The CRIPSR/Cas gene‐editing system—an immature but useful toolkit for experimental and clinical medicine

Abstract

A Chinese scientist, Jiankui He, and his creation of the world's first genetically altered baby made headlines recently. As a newly developed gene‐editing technique, the CRISPR /Cas system should not be applied to human beings for reproductive purposes until it has been extensively tested. However, numerous experimental research studies in human somatic, germline cells, and even in embryos, have been conducted, which have shown CRISPR /Cas to be a useful tool for human genome editing and a potential therapeutic method for future clinical use.

Gene editing and CRISPR in the clinic: current and future perspectives

“…The rapidly advancing CRISPR toolkit holds many promising developments toward advancement of clinical medicine, although there are several safety and ethical concerns still to be addressed. One of the areas of greatest concern is germline editing, which creates a heritable alteration in the genome [144,145]. While an in-depth discussion of the ethical concerns and governmental regulatory concerns raised by germline editing are beyond the scope of this review, the actions in this area have the potential for far reaching effects in clinical genome editing and public image of these technologies [145].…”

Macrocyclic‐Amphiphile‐Based Self‐Assembled Nanoparticles for Ratiometric Delivery of Therapeutic Combinations to Tumors

“…Tumor is characterized by cell aberrant metabolic and proliferative behaviors, leading to the elevation of physiologic indicators (including pH, redox, or enzymes) and the formation of unique tumor microenvironment. [ 18 ] To this end, a hypoxia‐responsive macrocyclic amphiphile, QAAC4A‐12C (detailed synthesis in Supporting Information), was first synthesized as the fundamental component of MASN to provide the capability of precise loading and controlled release of drugs. In the design of QAAC4A‐12C, NH 2 C4A‐12C, which is a calixarene with four dodecyl chains attached at the lower rim, were employed as the macrocyclic scaffold due to its facile modification as well as its amphiphilic properties.…”

Dclre1c-Mutation-Induced Immunocompromised Mice Are a Novel Model for Human Xenograft Research

“…The advent of CRISPR/Cas9 gene editing has revolutionized the generation of targeted gene modifications. In this system, a single-guide RNA (sgRNA) directs the Cas9 nuclease to a specific DNA sequence, inducing a double-strand break that, upon cellular repair, results in gene knockout or knockin [13,14]. This study harnesses CRISPR/Cas9 technology to disrupt a segment of exon 10 within the mouse dclre1c gene.…”