Abstract
During recent years, clustered regularly interspaced short palindromic repeat (CRISPR)/CRISPR-associated protein 9 (Cas9) technologies have been noticed as a rapidly evolving tool to deliver a possibility for modifying target sequence expression and function. The CRISPR/Cas9 tool is currently being used to treat a myriad of human disorders, ranging from genetic diseases and infections to cancers. Preliminary reports have shown that CRISPR technology could result in valued consequences for the treatment of Duchenne muscular dystrophy (DMD), cystic fibrosis (CF), β-thalassemia, Huntington’s diseases (HD), etc. Nonetheless, high rates of off-target effects may hinder its application in clinics. Thereby, recent studies have focused on the finding of the novel strategies to ameliorate these off-target effects and thereby lead to a high rate of fidelity and accuracy in human, animals, prokaryotes, and also plants. Meanwhile, there is clear evidence indicating that the design of the specific sgRNA with high efficiency is of paramount importance. Correspondingly, elucidation of the principal parameters that contributed to determining the sgRNA efficiencies is a prerequisite. Herein, we will deliver an overview regarding the therapeutic application of CRISPR technology to treat human disorders. More importantly, we will discuss the potent influential parameters (e.g., sgRNA structure and feature) implicated in affecting the sgRNA efficacy in CRISPR/Cas9 technology, with special concentration on human and animal studies.
Highlights
Regarding the engineered or bacterial nucleases, the evolution of genome editing technology has ensured the opportunity of direct and selective detecting and amendment of genomic sequences, more importantly in all eukaryotic cells (Bedell et al, 2012; Adli, 2018)
In addition to verify that the GC content between 40% and 60% is favored for knockdown of a target gene, SOCS1, by single-stranded guide RNA (sgRNA), this study revealed that the positive traits in sgRNAs are the circumvention of both a C at position 3 and a G at position 16 (Bruegmann et al, 2019)
During the last three decades, life sciences have been developed by genome editing technology, in particular through clustered regularly interspaced short palindromic repeat (CRISPR)/Cas systems, enabling the targeted alteration of genomic DNA of all organisms
Summary
Regarding the engineered or bacterial nucleases, the evolution of genome editing technology has ensured the opportunity of direct and selective detecting and amendment of genomic sequences, more importantly in all eukaryotic cells (Bedell et al, 2012; Adli, 2018). The CRISPR/cas9mediated correction of hemoglobin E mutation in patientderived iPSCs and their efficient differentiation into HSCs offers the rationality of the autologous transplantation in patients with HbE/β-thalassemia in the clinic These HSCs can be cultured in the erythroid liquid culture system and developed into red blood cells (RBC) expressing mature β-globin gene and β-globin protein (Wattanapanitch et al, 2018).
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