Woong Y., Joung, J. Keith, Maeder, Morgan L., Peterson, Randall T., Reyon, Deepak, Sander, Jeffry D., Tsai, Shengdar Q., Yeh, J.-R. Joanna   +8 more
core   +1 more source

CRISPRtracrRNA: robust approach for CRISPR tracrRNA detection

Bioinform., 2022
Motivation The CRISPR-Cas9 system is a Type II CRISPR system that has rapidly become the most versatile and widespread tool for genome engineering. It consists of two components, the Cas9 effector protein, and a single guide RNA that combines the spacer (
A. Mitrofanov, Marcus Ziemann, Omer S. Alkhnbashi, W. Hess, R. Backofen   +4 more
semanticscholar   +1 more source

CRISPR in MOF Formulation with Enhanced Stability, Activity, and Altered PAM Specificity for Broad‐Spectrum Diagnosis of Bacterial Sepsis

Advanced Science, EarlyView.
CRISPR‐FLEXMO (CRISPR with flexible PAM in MOF encapsulation) integrates a PAM‐relaxed Cas12a K607R enzyme with a manganese‐coordinated MOF for broad‐spectrum diagnosis of bacterial sepsis. The MOF enhances activity and preserves functionality under heat, solvent, and chaotropic stress, supporting long‐term room‐temperature storage.
Tathagata Pal, Zilong Liu, Meera G. Nair, Juhong Chen   +3 more
wiley   +1 more source