Colorado Patent of the Month – February 2021
Biomedical research has been working on manipulating gene sequences to make precise changes to the genome. Certain nucleases (e.g. nucleic acid-guided nucleases) make these precise changes possible. These nucleases are restricted by the presence of specific PAM near the target sequence. A PAM is a short nucleotide sequence, usually 2-7 base pairs away from the target sequence. Without a specific PAM present, the nuclease will not be able to recognize the target sequence. Different nucleases require different PAMs. Inscripta, Inc. has been developing novel nucleases with varied PAM preferences, called MAD-series nucleases.
MAD-series nucleases increase the number of target sequences that can be targeted for editing which means there is less genome that cannot be edited. MAD-series nucleases may be delivered to cells to be edited as a polypeptide. Alternatively, a polynucleotide sequence encoding the mined MAD-series nucleases could be transformed or transfected into the cells to be edited. The MAD-series nuclease of choice will depend on the desired edit and the presence of an appropriate PAM. They then use a guide nucleic acid (gRNA) to join with the nuclease and direct the nuclease to the target sequence. The MAD-series nucleases have a The MAD-series nucleases are able to make their gene edits and inactivate existing PAM sequences, ensuring that future permutations will develop without the PAM, and will not be subject to future editing. The MAD-series nucleases are then able to edit only the required cells, and not the future versions, thereby providing a definitive end-date for editing.
Inscripta’s primary purpose is to empower scientists whose gene editing research is stifled by current technical and licensing limitations. They have made the MAD7™ CRISPR nuclease free for research purposes. Their technology incorporates specific biosecurity practices for ethical genome engineering
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