Results 81 to 90 of about 60,004 (331)
Progranulin (PGRN) is a secreted glycoprotein with multiple biological functions in early embryogenesis, anti‐inflammation, and neurodegeneration. A good model for the functional study of PGRN is the zebrafish with knockdown or knockout of grn, the gene ...
Jiuling Zhu +10 more
semanticscholar +1 more source
Practical guidance for the implementation of the CRISPR genome editing tool in filamentous fungi [PDF]
Background: Within the last years, numerous reports described successful application of the CRISPR nucleases Cas9 and Cpf1 for genome editing in filamentous fungi.
Haefner, Stefan +4 more
core +1 more source
Novel gRNA design pipeline to develop broad-spectrum CRISPR/Cas9 gRNAs for safe targeting of the HIV-1 quasispecies in patients [PDF]
AbstractThe CRISPR/Cas9 system has been proposed as a cure strategy for HIV. However, few published guide RNAs (gRNAs) are predicted to cleave the majority of HIV-1 viral quasispecies (vQS) observed within and among patients. We report the design of a novel pipeline to identify gRNAs that target HIV across a large number of infected individuals.
Neil T. Sullivan +19 more
openaire +2 more sources
Machine learning based CRISPR gRNA design for therapeutic exon skipping
Restoring gene function by the induced skipping of deleterious exons has been shown to be effective for treating genetic disorders. However, many of the clinically successful therapies for exon skipping are transient oligonucleotide-based treatments that require frequent dosing.
Wilson Louie +7 more
openaire +4 more sources
Alfalfa (Medicago sativa) is an outcrossing tetraploid legume species widely cultivated in the world. The clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein 9 (CRISPR/Cas9) system has been successfully used for ...
Tezera W. Wolabu +14 more
doaj +1 more source
Cell Compartment-Specific Folding of Ty1 Long Terminal Repeat Retrotransposon RNA Genome
The structural transitions RNAs undergo during trafficking are not well understood. Here, we used the well-developed yeast Ty1 retrotransposon to provide the first structural model of genome (g) RNA in the nucleus from a retrovirus-like transposon ...
Małgorzata Zawadzka +4 more
doaj +1 more source
CRISPR/Cas9 gene editing is now revolutionizing the ability to effectively modify plant genomes in the absence of efficient homologous recombination mechanisms that exist in other organisms.
P. Do +7 more
semanticscholar +1 more source
CAS9 is a genome mutator by directly disrupting DNA-PK dependent DNA repair pathway. [PDF]
With its high efficiency for site-specific genome editing and easy manipulation, the clustered regularly interspaced short palindromic repeats (CRISPR)/ CRISPR associated protein 9 (CAS9) system has become the most widely used gene editing technology in ...
Chen, Qu +6 more
core
Reactivating Fetal Hemoglobin Expression in Human Adult Erythroblasts Through BCL11A Knockdown Using Targeted Endonucleases. [PDF]
We examined the efficiency, specificity, and mutational signatures of zinc finger nucleases (ZFNs), transcriptional activator-like effector nucleases (TALENs), and clustered regularly interspaced short palindromic repeat (CRISPR)/Cas9 systems designed to
Bjurström, Carmen F +11 more
core +3 more sources
A bacterial gene-drive system efficiently edits and inactivates a high copy number antibiotic resistance locus. [PDF]
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

