CRISPR-Enabled Autonomous Transposable Element (CREATE) for RNA-based gene editing and delivery
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To realize the potential of genome editing for broad therapeutic applications, new tools are needed for delivery of multi-kilobase (kb) payloads at desired genomic sites with high precision. Here we introduce the CRISPR-Enabled Autonomous Transposable Element (CREATE), an RNA-based genome editing system that merges CRISPR/Cas9 with the human L1 retrotransposon to insert gene-sized payloads without DNA donors or double strand breaks. CREATE enables L1-mediated reverse transcription and integration of an RNA-encoded payload gene specifically at two Cas9-induced nick sites. CREATE is delivered using mRNA components and achieves integration of >1 kb payload in mammalian cells, opening the door to mRNA mediated therapeutic genome editing in vivo.
One-Sentence Summary
An mRNA-based, large-scale genome editing technology integrating CRISPR/Cas9 and L1 retroelement.
