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filingDate 2020-04-29-04:00^^<http://www.w3.org/2001/XMLSchema#date>
inventor http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_8a8012d510ff4a1a982da7ff9abebbba
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publicationDate 2021-12-16-04:00^^<http://www.w3.org/2001/XMLSchema#date>
publicationNumber AU-2020264438-A1
titleOfInvention Intrathecal and intravenous combination gene therapy for the treatment of infantile batten disease
abstract Methods for treating IBD or an IBD related disorder in a subject in need thereof are provided that comprise combined intrathecal administration of a polynucleotide comprising a CLN1 open reading frame and intravenous administration of the polynucleotide. The polynucleotide comprising the CLN1 open reading frame is a wild-type CLN1 polynucleotide. In another aspect, the polynucleotide comprising the CLN1 open reading frame comprises codon-optimized polynucleotide sequence of the polynucleotide or its complement and is codon-optimized for expression in a human cell.
priorityDate 2019-04-29-04:00^^<http://www.w3.org/2001/XMLSchema#date>
type http://data.epo.org/linked-data/def/patent/Publication

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