Sickle cell disease (SCD) is the most common monogenic hematologic disorder and is
essentially congenital hemolytic anemia caused by an inherited point mutation in the β …

Clustered regularly interspaced short palindromic repeats (CRISPR) technology has
transformed molecular biology and the future of gene-targeted therapeutics. CRISPR …

Chemical modifications of CRISPR-Cas nucleases help decrease off-target editing and
expand the biomedical applications of CRISPR-based gene manipulation tools. Here, we …

Currently, the CRISPR-Cas9 system serves as a prevalent tool for genome editing and gene
expression regulation. Its therapeutic application is limited by off-target effects that can affect …

Therapeutic mRNAs are generated using modified nucleotides, namely N 1-
methylpseudouridine (m 1 Ψ) triphosphate, so that the mRNA evades detection by the …

The design of controllable and precise RNA-targeted CRISPR/Cas9 (Clustered Regularly
Interspaced Short Palindromic Repeats) systems is an important problem of modern …

Research on Cas9 nucleases from different organisms holds great promise for advancing
genome engineering and gene therapy tools, as it could provide novel structural insights …

At present, there are many strategies to improve the activity of CRISPR/Cas9. A well-known
and effective approach is guide RNA modification. Many chemical guide RNA modifications …

Post-transcriptional modifications on the guide RNAs utilized in the Cas9 system may have
the potential to impact the activity of Cas9. In this study, we synthesized a series of …

Research on Cas9 nucleases from different organisms holds great promise for advancing
genome engineering and gene therapy tools as it could provide novel structural insights into …