Predicate |
Object |
assignee |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_dabaf4d71bff5405130eead7b7cd88f7 |
classificationCPCAdditional |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N2310-20 |
classificationCPCInventive |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/C12N15-113 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K35-545 http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K35-28 |
filingDate |
2021-12-02-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
inventor |
http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_2c8f3ab915bca10c91ab412d736a83c4 http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_c401c73e183bf06861cb67067c4d0498 http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_b965947e77b7e4635a4a51b06fc35758 http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_c892ed125a286c54c89c7a90f85949e6 http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_ec02ea9401e485b1bdf1e4dd90dcc324 http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_df6bf4c824978cce13ffc1dd1d5fb1aa http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_f18e34840bb162b9decc52f2c192e962 |
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 |