Due to its simplicity, versatility, and high efficiency, the clustered regularly interspaced short
palindromic repeat (CRISPR)/Cas9 technology has emerged as one of the most promising …

The translational gap to treatments based on gene therapy has been reduced in recent
years because of improvements in gene editing tools, such as the CRISPR/Cas9 system and …

Protein-based therapeutics can activate the adaptive immune system, leading to the
production of neutralizing antibodies and the clearance of the treated cells mediated by …

The discovery of RNA guided endonucleases has emerged as one of the most important
tools for gene edition and biotechnology. The selectivity and simplicity of the CRISPR/Cas9 …

The toolbox for genetic engineering has quickly evolved from CRISPR/Cas9 to a myriad of
different gene editors, each with promising properties and enormous clinical potential …

Cyclodextrins (CDs) are cyclic polysaccharides characterized by their unique hollow
structure, making them highly effective carriers for pharmaceutical agents. CD-based …

CRISPR/Cas9 has proved its efficiency in vitro, where we now know that this tool can
efficiently target specific parts of the genome. These modifications can be used to generate …

Clonal cell lines harboring loss-of-function mutations in genes of interest are crucial for
studying the cellular functions of the encoded proteins. Recent advances in genome …

ABSTRACT A major hurdle in protein-based therapeutics is the interaction with the adaptive
immune system, which can lead to neutralization by circulating antibodies and clearance of …

Genome engineering using programmable nucleases is a rapidly evolving technique that
enables precise genetic manipulations within complex genomes. Although this technology …