The advent of CRISPR-Cas9 as an RNA-guided site-specific DNA endonuclease facilitates genome modification in a precise and efficient way and brings a new dawn to targeted gene therapy.3,4However, this genome editing tool has suffered from controversy due to its off-target effects. In 2016, ba...
fine-mapping of urate-associated loci and colocalization with gene expression in 47 tissues implicated the kidney and liver as the main target organs and prioritized potentially causal genes and variants, including the transcriptional master regulators in the liver and kidney,HNF1AandHNF4A. Experimental...
single-stranded nucleic acid having a structure of X-Y-Z and comprising a nucleotide sequence that can form a complementary bond to all or part of the nucleotide sequence of a single target gene containing a genetic variation such as single nucleotide polymorphism (SNP), point mutation, or ...
A mutation creating a potential illegitimate microRNA target site in the myostatin gene affects muscularity in sheep 2006. A mutation creating a potential illegitimate microRNA target site in the myostatin gene affects muscularity in sheep. Nat. Genet. 38:813-818. http... Clop A,Marcq F,...
MicroRNAs (miRNAs) are an abundant class of short, non-coding, endogenous RNAs that regulate gene expression at the post-transcriptional level [1, 2]. The mature miRNA is a ~20-23 nucleotides long single stranded sequence, which derives from primary transcript miRNA (pri-miRNAs) [3]. One ...
single-handedly transform the expression of a broad cast of large neurogenic genes. The use of genome editing tools to change/delete specific sites or gene regions will certainly assist us in understanding the genetic etiology of behavior.
Genetic Instruments for Drug Target Gene Expression We used publicly available data from the eQTLGen consortium (n = 31 684)12 (eTable 1 in Supplement 2) to identify common (minor allele frequency >1%) single-nucleotide variants (SNVs) associated with the expression of antihypertensive dru...
These crystals were crushed and used to microseed the MCSG1 screen again at a ratio of 3:2:1 protein:precipitant:seed stock, resulting in single plate-like crystals grown from 0.2 M ammonium sulfate, 0.1 M bis-Tris (pH 6.5) and 25% PEG 3350. After further optimization to 0....
Expanding on the idea that multiple CREs can converge to regulate the expression of a single target gene31,32,42, we asked whether the CREs we identified collaboratively regulateFOXA1mRNA expression. Here, we applied a transient approach that delivers Cas9 protein:gRNA as a ribonucleoprotein (RNP...
89The fundamental principle of synthetic lethality is that the combination of two gene mutations occurring either simultaneously or sequentially leads to death, while the adverse effect of single mutation of such a gene pair is buffered by another unmutated gene. From the molecular aspect, synthetic ...