http://rdf.ncbi.nlm.nih.gov/pubchem/patent/WO-2022120094-A2

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filingDate 2021-12-02-04:00^^<http://www.w3.org/2001/XMLSchema#date>
inventor http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_2c8f3ab915bca10c91ab412d736a83c4
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publicationDate 2022-06-09-04:00^^<http://www.w3.org/2001/XMLSchema#date>
publicationNumber WO-2022120094-A2
titleOfInvention Compositions and methods for the targeting of bcl11a
abstract Provided herein are systems comprising Class 2, Type V CRISPR polypeptides, guide nucleic acids (gNA), and optionally donor template nucleic acids useful in the modification of a BCL11A gene. The systems are also useful for the modification of cells in subjects with a hemoglobinopathy-related disease. Also provided are methods of treatment of subjects having a hemoglobinopathy-related disease by administration of the systems or nucleic acids encoding such systems that target the BCL11A gene in such subjects.
priorityDate 2020-12-03-04:00^^<http://www.w3.org/2001/XMLSchema#date>
type http://data.epo.org/linked-data/def/patent/Publication

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