Abstract
CRISPR-based genome editing has revolutionized experimental biology and is being harnessed for human gene therapy. The fundamental capability of guiding a nucleotide-altering enzyme to a specific genomic or RNA sequence by means of a short RNA guide (sgRNA) allows for precise nucleic acid alterations to be made in human cells and a wide variety of different organisms. While initial applications of the CRISPR-Cas system were primarily based on the Cas9 endonuclease function, protein engineering has enabled a variety of other DNA- and RNA-altering functions to be guided by the Cas-sgRNA mechanism. Human Gene Therapy is now seeking original papers and review articles that describe the design, optimization, and application of these next-generation CRISPR approaches, including, but not limited to, the following technologies: Base editing Epigenome editing CRISPR-transactivation RNA editing Prime editing Anti-CRISPR proteins Novel Cas-based fusion proteins that confer novel functions, sequence-targeted by Cas-sgRNA
Demonstrations of the utility of one or more of such technologies in animal models of specific human diseases, including monogenic disorders, is of particular interest.
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