The NIH Somatic Cell Genome Editing program - Nature.com
Apr 07, 2021 7 mins, 55 secs
These strategies include base editing21 (in which fused deaminase enzymes rewrite individual nucleotides without inducing double-strand breaks)22,23,24 and prime editing (in which a fused reverse transcriptase introduces edits templated by an extended guide RNA)25.

These studies highlight the great potential of genome editing to improve human health.

In fact, base editing has already been used to correct pathogenic mutations, and in some cases has resulted in phenotypic rescue of the disease52,53,54,55,56,57,58,59,60,61,62,63.

Along with data, we aim to deliver a collection of tools, reagents, methods and best-practices that will be assembled into the SCGE Toolkit for Therapeutic Genome Editing (or SCGE Toolkit in short, Fig. 1b).

This approach is exemplified by the recent development of Cas12j, the smallest CRISPR–Cas genome editing system yet discovered, which was supported by the SCGE program87.

Major classes of genome editors include nucleases, base editors (BE), prime editors, PNAs, RNA editors and epigenome editors.

However, the broad application of genome editing will require nanoparticles that can target the many different types of tissue in the body.

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