http://rdf.ncbi.nlm.nih.gov/pubchem/patent/AR-110786-A1
Outgoing Links
| Predicate | Object |
|---|---|
| assignee | http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_3f06161c8222218d91d43831cf9cc228 |
| classificationIPCInventive | http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61P25-28 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61K38-43 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61K38-00 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61K48-00 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61P25-00 http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/C12N5-09 |
| filingDate | 2018-01-30-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| publicationDate | 2019-05-02-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| publicationNumber | AR-110786-A1 |
| titleOfInvention | TREATMENT OF MUCOPOLISACARIDOSIS I WITH a-L-IDURONIDASA (IDUA) HUMAN GLUCOSILADA COMPLETELY HUMAN |
| abstract | Compositions and methods for the administration of a fully human glycosylated a-L-iduronidase (IDUA) (HuGly) to the cerebrospinal fluid of the central nervous system (CNS) of a human subject with a diagnosis of mucopolysaccharidosis I (MPS I). Claim 1: A method for treating a human subject with a diagnosis of mucopolysaccharidosis I (MPS I), characterized in that it comprises delivering to the cerebrospinal fluid of the brain of said human subject a therapeutically effective amount of recombinant human aL-iduronidase (IDUA) produced by cells human neuronal Claim 2: A method for treating a human subject with a diagnosis of MPS I, characterized in that it comprises delivering therapeutically effective amount of recombinant human IDUA produced by human glial cells to the cerebrospinal fluid of the brain of said human subject. Claim 7: A method for the treatment of a human subject with a diagnosis of mucopolysaccharidosis I (MPS I), characterized in that it comprises: delivering to the cerebrospinal fluid of the brain of said human subject a therapeutically effective amount of a human IDUA containing tyrosine sulfation; and administering an immune suppression therapy to said subject before or at the same time as the treatment of human IDUA and then continuing the immune suppression therapy. Claim 8: A method for the treatment of a human subject with a diagnosis of mucopolysaccharidosis I (MPS I), characterized in that it comprises: administering to the brain of said human subject an expression vector encoding the human IDUA, wherein said IDUA is a2.6 -sialylated after the expression of said expression vector in an immortalized human neuronal cell; and administering an immune suppression therapy to said subject before or at the same time as the administration of the expression vector and then continuing the immune suppression therapy. Claim 12: A method for the treatment of a human subject with a diagnosis of mucopolysaccharidosis I (MPS I), characterized in that it comprises: administering to the cerebrospinal fluid of the brain of said human subject a therapeutically effective amount of a recombinant nucleotide expression vector encoding the human IDUA, so that a deposit is formed that releases said IDUA containing an a2,6-sialylated glycan; and administering an immune suppression therapy to said subject before or at the same time as the administration of the expression vector and then continuing the immune suppression therapy. Claim 16: The method of any one of claims 3 to 15, characterized in that the immune suppression therapy comprises administering a combination of (a) tacrolimus and mycophenolic acid, (b) rapamycin and mycophenolic acid or (c) tacrolimus, rapamycin and a corticosteroid such as prednisolone and / or methylprednisolone to said subject before or at the same time as the treatment of human IDUA and then continue. |
| priorityDate | 2017-01-31-04:00^^<http://www.w3.org/2001/XMLSchema#date> |
| type | http://data.epo.org/linked-data/def/patent/Publication |
Incoming Links
Total number of triples: 26.