RNA Editing for Programmable A to I Replacement (REPAIR) was developed by Feng Zhang at the Broad Institute at MIT and Harvard based on CRISPR-Cas13 was developed by Zhang’s lab to edit RNA transcripts with pathogenic mutations . The Cas13b ortholog from the bacterium Prevotella sp. P5–125 was engineered to not cleave RNA (dCas13b) and fused to an adensosine deaminase enzyme which changes A to I in targeted spot on an RNA transcript as determined by the user supplied guide RNA. Zhang’s team used REPAIR to correct RNA transcripts in cell lines from patients with mutations causing Fanconi anemia and X-linked Nephrogenic diabetes insipidus. The REPAIR system is engineered for viral delivery into cells. There are less sequence constraints for CRISPR-Cas13 RNA editing than for CRISPR-Cas9 DNA editing, making it more flexible. However since the REPAIR system targets RNA not DNA, effects are temporary. Zheng's group suggests it would be suitable to treat diseases with acute phases like inflammation or modify proteins in the disease pathway to slow disease progression.
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- Synthetic biologySynthetic biology is a multidisciplinary area of research that seeks to create new biological parts, devices, and systems, or to redesign systems that are already found in nature to have new abilities.
- BiotechnologyBiotechnology in a broad sense includes the use of living systems and organisms, as well as their parts for the development or production of products.
- CRISPR/Cas ToolsClustered regularly interspaced short palindromic repeats (CRISPR) and CRISPR associated (Cas) proteins perform adaptive immune functions in prokaryotic organisms defending against foreign nucleic acids such as viruses. CRISPR/Cas tools have been adapted for use in genome editing and other DNA and RNA targeting applications.
- CRISPR-Cas13A nucleic acid editing technology that targets RNA, analogous to the CRISPR-Cas9 system
- CRISPR-Cas13aThe CRISPR-Cas13a, like other CRISPR-Cas systems functions in bacterial immunity but this system targets invading RNA rather than DNA. CRISPR-Cas13a is being engineered for RNA sensing applications such as live cell RNA tracking and detection of pathogenic viruses or bacteria.
- CRISPRClustered regularly interspaced short palindromic repeats (CRISPR) is a prokaryotic adaptive immune response that provides immunity against foreign nucleic acids, such as viral DNA and bacterial plasmids, through the use of crRNAs (CRISPR RNAs) and associated Cas genes.