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publicationNumber US-10792343-B2
titleOfInvention Compositions and methods for treating MPSI
abstract A vector have an expression cassette containing have a hIDUA gene has a sequence of SEQ ID NO: 1 (FIG. 1 ) or a sequence at least about 95% identical thereto which encodes a functional human alpha-L-iduronidase is provided. The vector may be a production vector or a rAAV8. Also provided are compositions containing these vectors and methods of treating MPSI and the symptoms associated with Hurler, Hurler-Scheie and Scheie syndromes.
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http://rdf.ncbi.nlm.nih.gov/pubchem/compound/CID128746
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP79780
http://rdf.ncbi.nlm.nih.gov/pubchem/gene/GID537655
http://rdf.ncbi.nlm.nih.gov/pubchem/gene/GID11461
http://rdf.ncbi.nlm.nih.gov/pubchem/gene/GID30148
http://rdf.ncbi.nlm.nih.gov/pubchem/substance/SID447627639
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCQ08867
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP17108
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCO77620
http://rdf.ncbi.nlm.nih.gov/pubchem/gene/GID107840369
http://rdf.ncbi.nlm.nih.gov/pubchem/gene/GID12096
http://rdf.ncbi.nlm.nih.gov/pubchem/gene/GID25295
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCF1R0H8
http://rdf.ncbi.nlm.nih.gov/pubchem/anatomy/ANATOMYID11757
http://rdf.ncbi.nlm.nih.gov/pubchem/compound/CID86583360
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP23239
http://rdf.ncbi.nlm.nih.gov/pubchem/substance/SID226395888
http://rdf.ncbi.nlm.nih.gov/pubchem/compound/CID53398693
http://rdf.ncbi.nlm.nih.gov/pubchem/gene/GID100144460
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP83005
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCP68291
http://rdf.ncbi.nlm.nih.gov/pubchem/protein/ACCA1A4R0

Total number of triples: 328.