Results 121 to 130 of about 251,083 (336)

Genome Engineering ofDrosophilawith the CRISPR RNA-Guided Cas9 Nuclease [PDF]

open access: bronze, 2013
Scott J. Gratz   +7 more
openalex   +1 more source

A bacterial gene-drive system efficiently edits and inactivates a high copy number antibiotic resistance locus. [PDF]

open access: yes, 2019
Gene-drive systems in diploid organisms bias the inheritance of one allele over another. CRISPR-based gene-drive expresses a guide RNA (gRNA) into the genome at the site where the gRNA directs Cas9-mediated cleavage.
Bier, Ethan   +3 more
core   +1 more source

Exploring AAV‐Mediated Gene Therapy for Inner Ear Diseases: from Preclinical Success to Clinical Potential

open access: yesAdvanced Science, EarlyView.
Current preclinical studies of AAV‐mediated gene therapy explore different strategies based on the characteristics of inner ear diseases. For genetic hearing loss, approaches include the replacement of a “good gene,” removal of a “bad gene,” or direct correction of mutations through base editing.
Fan Wu   +7 more
wiley   +1 more source

Single step production of Cas9 mRNA for zygote injection

open access: yesBioTechniques, 2018
Production of Cas9 mRNA in vitro typically requires the addition of a 5´ cap and 3´ polyadenylation. A plasmid was constructed that harbored the T7 promoter followed by the EMCV IRES and a Cas9 coding region.
Bethany K Redel   +8 more
doaj   +1 more source

Correction of a Genetic Disease in Mouse via Use of CRISPR-Cas9 [PDF]

open access: hybrid, 2013
Yuxuan Wu   +8 more
openalex   +1 more source

NOX2 Contributes to High‐Frequency Outer Hair Cell Vulnerability in the Cochlea

open access: yesAdvanced Science, EarlyView.
This study first identifies NOX2 as a differentially expressed gene related to oxidative damage in the apical and basal turns through single‐cell RNA sequencing. NOX2 gene knockout mitigates OHCs damage caused by neomycin and noise and enhances Nrf2 expression and nuclear translocation.
Meihao Qi   +16 more
wiley   +1 more source

Recent Advances in Genome Editing Using CRISPR/Cas9

open access: yesFrontiers in Plant Science, 2016
The CRISPR (clustered regularly interspaced short palindromic repeat)-Cas9 (CRISPR-associated nuclease 9) system is a versatile tool for genome engineering that uses a guide RNA (gRNA) to target Cas9 to a specific sequence.
Yuduan eDing   +6 more
doaj   +1 more source

Robust and stable transcriptional repression in Giardia using CRISPRi. [PDF]

open access: yes, 2019
Giardia lamblia is a binucleate protistan parasite causing significant diarrheal disease worldwide. An inability to target Cas9 to both nuclei, combined with the lack of nonhomologous end joining and markers for positive selection, has stalled the ...
Booker, J   +7 more
core   +1 more source

The Reconstruction of Peripheral Auditory Circuit: Recent Advances and Future Challenges

open access: yesAdvanced Science, EarlyView.
This paper summarizes the potential of biomaterials, stem cells, and gene editing technologies in the regeneration of inner ear hair cells, spiral ganglion neurons, and inner ear organoids. Challenges and potential developments are discussed and explored.
Zhe Li   +3 more
wiley   +1 more source

High-Throughput Screens of PAM-Flexible Cas9 Variants for Gene Knockout and Transcriptional Modulation

open access: yesCell Reports, 2020
Summary: A key limitation of the widely used CRISPR enzyme S. pyogenes Cas9 is the strict requirement of an NGG protospacer-adjacent motif (PAM) at the target site. This constraint can be limiting for genome editing applications that require precise Cas9
Mateusz Legut   +6 more
doaj  

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