CRISPR is an abbreviation of Clustered Regularly Interspaced Short Palindromic Repeats. The name was minted at a time when the origin and use of the interspacing subsequences were not known. At that time the CRISPRs were described as segments of prokaryotic DNA containing short, repetitive base sequences. In a palindromic repeat, the sequence of nucleotides is the same in both directions. Each repetition is followed by short segments of spacer DNA from previous exposures to foreign DNA (e.g., a virus or plasmid). Small clusters of cas (CRISPR-associated) genes are located next to CRISPR sequences.
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Genome editing with an expanded CRISPR-Cas tool set increases the opportunity to make intentional, targeted changes in the genome. Furthermore, improved specificity of genome editing and abi...
A repeat expansion in the C9orf72-SMCR8 complex subunit (C9orf72) represents the most common genetic cause of two fatal neurodegenerative diseases: frontotemporal dementia (FTD) and amyotrop...
The adenosine analogue remdesivir has emerged as a front-line antiviral treatment for SARS-CoV-2, with preliminary evidence that it reduces the duration and severity of illness. Prior clinic...
An extensively debated concern about CRISPR-Cas9 genome editing is that unspecific guide RNA (gRNA) binding may induce off-target mutations. However, accurate prediction of CRISPR-Cas9 off-t...
Combinatorial inhibition of effector and feedback pathways is a promising treatment strategy for KRAS mutant cancers. However, the particular pathways that should be targeted to optimize the...