CN101404927A - 慢速脑室内递送 - Google Patents
慢速脑室内递送 Download PDFInfo
- Publication number
- CN101404927A CN101404927A CNA2007800098972A CN200780009897A CN101404927A CN 101404927 A CN101404927 A CN 101404927A CN A2007800098972 A CNA2007800098972 A CN A2007800098972A CN 200780009897 A CN200780009897 A CN 200780009897A CN 101404927 A CN101404927 A CN 101404927A
- Authority
- CN
- China
- Prior art keywords
- agent
- patient
- brain
- cerebrospinal fluid
- rate
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Pending
Links
- 238000007914 intraventricular administration Methods 0.000 title abstract description 25
- 210000004556 brain Anatomy 0.000 claims abstract description 69
- 239000003814 drug Substances 0.000 claims abstract description 59
- 208000015439 Lysosomal storage disease Diseases 0.000 claims abstract description 15
- 229940124597 therapeutic agent Drugs 0.000 claims abstract description 8
- 229940079593 drug Drugs 0.000 claims abstract 7
- 238000000034 method Methods 0.000 claims description 75
- 210000001175 cerebrospinal fluid Anatomy 0.000 claims description 45
- 239000003795 chemical substances by application Substances 0.000 claims description 42
- 102000004190 Enzymes Human genes 0.000 claims description 28
- 108090000790 Enzymes Proteins 0.000 claims description 28
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 23
- 201000010099 disease Diseases 0.000 claims description 22
- 108010061312 Sphingomyelin Phosphodiesterase Proteins 0.000 claims description 16
- 230000002950 deficient Effects 0.000 claims description 9
- 210000002966 serum Anatomy 0.000 claims description 9
- 208000011580 syndromic disease Diseases 0.000 claims description 9
- 210000000278 spinal cord Anatomy 0.000 claims description 8
- 210000002330 subarachnoid space Anatomy 0.000 claims description 6
- 208000015872 Gaucher disease Diseases 0.000 claims description 4
- 201000000791 Niemann-Pick disease type B Diseases 0.000 claims description 4
- 208000008291 Type B Niemann-Pick Disease Diseases 0.000 claims description 4
- 210000004055 fourth ventricle Anatomy 0.000 claims description 4
- 108010028144 alpha-Glucosidases Proteins 0.000 claims description 3
- 239000002872 contrast media Substances 0.000 claims description 3
- 102000011971 Sphingomyelin Phosphodiesterase Human genes 0.000 claims description 2
- 208000022292 Tay-Sachs disease Diseases 0.000 claims description 2
- 108010030291 alpha-Galactosidase Proteins 0.000 claims description 2
- 102000005840 alpha-Galactosidase Human genes 0.000 claims description 2
- 210000003140 lateral ventricle Anatomy 0.000 claims 5
- 102100033448 Lysosomal alpha-glucosidase Human genes 0.000 claims 2
- 239000012216 imaging agent Substances 0.000 claims 2
- 102100022548 Beta-hexosaminidase subunit alpha Human genes 0.000 claims 1
- 208000035374 Chronic visceral acid sphingomyelinase deficiency Diseases 0.000 claims 1
- 208000024720 Fabry Disease Diseases 0.000 claims 1
- 102000004547 Glucosylceramidase Human genes 0.000 claims 1
- 108010017544 Glucosylceramidase Proteins 0.000 claims 1
- 208000032007 Glycogen storage disease due to acid maltase deficiency Diseases 0.000 claims 1
- 206010053185 Glycogen storage disease type II Diseases 0.000 claims 1
- 102000002268 Hexosaminidases Human genes 0.000 claims 1
- 108010000540 Hexosaminidases Proteins 0.000 claims 1
- 230000003444 anaesthetic effect Effects 0.000 claims 1
- 210000003037 cerebral aqueduct Anatomy 0.000 claims 1
- 238000012632 fluorescent imaging Methods 0.000 claims 1
- 201000004502 glycogen storage disease II Diseases 0.000 claims 1
- 239000011148 porous material Substances 0.000 claims 1
- 230000002285 radioactive effect Effects 0.000 claims 1
- 210000000211 third ventricle Anatomy 0.000 claims 1
- 230000000694 effects Effects 0.000 abstract description 8
- 230000008499 blood brain barrier function Effects 0.000 abstract description 5
- 210000001218 blood-brain barrier Anatomy 0.000 abstract description 5
- 239000000032 diagnostic agent Substances 0.000 abstract description 4
- 208000012902 Nervous system disease Diseases 0.000 abstract description 2
- 208000025966 Neurological disease Diseases 0.000 abstract 1
- 230000000857 drug effect Effects 0.000 abstract 1
- 239000003193 general anesthetic agent Substances 0.000 abstract 1
- 108090000623 proteins and genes Proteins 0.000 description 46
- 238000001802 infusion Methods 0.000 description 42
- 210000004027 cell Anatomy 0.000 description 28
- 229940088598 enzyme Drugs 0.000 description 26
- 241000700605 Viruses Species 0.000 description 25
- 241000701161 unidentified adenovirus Species 0.000 description 24
- 239000013598 vector Substances 0.000 description 19
- 241000699670 Mus sp. Species 0.000 description 17
- 102000010126 acid sphingomyelin phosphodiesterase activity proteins Human genes 0.000 description 14
- 238000011282 treatment Methods 0.000 description 14
- 241000699666 Mus <mouse, genus> Species 0.000 description 12
- 208000014060 Niemann-Pick disease Diseases 0.000 description 12
- HVYWMOMLDIMFJA-DPAQBDIFSA-N cholesterol Chemical compound C1C=C2C[C@@H](O)CC[C@]2(C)[C@@H]2[C@@H]1[C@@H]1CC[C@H]([C@H](C)CCCC(C)C)[C@@]1(C)CC2 HVYWMOMLDIMFJA-DPAQBDIFSA-N 0.000 description 10
- 241000725303 Human immunodeficiency virus Species 0.000 description 9
- 210000004185 liver Anatomy 0.000 description 9
- 238000012360 testing method Methods 0.000 description 9
- 102100021244 Integral membrane protein GPR180 Human genes 0.000 description 8
- 230000008901 benefit Effects 0.000 description 8
- 230000034994 death Effects 0.000 description 8
- 210000004072 lung Anatomy 0.000 description 8
- 230000002132 lysosomal effect Effects 0.000 description 8
- 235000018102 proteins Nutrition 0.000 description 8
- 102000004169 proteins and genes Human genes 0.000 description 8
- 102000004157 Hydrolases Human genes 0.000 description 7
- 108090000604 Hydrolases Proteins 0.000 description 7
- 210000003712 lysosome Anatomy 0.000 description 7
- 230000001868 lysosomic effect Effects 0.000 description 7
- 239000000758 substrate Substances 0.000 description 7
- 238000012546 transfer Methods 0.000 description 7
- 241001430294 unidentified retrovirus Species 0.000 description 7
- 230000003612 virological effect Effects 0.000 description 7
- 210000001835 viscera Anatomy 0.000 description 7
- 108091032973 (ribonucleotides)n+m Proteins 0.000 description 6
- 108020004414 DNA Proteins 0.000 description 6
- WSFSSNUMVMOOMR-UHFFFAOYSA-N Formaldehyde Chemical group O=C WSFSSNUMVMOOMR-UHFFFAOYSA-N 0.000 description 6
- 241000282414 Homo sapiens Species 0.000 description 6
- 210000003169 central nervous system Anatomy 0.000 description 6
- 238000002513 implantation Methods 0.000 description 6
- 230000010354 integration Effects 0.000 description 6
- 230000004060 metabolic process Effects 0.000 description 6
- 239000006187 pill Substances 0.000 description 6
- 210000001519 tissue Anatomy 0.000 description 6
- 239000002253 acid Substances 0.000 description 5
- 235000012000 cholesterol Nutrition 0.000 description 5
- 210000000349 chromosome Anatomy 0.000 description 5
- 238000002347 injection Methods 0.000 description 5
- 239000007924 injection Substances 0.000 description 5
- 239000000203 mixture Substances 0.000 description 5
- 108020004707 nucleic acids Proteins 0.000 description 5
- 102000039446 nucleic acids Human genes 0.000 description 5
- 150000007523 nucleic acids Chemical class 0.000 description 5
- 239000002773 nucleotide Substances 0.000 description 5
- 125000003729 nucleotide group Chemical group 0.000 description 5
- 210000000056 organ Anatomy 0.000 description 5
- 238000012856 packing Methods 0.000 description 5
- 239000002574 poison Substances 0.000 description 5
- 231100000614 poison Toxicity 0.000 description 5
- 210000000952 spleen Anatomy 0.000 description 5
- 230000029812 viral genome replication Effects 0.000 description 5
- 210000002845 virion Anatomy 0.000 description 5
- 208000031277 Amaurotic familial idiocy Diseases 0.000 description 4
- 208000002537 Neuronal Ceroid-Lipofuscinoses Diseases 0.000 description 4
- FAPWRFPIFSIZLT-UHFFFAOYSA-M Sodium chloride Chemical compound [Na+].[Cl-] FAPWRFPIFSIZLT-UHFFFAOYSA-M 0.000 description 4
- 210000000576 arachnoid Anatomy 0.000 description 4
- 230000001413 cellular effect Effects 0.000 description 4
- 230000002490 cerebral effect Effects 0.000 description 4
- 238000012217 deletion Methods 0.000 description 4
- 230000037430 deletion Effects 0.000 description 4
- 208000015181 infectious disease Diseases 0.000 description 4
- 208000017476 juvenile neuronal ceroid lipofuscinosis Diseases 0.000 description 4
- 210000005036 nerve Anatomy 0.000 description 4
- 201000007607 neuronal ceroid lipofuscinosis 3 Diseases 0.000 description 4
- 238000002360 preparation method Methods 0.000 description 4
- 230000001177 retroviral effect Effects 0.000 description 4
- 238000010839 reverse transcription Methods 0.000 description 4
- 208000030507 AIDS Diseases 0.000 description 3
- LFQSCWFLJHTTHZ-UHFFFAOYSA-N Ethanol Chemical compound CCO LFQSCWFLJHTTHZ-UHFFFAOYSA-N 0.000 description 3
- 150000001413 amino acids Chemical class 0.000 description 3
- 210000004369 blood Anatomy 0.000 description 3
- 239000008280 blood Substances 0.000 description 3
- 230000037396 body weight Effects 0.000 description 3
- 230000007547 defect Effects 0.000 description 3
- 229940039227 diagnostic agent Drugs 0.000 description 3
- 238000009826 distribution Methods 0.000 description 3
- 238000005516 engineering process Methods 0.000 description 3
- 238000002641 enzyme replacement therapy Methods 0.000 description 3
- 230000006870 function Effects 0.000 description 3
- 230000002068 genetic effect Effects 0.000 description 3
- 239000000463 material Substances 0.000 description 3
- 239000003921 oil Substances 0.000 description 3
- 235000019198 oils Nutrition 0.000 description 3
- 230000036285 pathological change Effects 0.000 description 3
- 231100000915 pathological change Toxicity 0.000 description 3
- 210000001202 rhombencephalon Anatomy 0.000 description 3
- 230000009261 transgenic effect Effects 0.000 description 3
- 210000003462 vein Anatomy 0.000 description 3
- 239000013603 viral vector Substances 0.000 description 3
- XLYOFNOQVPJJNP-UHFFFAOYSA-N water Substances O XLYOFNOQVPJJNP-UHFFFAOYSA-N 0.000 description 3
- CIWBSHSKHKDKBQ-JLAZNSOCSA-N Ascorbic acid Chemical compound OC[C@H](O)[C@H]1OC(=O)C(O)=C1O CIWBSHSKHKDKBQ-JLAZNSOCSA-N 0.000 description 2
- 102100026189 Beta-galactosidase Human genes 0.000 description 2
- 241000713704 Bovine immunodeficiency virus Species 0.000 description 2
- 101150044789 Cap gene Proteins 0.000 description 2
- 241000557626 Corvus corax Species 0.000 description 2
- 241000702421 Dependoparvovirus Species 0.000 description 2
- WQZGKKKJIJFFOK-GASJEMHNSA-N Glucose Natural products OC[C@H]1OC(O)[C@H](O)[C@@H](O)[C@@H]1O WQZGKKKJIJFFOK-GASJEMHNSA-N 0.000 description 2
- WHUUTDBJXJRKMK-UHFFFAOYSA-N Glutamic acid Natural products OC(=O)C(N)CCC(O)=O WHUUTDBJXJRKMK-UHFFFAOYSA-N 0.000 description 2
- UXDDRFCJKNROTO-UHFFFAOYSA-N Glycerol 1,2-diacetate Chemical compound CC(=O)OCC(CO)OC(C)=O UXDDRFCJKNROTO-UHFFFAOYSA-N 0.000 description 2
- 208000028782 Hereditary disease Diseases 0.000 description 2
- VEXZGXHMUGYJMC-UHFFFAOYSA-N Hydrochloric acid Chemical compound Cl VEXZGXHMUGYJMC-UHFFFAOYSA-N 0.000 description 2
- 241000713666 Lentivirus Species 0.000 description 2
- 108090000856 Lyases Proteins 0.000 description 2
- 102000004317 Lyases Human genes 0.000 description 2
- 206010028095 Mucopolysaccharidosis IV Diseases 0.000 description 2
- 241000714177 Murine leukemia virus Species 0.000 description 2
- 108010077850 Nuclear Localization Signals Proteins 0.000 description 2
- 101710087110 ORF6 protein Proteins 0.000 description 2
- ISWSIDIOOBJBQZ-UHFFFAOYSA-N Phenol Chemical compound OC1=CC=CC=C1 ISWSIDIOOBJBQZ-UHFFFAOYSA-N 0.000 description 2
- 101100401683 Schizosaccharomyces pombe (strain 972 / ATCC 24843) mis13 gene Proteins 0.000 description 2
- 241000700584 Simplexvirus Species 0.000 description 2
- 102100026263 Sphingomyelin phosphodiesterase Human genes 0.000 description 2
- 101710095001 Uncharacterized protein in nifU 5'region Proteins 0.000 description 2
- ZSLZBFCDCINBPY-ZSJPKINUSA-N acetyl-CoA Chemical compound O[C@@H]1[C@H](OP(O)(O)=O)[C@@H](COP(O)(=O)OP(O)(=O)OCC(C)(C)[C@@H](O)C(=O)NCCC(=O)NCCSC(=O)C)O[C@H]1N1C2=NC=NC(N)=C2N=C1 ZSLZBFCDCINBPY-ZSJPKINUSA-N 0.000 description 2
- 108010070626 acid beta-galactosidase Proteins 0.000 description 2
- 235000001014 amino acid Nutrition 0.000 description 2
- 238000004458 analytical method Methods 0.000 description 2
- 108010005774 beta-Galactosidase Proteins 0.000 description 2
- 230000033228 biological regulation Effects 0.000 description 2
- 239000000872 buffer Substances 0.000 description 2
- 210000000234 capsid Anatomy 0.000 description 2
- 238000006555 catalytic reaction Methods 0.000 description 2
- 238000006243 chemical reaction Methods 0.000 description 2
- 238000011109 contamination Methods 0.000 description 2
- 230000003013 cytotoxicity Effects 0.000 description 2
- 231100000135 cytotoxicity Toxicity 0.000 description 2
- 238000011161 development Methods 0.000 description 2
- 238000003745 diagnosis Methods 0.000 description 2
- VYFYYTLLBUKUHU-UHFFFAOYSA-N dopamine Chemical compound NCCC1=CC=C(O)C(O)=C1 VYFYYTLLBUKUHU-UHFFFAOYSA-N 0.000 description 2
- 238000012377 drug delivery Methods 0.000 description 2
- 238000004043 dyeing Methods 0.000 description 2
- 150000002148 esters Chemical class 0.000 description 2
- 239000003925 fat Substances 0.000 description 2
- 229930182830 galactose Natural products 0.000 description 2
- 238000001415 gene therapy Methods 0.000 description 2
- 239000008103 glucose Substances 0.000 description 2
- 235000013922 glutamic acid Nutrition 0.000 description 2
- 239000004220 glutamic acid Substances 0.000 description 2
- LNEPOXFFQSENCJ-UHFFFAOYSA-N haloperidol Chemical compound C1CC(O)(C=2C=CC(Cl)=CC=2)CCN1CCCC(=O)C1=CC=C(F)C=C1 LNEPOXFFQSENCJ-UHFFFAOYSA-N 0.000 description 2
- 238000001727 in vivo Methods 0.000 description 2
- CGIGDMFJXJATDK-UHFFFAOYSA-N indomethacin Chemical compound CC1=C(CC(O)=O)C2=CC(OC)=CC=C2N1C(=O)C1=CC=C(Cl)C=C1 CGIGDMFJXJATDK-UHFFFAOYSA-N 0.000 description 2
- 230000006698 induction Effects 0.000 description 2
- 230000002458 infectious effect Effects 0.000 description 2
- 210000003734 kidney Anatomy 0.000 description 2
- 210000002540 macrophage Anatomy 0.000 description 2
- 208000030159 metabolic disease Diseases 0.000 description 2
- 230000002503 metabolic effect Effects 0.000 description 2
- VYQNWZOUAUKGHI-UHFFFAOYSA-N monobenzone Chemical compound C1=CC(O)=CC=C1OCC1=CC=CC=C1 VYQNWZOUAUKGHI-UHFFFAOYSA-N 0.000 description 2
- BQJCRHHNABKAKU-KBQPJGBKSA-N morphine Chemical compound O([C@H]1[C@H](C=C[C@H]23)O)C4=C5[C@@]12CCN(C)[C@@H]3CC5=CC=C4O BQJCRHHNABKAKU-KBQPJGBKSA-N 0.000 description 2
- -1 muscinol Chemical compound 0.000 description 2
- 229950006780 n-acetylglucosamine Drugs 0.000 description 2
- 238000004806 packaging method and process Methods 0.000 description 2
- 230000001717 pathogenic effect Effects 0.000 description 2
- 230000007170 pathology Effects 0.000 description 2
- YHHSONZFOIEMCP-UHFFFAOYSA-O phosphocholine Chemical compound C[N+](C)(C)CCOP(O)(O)=O YHHSONZFOIEMCP-UHFFFAOYSA-O 0.000 description 2
- 239000013600 plasmid vector Substances 0.000 description 2
- 108090000765 processed proteins & peptides Proteins 0.000 description 2
- 239000000047 product Substances 0.000 description 2
- 210000004129 prosencephalon Anatomy 0.000 description 2
- 239000013608 rAAV vector Substances 0.000 description 2
- 230000008521 reorganization Effects 0.000 description 2
- 101150066583 rep gene Proteins 0.000 description 2
- 239000000523 sample Substances 0.000 description 2
- 230000028327 secretion Effects 0.000 description 2
- 238000003860 storage Methods 0.000 description 2
- 238000002560 therapeutic procedure Methods 0.000 description 2
- 238000013518 transcription Methods 0.000 description 2
- 230000035897 transcription Effects 0.000 description 2
- 238000001890 transfection Methods 0.000 description 2
- WWUZIQQURGPMPG-UHFFFAOYSA-N (-)-D-erythro-Sphingosine Natural products CCCCCCCCCCCCCC=CC(O)C(N)CO WWUZIQQURGPMPG-UHFFFAOYSA-N 0.000 description 1
- 102000040650 (ribonucleotides)n+m Human genes 0.000 description 1
- RRTQTOBOLIGMED-UHFFFAOYSA-N 2-(carboxyamino)-2-phenylacetic acid Chemical compound OC(=O)NC(C(O)=O)C1=CC=CC=C1 RRTQTOBOLIGMED-UHFFFAOYSA-N 0.000 description 1
- LRYZPFWEZHSTHD-HEFFAWAOSA-O 2-[[(e,2s,3r)-2-formamido-3-hydroxyoctadec-4-enoxy]-hydroxyphosphoryl]oxyethyl-trimethylazanium Chemical class CCCCCCCCCCCCC\C=C\[C@@H](O)[C@@H](NC=O)COP(O)(=O)OCC[N+](C)(C)C LRYZPFWEZHSTHD-HEFFAWAOSA-O 0.000 description 1
- JYLNVJYYQQXNEK-UHFFFAOYSA-N 3-amino-2-(4-chlorophenyl)-1-propanesulfonic acid Chemical compound OS(=O)(=O)CC(CN)C1=CC=C(Cl)C=C1 JYLNVJYYQQXNEK-UHFFFAOYSA-N 0.000 description 1
- 102000006772 Acid Ceramidase Human genes 0.000 description 1
- 108020005296 Acid Ceramidase Proteins 0.000 description 1
- 101100524317 Adeno-associated virus 2 (isolate Srivastava/1982) Rep40 gene Proteins 0.000 description 1
- 101100524319 Adeno-associated virus 2 (isolate Srivastava/1982) Rep52 gene Proteins 0.000 description 1
- 101100524321 Adeno-associated virus 2 (isolate Srivastava/1982) Rep68 gene Proteins 0.000 description 1
- 206010002091 Anaesthesia Diseases 0.000 description 1
- 241000153246 Anteros Species 0.000 description 1
- 108020005544 Antisense RNA Proteins 0.000 description 1
- 102100031491 Arylsulfatase B Human genes 0.000 description 1
- 206010068220 Aspartylglucosaminuria Diseases 0.000 description 1
- KPYSYYIEGFHWSV-UHFFFAOYSA-N Baclofen Chemical compound OC(=O)CC(CN)C1=CC=C(Cl)C=C1 KPYSYYIEGFHWSV-UHFFFAOYSA-N 0.000 description 1
- 101710124976 Beta-hexosaminidase A Proteins 0.000 description 1
- 101710124978 Beta-hexosaminidase B Proteins 0.000 description 1
- 102100032487 Beta-mannosidase Human genes 0.000 description 1
- 208000014644 Brain disease Diseases 0.000 description 1
- YDNKGFDKKRUKPY-JHOUSYSJSA-N C16 ceramide Natural products CCCCCCCCCCCCCCCC(=O)N[C@@H](CO)[C@H](O)C=CCCCCCCCCCCCCC YDNKGFDKKRUKPY-JHOUSYSJSA-N 0.000 description 1
- 101100080277 Caenorhabditis elegans ncr-1 gene Proteins 0.000 description 1
- 102000014914 Carrier Proteins Human genes 0.000 description 1
- 108010078791 Carrier Proteins Proteins 0.000 description 1
- 102000005572 Cathepsin A Human genes 0.000 description 1
- 108010059081 Cathepsin A Proteins 0.000 description 1
- LRBQNJMCXXYXIU-PPKXGCFTSA-N Chinese gallotannin Chemical compound OC1=C(O)C(O)=CC(C(=O)OC=2C(=C(O)C=C(C=2)C(=O)OC[C@@H]2[C@H]([C@H](OC(=O)C=3C=C(OC(=O)C=4C=C(O)C(O)=C(O)C=4)C(O)=C(O)C=3)[C@@H](OC(=O)C=3C=C(OC(=O)C=4C=C(O)C(O)=C(O)C=4)C(O)=C(O)C=3)[C@H](OC(=O)C=3C=C(OC(=O)C=4C=C(O)C(O)=C(O)C=4)C(O)=C(O)C=3)O2)OC(=O)C=2C=C(OC(=O)C=3C=C(O)C(O)=C(O)C=3)C(O)=C(O)C=2)O)=C1 LRBQNJMCXXYXIU-PPKXGCFTSA-N 0.000 description 1
- 206010011777 Cystinosis Diseases 0.000 description 1
- DYDCUQKUCUHJBH-UWTATZPHSA-N D-Cycloserine Chemical compound N[C@@H]1CONC1=O DYDCUQKUCUHJBH-UWTATZPHSA-N 0.000 description 1
- DYDCUQKUCUHJBH-UHFFFAOYSA-N D-Cycloserine Natural products NC1CONC1=O DYDCUQKUCUHJBH-UHFFFAOYSA-N 0.000 description 1
- FBPFZTCFMRRESA-FSIIMWSLSA-N D-Glucitol Natural products OC[C@H](O)[C@H](O)[C@@H](O)[C@H](O)CO FBPFZTCFMRRESA-FSIIMWSLSA-N 0.000 description 1
- FBPFZTCFMRRESA-KVTDHHQDSA-N D-Mannitol Chemical compound OC[C@@H](O)[C@@H](O)[C@H](O)[C@H](O)CO FBPFZTCFMRRESA-KVTDHHQDSA-N 0.000 description 1
- FBPFZTCFMRRESA-JGWLITMVSA-N D-glucitol Chemical compound OC[C@H](O)[C@@H](O)[C@H](O)[C@H](O)CO FBPFZTCFMRRESA-JGWLITMVSA-N 0.000 description 1
- 230000004543 DNA replication Effects 0.000 description 1
- 241000450599 DNA viruses Species 0.000 description 1
- 108010014303 DNA-directed DNA polymerase Proteins 0.000 description 1
- 102000016928 DNA-directed DNA polymerase Human genes 0.000 description 1
- 208000032274 Encephalopathy Diseases 0.000 description 1
- 108700039887 Essential Genes Proteins 0.000 description 1
- 208000001948 Farber Lipogranulomatosis Diseases 0.000 description 1
- 201000011240 Frontotemporal dementia Diseases 0.000 description 1
- 201000008892 GM1 Gangliosidosis Diseases 0.000 description 1
- 208000017462 Galactosialidosis Diseases 0.000 description 1
- 208000034826 Genetic Predisposition to Disease Diseases 0.000 description 1
- 208000010055 Globoid Cell Leukodystrophy Diseases 0.000 description 1
- 102000053187 Glucuronidase Human genes 0.000 description 1
- 108010060309 Glucuronidase Proteins 0.000 description 1
- 208000031886 HIV Infections Diseases 0.000 description 1
- 101000923070 Homo sapiens Arylsulfatase B Proteins 0.000 description 1
- 241000598171 Human adenovirus sp. Species 0.000 description 1
- 241000713772 Human immunodeficiency virus 1 Species 0.000 description 1
- 241000713340 Human immunodeficiency virus 2 Species 0.000 description 1
- 208000023105 Huntington disease Diseases 0.000 description 1
- 208000026350 Inborn Genetic disease Diseases 0.000 description 1
- 206010021703 Indifference Diseases 0.000 description 1
- 102100034349 Integrase Human genes 0.000 description 1
- YQEZLKZALYSWHR-UHFFFAOYSA-N Ketamine Chemical compound C=1C=CC=C(Cl)C=1C1(NC)CCCCC1=O YQEZLKZALYSWHR-UHFFFAOYSA-N 0.000 description 1
- WHUUTDBJXJRKMK-VKHMYHEASA-N L-glutamic acid Chemical compound OC(=O)[C@@H](N)CCC(O)=O WHUUTDBJXJRKMK-VKHMYHEASA-N 0.000 description 1
- MKXZASYAUGDDCJ-SZMVWBNQSA-N LSM-2525 Chemical compound C1CCC[C@H]2[C@@]3([H])N(C)CC[C@]21C1=CC(OC)=CC=C1C3 MKXZASYAUGDDCJ-SZMVWBNQSA-N 0.000 description 1
- 208000032420 Latent Infection Diseases 0.000 description 1
- NNJVILVZKWQKPM-UHFFFAOYSA-N Lidocaine Chemical compound CCN(CC)CC(=O)NC1=C(C)C=CC=C1C NNJVILVZKWQKPM-UHFFFAOYSA-N 0.000 description 1
- 102000004882 Lipase Human genes 0.000 description 1
- 108090001060 Lipase Proteins 0.000 description 1
- 208000008771 Lymphadenopathy Diseases 0.000 description 1
- 102100024295 Maltase-glucoamylase Human genes 0.000 description 1
- 229930195725 Mannitol Natural products 0.000 description 1
- 208000024556 Mendelian disease Diseases 0.000 description 1
- 206010027336 Menstruation delayed Diseases 0.000 description 1
- 208000036626 Mental retardation Diseases 0.000 description 1
- 201000011442 Metachromatic leukodystrophy Diseases 0.000 description 1
- 208000008955 Mucolipidoses Diseases 0.000 description 1
- 206010072927 Mucolipidosis type I Diseases 0.000 description 1
- CRJGESKKUOMBCT-VQTJNVASSA-N N-acetylsphinganine Chemical compound CCCCCCCCCCCCCCC[C@@H](O)[C@H](CO)NC(C)=O CRJGESKKUOMBCT-VQTJNVASSA-N 0.000 description 1
- 108700019961 Neoplasm Genes Proteins 0.000 description 1
- 102000048850 Neoplasm Genes Human genes 0.000 description 1
- 206010029260 Neuroblastoma Diseases 0.000 description 1
- 101100459404 Neurospora crassa (strain ATCC 24698 / 74-OR23-1A / CBS 708.71 / DSM 1257 / FGSC 987) npc-1 gene Proteins 0.000 description 1
- 108091093105 Nuclear DNA Proteins 0.000 description 1
- 229910019142 PO4 Inorganic materials 0.000 description 1
- 208000002193 Pain Diseases 0.000 description 1
- 208000018737 Parkinson disease Diseases 0.000 description 1
- QGMRQYFBGABWDR-UHFFFAOYSA-M Pentobarbital sodium Chemical compound [Na+].CCCC(C)C1(CC)C(=O)NC(=O)[N-]C1=O QGMRQYFBGABWDR-UHFFFAOYSA-M 0.000 description 1
- 102000035195 Peptidases Human genes 0.000 description 1
- 108091005804 Peptidases Proteins 0.000 description 1
- 241000288049 Perdix perdix Species 0.000 description 1
- 208000000609 Pick Disease of the Brain Diseases 0.000 description 1
- 208000024571 Pick disease Diseases 0.000 description 1
- 208000012641 Pigmentation disease Diseases 0.000 description 1
- 229920002685 Polyoxyl 35CastorOil Polymers 0.000 description 1
- 241000288906 Primates Species 0.000 description 1
- 108010076504 Protein Sorting Signals Proteins 0.000 description 1
- 241000125945 Protoparvovirus Species 0.000 description 1
- 108010092799 RNA-directed DNA polymerase Proteins 0.000 description 1
- 101100449397 Rattus norvegicus Gpr6 gene Proteins 0.000 description 1
- 206010038997 Retroviral infections Diseases 0.000 description 1
- 241000283984 Rodentia Species 0.000 description 1
- 101100058498 Saccharomyces cerevisiae (strain ATCC 204508 / S288c) CNL1 gene Proteins 0.000 description 1
- 101100294209 Schizosaccharomyces pombe (strain 972 / ATCC 24843) cnl2 gene Proteins 0.000 description 1
- 108020004459 Small interfering RNA Proteins 0.000 description 1
- 101710172711 Structural protein Proteins 0.000 description 1
- 206010042566 Superinfection Diseases 0.000 description 1
- 210000001744 T-lymphocyte Anatomy 0.000 description 1
- 239000004098 Tetracycline Substances 0.000 description 1
- 102000014384 Type C Phospholipases Human genes 0.000 description 1
- 108010079194 Type C Phospholipases Proteins 0.000 description 1
- 108010003533 Viral Envelope Proteins Proteins 0.000 description 1
- 108700005077 Viral Genes Proteins 0.000 description 1
- 108020000999 Viral RNA Proteins 0.000 description 1
- 101710165741 Virion-associated protein Proteins 0.000 description 1
- 206010048214 Xanthoma Diseases 0.000 description 1
- 206010048215 Xanthomatosis Diseases 0.000 description 1
- 229940100228 acetyl coenzyme a Drugs 0.000 description 1
- 208000005298 acute pain Diseases 0.000 description 1
- 208000013228 adenopathy Diseases 0.000 description 1
- 239000002671 adjuvant Substances 0.000 description 1
- 239000000556 agonist Substances 0.000 description 1
- UPEZCKBFRMILAV-UHFFFAOYSA-N alpha-Ecdysone Natural products C1C(O)C(O)CC2(C)C(CCC3(C(C(C(O)CCC(C)(C)O)C)CCC33O)C)C3=CC(=O)C21 UPEZCKBFRMILAV-UHFFFAOYSA-N 0.000 description 1
- 108010012864 alpha-Mannosidase Proteins 0.000 description 1
- 102000019199 alpha-Mannosidase Human genes 0.000 description 1
- 201000008333 alpha-mannosidosis Diseases 0.000 description 1
- 230000037005 anaesthesia Effects 0.000 description 1
- 238000010171 animal model Methods 0.000 description 1
- 239000010775 animal oil Substances 0.000 description 1
- 239000005557 antagonist Substances 0.000 description 1
- 239000003242 anti bacterial agent Substances 0.000 description 1
- 230000000844 anti-bacterial effect Effects 0.000 description 1
- 230000000843 anti-fungal effect Effects 0.000 description 1
- 239000002260 anti-inflammatory agent Substances 0.000 description 1
- 229940121363 anti-inflammatory agent Drugs 0.000 description 1
- 229940088710 antibiotic agent Drugs 0.000 description 1
- 229940121375 antifungal agent Drugs 0.000 description 1
- 239000000427 antigen Substances 0.000 description 1
- 108091007433 antigens Proteins 0.000 description 1
- 102000036639 antigens Human genes 0.000 description 1
- NOFOAYPPHIUXJR-APNQCZIXSA-N aphidicolin Chemical compound C1[C@@]23[C@@]4(C)CC[C@@H](O)[C@@](C)(CO)[C@@H]4CC[C@H]3C[C@H]1[C@](CO)(O)CC2 NOFOAYPPHIUXJR-APNQCZIXSA-N 0.000 description 1
- SEKZNWAQALMJNH-YZUCACDQSA-N aphidicolin Natural products C[C@]1(CO)CC[C@]23C[C@H]1C[C@@H]2CC[C@H]4[C@](C)(CO)[C@H](O)CC[C@]34C SEKZNWAQALMJNH-YZUCACDQSA-N 0.000 description 1
- 238000013459 approach Methods 0.000 description 1
- 229960005070 ascorbic acid Drugs 0.000 description 1
- 235000010323 ascorbic acid Nutrition 0.000 description 1
- 239000011668 ascorbic acid Substances 0.000 description 1
- 229960000794 baclofen Drugs 0.000 description 1
- 230000003385 bacteriostatic effect Effects 0.000 description 1
- 238000013542 behavioral therapy Methods 0.000 description 1
- WQZGKKKJIJFFOK-VFUOTHLCSA-N beta-D-glucose Chemical compound OC[C@H]1O[C@@H](O)[C@H](O)[C@@H](O)[C@@H]1O WQZGKKKJIJFFOK-VFUOTHLCSA-N 0.000 description 1
- 108010055059 beta-Mannosidase Proteins 0.000 description 1
- 201000006486 beta-mannosidosis Diseases 0.000 description 1
- 238000012742 biochemical analysis Methods 0.000 description 1
- 238000010241 blood sampling Methods 0.000 description 1
- 210000004899 c-terminal region Anatomy 0.000 description 1
- 239000000969 carrier Substances 0.000 description 1
- 230000022131 cell cycle Effects 0.000 description 1
- 230000003915 cell function Effects 0.000 description 1
- 230000004663 cell proliferation Effects 0.000 description 1
- 230000005101 cell tropism Effects 0.000 description 1
- 229940106189 ceramide Drugs 0.000 description 1
- ZVEQCJWYRWKARO-UHFFFAOYSA-N ceramide Natural products CCCCCCCCCCCCCCC(O)C(=O)NC(CO)C(O)C=CCCC=C(C)CCCCCCCCC ZVEQCJWYRWKARO-UHFFFAOYSA-N 0.000 description 1
- 210000004289 cerebral ventricle Anatomy 0.000 description 1
- 229940049197 cerezyme Drugs 0.000 description 1
- 230000008859 change Effects 0.000 description 1
- OSASVXMJTNOKOY-UHFFFAOYSA-N chlorobutanol Chemical compound CC(C)(O)C(Cl)(Cl)Cl OSASVXMJTNOKOY-UHFFFAOYSA-N 0.000 description 1
- 239000013611 chromosomal DNA Substances 0.000 description 1
- 230000001684 chronic effect Effects 0.000 description 1
- 229940045896 cnl8 Drugs 0.000 description 1
- 208000010877 cognitive disease Diseases 0.000 description 1
- 230000003920 cognitive function Effects 0.000 description 1
- 239000003184 complementary RNA Substances 0.000 description 1
- 230000001276 controlling effect Effects 0.000 description 1
- 239000003255 cyclooxygenase 2 inhibitor Substances 0.000 description 1
- 229960003077 cycloserine Drugs 0.000 description 1
- 235000018417 cysteine Nutrition 0.000 description 1
- XUJNEKJLAYXESH-UHFFFAOYSA-N cysteine Natural products SCC(N)C(O)=O XUJNEKJLAYXESH-UHFFFAOYSA-N 0.000 description 1
- 230000009849 deactivation Effects 0.000 description 1
- 230000007812 deficiency Effects 0.000 description 1
- 230000008021 deposition Effects 0.000 description 1
- 238000013461 design Methods 0.000 description 1
- 229960001985 dextromethorphan Drugs 0.000 description 1
- 238000009792 diffusion process Methods 0.000 description 1
- 239000003085 diluting agent Substances 0.000 description 1
- 208000037771 disease arising from reactivation of latent virus Diseases 0.000 description 1
- 208000035475 disorder Diseases 0.000 description 1
- 239000006185 dispersion Substances 0.000 description 1
- 238000006073 displacement reaction Methods 0.000 description 1
- LBOJYSIDWZQNJS-CVEARBPZSA-N dizocilpine Chemical compound C12=CC=CC=C2[C@]2(C)C3=CC=CC=C3C[C@H]1N2 LBOJYSIDWZQNJS-CVEARBPZSA-N 0.000 description 1
- 229950004794 dizocilpine Drugs 0.000 description 1
- 229960003638 dopamine Drugs 0.000 description 1
- 231100000673 dose–response relationship Toxicity 0.000 description 1
- 239000003937 drug carrier Substances 0.000 description 1
- 241001493065 dsRNA viruses Species 0.000 description 1
- 239000000839 emulsion Substances 0.000 description 1
- 239000003623 enhancer Substances 0.000 description 1
- 108700004025 env Genes Proteins 0.000 description 1
- 101150030339 env gene Proteins 0.000 description 1
- 230000002255 enzymatic effect Effects 0.000 description 1
- 206010015037 epilepsy Diseases 0.000 description 1
- 238000011156 evaluation Methods 0.000 description 1
- 239000012530 fluid Substances 0.000 description 1
- 239000012634 fragment Substances 0.000 description 1
- 201000008049 fucosidosis Diseases 0.000 description 1
- 230000002538 fungal effect Effects 0.000 description 1
- 108700004026 gag Genes Proteins 0.000 description 1
- 101150098622 gag gene Proteins 0.000 description 1
- 210000004907 gland Anatomy 0.000 description 1
- 229960002989 glutamic acid Drugs 0.000 description 1
- AWUCVROLDVIAJX-UHFFFAOYSA-N glycerol 1-phosphate Chemical compound OCC(O)COP(O)(O)=O AWUCVROLDVIAJX-UHFFFAOYSA-N 0.000 description 1
- 150000002333 glycines Chemical class 0.000 description 1
- 201000008977 glycoproteinosis Diseases 0.000 description 1
- 150000002337 glycosamines Chemical class 0.000 description 1
- 229930182470 glycoside Natural products 0.000 description 1
- 150000002338 glycosides Chemical class 0.000 description 1
- 230000012010 growth Effects 0.000 description 1
- 229960003878 haloperidol Drugs 0.000 description 1
- 108010089932 heparan sulfate sulfatase Proteins 0.000 description 1
- 206010019847 hepatosplenomegaly Diseases 0.000 description 1
- 238000010231 histologic analysis Methods 0.000 description 1
- GFZHNFOGCMEYTA-UHFFFAOYSA-N hydron;4-[6-imino-3-(4-methoxyphenyl)pyridazin-1-yl]butanoic acid;bromide Chemical compound [Br-].C1=CC(OC)=CC=C1C1=CC=C(N)[N+](CCCC(O)=O)=N1 GFZHNFOGCMEYTA-UHFFFAOYSA-N 0.000 description 1
- 108010039650 imiglucerase Proteins 0.000 description 1
- 229960003444 immunosuppressant agent Drugs 0.000 description 1
- 230000001861 immunosuppressant effect Effects 0.000 description 1
- 239000003018 immunosuppressive agent Substances 0.000 description 1
- 230000008676 import Effects 0.000 description 1
- 229960000905 indomethacin Drugs 0.000 description 1
- 230000005764 inhibitory process Effects 0.000 description 1
- 238000003780 insertion Methods 0.000 description 1
- 230000037431 insertion Effects 0.000 description 1
- 238000000185 intracerebroventricular administration Methods 0.000 description 1
- 238000007917 intracranial administration Methods 0.000 description 1
- 239000007951 isotonicity adjuster Substances 0.000 description 1
- 230000000366 juvenile effect Effects 0.000 description 1
- 229960003299 ketamine Drugs 0.000 description 1
- 229960004194 lidocaine Drugs 0.000 description 1
- 150000002632 lipids Chemical class 0.000 description 1
- 239000002502 liposome Substances 0.000 description 1
- 239000007788 liquid Substances 0.000 description 1
- 210000004698 lymphocyte Anatomy 0.000 description 1
- 238000012423 maintenance Methods 0.000 description 1
- 239000000594 mannitol Substances 0.000 description 1
- 235000010355 mannitol Nutrition 0.000 description 1
- 238000004519 manufacturing process Methods 0.000 description 1
- 230000007246 mechanism Effects 0.000 description 1
- 230000001404 mediated effect Effects 0.000 description 1
- AFVFQIVMOAPDHO-UHFFFAOYSA-M methanesulfonate group Chemical class CS(=O)(=O)[O-] AFVFQIVMOAPDHO-UHFFFAOYSA-M 0.000 description 1
- 230000000813 microbial effect Effects 0.000 description 1
- 244000005700 microbiome Species 0.000 description 1
- 239000002480 mineral oil Substances 0.000 description 1
- 235000010446 mineral oil Nutrition 0.000 description 1
- 229960005181 morphine Drugs 0.000 description 1
- 239000002105 nanoparticle Substances 0.000 description 1
- 230000004770 neurodegeneration Effects 0.000 description 1
- 210000002569 neuron Anatomy 0.000 description 1
- 230000008587 neuronal excitability Effects 0.000 description 1
- VVGIYYKRAMHVLU-UHFFFAOYSA-N newbouldiamide Natural products CCCCCCCCCCCCCCCCCCCC(O)C(O)C(O)C(CO)NC(=O)CCCCCCCCCCCCCCCCC VVGIYYKRAMHVLU-UHFFFAOYSA-N 0.000 description 1
- 239000000041 non-steroidal anti-inflammatory agent Substances 0.000 description 1
- 229940021182 non-steroidal anti-inflammatory drug Drugs 0.000 description 1
- 230000000269 nucleophilic effect Effects 0.000 description 1
- QUANRIQJNFHVEU-UHFFFAOYSA-N oxirane;propane-1,2,3-triol Chemical compound C1CO1.OCC(O)CO QUANRIQJNFHVEU-UHFFFAOYSA-N 0.000 description 1
- 125000000913 palmityl group Chemical group [H]C([*])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])[H] 0.000 description 1
- 230000001769 paralizing effect Effects 0.000 description 1
- 244000052769 pathogen Species 0.000 description 1
- 229960002275 pentobarbital sodium Drugs 0.000 description 1
- 125000001151 peptidyl group Chemical group 0.000 description 1
- 230000002093 peripheral effect Effects 0.000 description 1
- 239000012466 permeate Substances 0.000 description 1
- 229960003742 phenol Drugs 0.000 description 1
- 239000010452 phosphate Substances 0.000 description 1
- 239000008363 phosphate buffer Substances 0.000 description 1
- 125000002467 phosphate group Chemical group [H]OP(=O)(O[H])O[*] 0.000 description 1
- 230000019612 pigmentation Effects 0.000 description 1
- 239000013612 plasmid Substances 0.000 description 1
- 108700004029 pol Genes Proteins 0.000 description 1
- 101150088264 pol gene Proteins 0.000 description 1
- 230000008488 polyadenylation Effects 0.000 description 1
- 239000008389 polyethoxylated castor oil Substances 0.000 description 1
- 229920000642 polymer Polymers 0.000 description 1
- 229920001184 polypeptide Polymers 0.000 description 1
- 230000001566 pro-viral effect Effects 0.000 description 1
- 230000008569 process Effects 0.000 description 1
- 102000004196 processed proteins & peptides Human genes 0.000 description 1
- 238000012545 processing Methods 0.000 description 1
- 235000019833 protease Nutrition 0.000 description 1
- 230000004853 protein function Effects 0.000 description 1
- 239000002510 pyrogen Substances 0.000 description 1
- ZAHRKKWIAAJSAO-UHFFFAOYSA-N rapamycin Natural products COCC(O)C(=C/C(C)C(=O)CC(OC(=O)C1CCCCN1C(=O)C(=O)C2(O)OC(CC(OC)C(=CC=CC=CC(C)CC(C)C(=O)C)C)CCC2C)C(C)CC3CCC(O)C(C3)OC)C ZAHRKKWIAAJSAO-UHFFFAOYSA-N 0.000 description 1
- 230000008707 rearrangement Effects 0.000 description 1
- 230000001105 regulatory effect Effects 0.000 description 1
- 238000011160 research Methods 0.000 description 1
- 230000002441 reversible effect Effects 0.000 description 1
- 230000003248 secreting effect Effects 0.000 description 1
- 210000000582 semen Anatomy 0.000 description 1
- 230000035945 sensitivity Effects 0.000 description 1
- 238000004904 shortening Methods 0.000 description 1
- 208000011985 sialidosis Diseases 0.000 description 1
- 238000005245 sintering Methods 0.000 description 1
- 229960002930 sirolimus Drugs 0.000 description 1
- QFJCIRLUMZQUOT-HPLJOQBZSA-N sirolimus Chemical compound C1C[C@@H](O)[C@H](OC)C[C@@H]1C[C@@H](C)[C@H]1OC(=O)[C@@H]2CCCCN2C(=O)C(=O)[C@](O)(O2)[C@H](C)CC[C@H]2C[C@H](OC)/C(C)=C/C=C/C=C/[C@@H](C)C[C@@H](C)C(=O)[C@H](OC)[C@H](O)/C(C)=C/[C@@H](C)C(=O)C1 QFJCIRLUMZQUOT-HPLJOQBZSA-N 0.000 description 1
- 239000004055 small Interfering RNA Substances 0.000 description 1
- 239000011780 sodium chloride Substances 0.000 description 1
- 239000000243 solution Substances 0.000 description 1
- 239000002904 solvent Substances 0.000 description 1
- 210000001082 somatic cell Anatomy 0.000 description 1
- 239000000600 sorbitol Substances 0.000 description 1
- WWUZIQQURGPMPG-KRWOKUGFSA-N sphingosine Chemical compound CCCCCCCCCCCCC\C=C\[C@@H](O)[C@@H](N)CO WWUZIQQURGPMPG-KRWOKUGFSA-N 0.000 description 1
- 238000010561 standard procedure Methods 0.000 description 1
- 239000000126 substance Substances 0.000 description 1
- 150000005846 sugar alcohols Polymers 0.000 description 1
- QJJXYPPXXYFBGM-SHYZHZOCSA-N tacrolimus Natural products CO[C@H]1C[C@H](CC[C@@H]1O)C=C(C)[C@H]2OC(=O)[C@H]3CCCCN3C(=O)C(=O)[C@@]4(O)O[C@@H]([C@H](C[C@H]4C)OC)[C@@H](C[C@H](C)CC(=C[C@@H](CC=C)C(=O)C[C@H](O)[C@H]2C)C)OC QJJXYPPXXYFBGM-SHYZHZOCSA-N 0.000 description 1
- 229960002180 tetracycline Drugs 0.000 description 1
- 229930101283 tetracycline Natural products 0.000 description 1
- 235000019364 tetracycline Nutrition 0.000 description 1
- 150000003522 tetracyclines Chemical class 0.000 description 1
- 230000001225 therapeutic effect Effects 0.000 description 1
- RTKIYNMVFMVABJ-UHFFFAOYSA-L thimerosal Chemical compound [Na+].CC[Hg]SC1=CC=CC=C1C([O-])=O RTKIYNMVFMVABJ-UHFFFAOYSA-L 0.000 description 1
- 229940033663 thimerosal Drugs 0.000 description 1
- 150000007970 thio esters Chemical class 0.000 description 1
- 230000009466 transformation Effects 0.000 description 1
- 108091005703 transmembrane proteins Proteins 0.000 description 1
- 102000035160 transmembrane proteins Human genes 0.000 description 1
- 125000001493 tyrosinyl group Chemical group [H]OC1=C([H])C([H])=C(C([H])=C1[H])C([H])([H])C([H])(N([H])[H])C(*)=O 0.000 description 1
- 241001529453 unidentified herpesvirus Species 0.000 description 1
- 210000004291 uterus Anatomy 0.000 description 1
- 235000015112 vegetable and seed oil Nutrition 0.000 description 1
- 239000008158 vegetable oil Substances 0.000 description 1
- 239000003981 vehicle Substances 0.000 description 1
- 239000001993 wax Substances 0.000 description 1
Images
Classifications
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
- A61K9/0024—Solid, semi-solid or solidifying implants, which are implanted or injected in body tissue
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0085—Brain, e.g. brain implants; Spinal cord
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
- A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P11/00—Drugs for disorders of the respiratory system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P13/00—Drugs for disorders of the urinary system
- A61P13/12—Drugs for disorders of the urinary system of the kidneys
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P23/00—Anaesthetics
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/08—Antiepileptics; Anticonvulsants
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
- A61P25/16—Anti-Parkinson drugs
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
- A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P3/00—Drugs for disorders of the metabolism
- A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
- A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Biomedical Technology (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Diabetes (AREA)
- Genetics & Genomics (AREA)
- Epidemiology (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Obesity (AREA)
- Hematology (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Psychology (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Dermatology (AREA)
- Molecular Biology (AREA)
- Emergency Medicine (AREA)
- Endocrinology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Virology (AREA)
- Pulmonology (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US77145106P | 2006-02-09 | 2006-02-09 | |
| US60/771,451 | 2006-02-09 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CN101404927A true CN101404927A (zh) | 2009-04-08 |
Family
ID=38372004
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CNA2007800098972A Pending CN101404927A (zh) | 2006-02-09 | 2007-02-08 | 慢速脑室内递送 |
Country Status (19)
| Country | Link |
|---|---|
| US (3) | US20090123451A1 (https=) |
| EP (2) | EP1988823B1 (https=) |
| JP (4) | JP2009526066A (https=) |
| CN (1) | CN101404927A (https=) |
| AR (1) | AR059372A1 (https=) |
| BR (1) | BRPI0707900A2 (https=) |
| CA (2) | CA3168881C (https=) |
| CY (1) | CY1121822T1 (https=) |
| DK (1) | DK1988823T3 (https=) |
| ES (2) | ES2697502T3 (https=) |
| HU (1) | HUE040490T2 (https=) |
| IL (2) | IL193242A (https=) |
| LT (1) | LT1988823T (https=) |
| MX (4) | MX392030B (https=) |
| PL (1) | PL1988823T3 (https=) |
| PT (1) | PT1988823T (https=) |
| RU (1) | RU2452368C2 (https=) |
| SI (1) | SI1988823T1 (https=) |
| WO (1) | WO2007095056A2 (https=) |
Cited By (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US8865182B2 (en) | 2009-07-31 | 2014-10-21 | Paxvax, Inc. | Adenoviral-based vectors |
| CN106139133A (zh) * | 2010-06-25 | 2016-11-23 | 夏尔人类遗传性治疗公司 | 艾杜糖醛酸‑2‑硫酸酯酶的cns递送的方法和组合物 |
Families Citing this family (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AR059089A1 (es) * | 2006-01-20 | 2008-03-12 | Genzyme Corp | Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomal |
| DK1988823T3 (en) * | 2006-02-09 | 2018-12-03 | Genzyme Corp | SLOW INTRAVENTRICULAR ADMINISTRATION |
| US20120322861A1 (en) | 2007-02-23 | 2012-12-20 | Barry John Byrne | Compositions and Methods for Treating Diseases |
| PL3252161T3 (pl) | 2007-06-06 | 2022-03-07 | Genzyme Corporation | Terapia genowa dla lizosomalnych chorób spichrzeniowych |
| EP2167071B1 (en) * | 2007-06-13 | 2020-03-18 | Wayne State University Board Of Governors | A baclofen solution for low-volume therapeutic delivery |
| SI3998078T1 (sl) | 2009-08-28 | 2026-02-27 | Genzyme Corporation | Encimsko nadomestno zdravljenje z naraščajočim odmerkom za zdravljenje pomanjkanja kisle sfingomielinaze |
| RS61055B1 (sr) | 2012-06-19 | 2020-12-31 | Univ Florida | Kompozicije i postupci za lečenje bolesti |
| CN105377039A (zh) * | 2013-05-15 | 2016-03-02 | 明尼苏达大学董事会 | 腺相关病毒介导的基因向中枢神经系统转移 |
| GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
| TWI835747B (zh) | 2017-09-22 | 2024-03-21 | 賓州大學委員會 | 用於治療黏多醣病 ii 型之基因治療 |
| US12018087B2 (en) | 2018-08-02 | 2024-06-25 | Dyne Therapeutics, Inc. | Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject |
| US20220193250A1 (en) | 2018-08-02 | 2022-06-23 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
Citations (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5720720A (en) * | 1993-08-27 | 1998-02-24 | The United States Of America As Represented By The Department Of Health And Human Services | Convection-enhanced drug delivery |
| US20030022879A1 (en) * | 2001-03-02 | 2003-01-30 | Hesson David P. | Treatment of neurological disease |
| US20030163181A1 (en) * | 2001-12-07 | 2003-08-28 | Neuron Therapeutics, Inc. | Protection of neurological tissue by direct CNS perfusion cooling |
Family Cites Families (29)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| SE8303626D0 (sv) | 1983-06-23 | 1983-06-23 | Kabigen Ab | A recombinant plasmid a transformant microorganism, a polydoxyrebonucleotide segment, a process for producing a biologically active protein, and the protein thus produced |
| US5670488A (en) | 1992-12-03 | 1997-09-23 | Genzyme Corporation | Adenovirus vector for gene therapy |
| US5919676A (en) | 1993-06-24 | 1999-07-06 | Advec, Inc. | Adenoviral vector system comprising Cre-loxP recombination |
| CA2189067A1 (en) | 1994-04-28 | 1995-11-09 | Gary J. Nabel | Gene delivery vector using plasmid dna packaged into an adenovirus and a packaging cell line |
| DE69628744D1 (de) | 1995-04-17 | 2003-07-24 | Univ Texas System Austin Board | Adenovirus helfervirus system |
| SI0833934T2 (sl) | 1995-06-15 | 2013-04-30 | Crucell Holland B.V. | Pakirni sistemi za humani rekombinantni adenovirus za uporabo v genski terapiji |
| US6013516A (en) | 1995-10-06 | 2000-01-11 | The Salk Institute For Biological Studies | Vector and method of use for nucleic acid delivery to non-dividing cells |
| CA2239366A1 (en) | 1996-01-05 | 1997-07-17 | Genetic Therapy, Inc. | Recombinase-mediated generation of adenoviral vectors |
| US5735814A (en) * | 1996-04-30 | 1998-04-07 | Medtronic, Inc. | Techniques of treating neurodegenerative disorders by brain infusion |
| JP4001925B2 (ja) | 1996-09-13 | 2007-10-31 | トランスカーヨティック セラピーズ インク． | α―ガラクトシダーゼＡ欠損症の治療 |
| US7033598B2 (en) * | 1996-11-19 | 2006-04-25 | Intrabrain International N.V. | Methods and apparatus for enhanced and controlled delivery of a biologically active agent into the central nervous system of a mammal |
| US6042579A (en) * | 1997-04-30 | 2000-03-28 | Medtronic, Inc. | Techniques for treating neurodegenerative disorders by infusion of nerve growth factors into the brain |
| US5994136A (en) | 1997-12-12 | 1999-11-30 | Cell Genesys, Inc. | Method and means for producing high titer, safe, recombinant lentivirus vectors |
| WO1999057296A1 (en) | 1998-05-01 | 1999-11-11 | Genzyme Corporation | Partially deleted adenoviral vectors |
| US20060014166A1 (en) | 2004-01-27 | 2006-01-19 | Yossi Cohen | Novel nucleotide and amino acid sequences, and assays and methods of use thereof for diagnosis of endometriosis |
| US6702945B2 (en) | 2000-12-28 | 2004-03-09 | Exxonmobil Research And Engineering Company | Ionic membranes for organic sulfur separation from liquid hydrocarbon solutions |
| US7923032B2 (en) * | 2002-11-26 | 2011-04-12 | Seacoast Neuroscience, Inc. | Buoyant polymer particles for delivery of therapeutic agents to the central nervous system |
| RU2253486C2 (ru) * | 2003-03-03 | 2005-06-10 | Ростовский научно-исследовательский онкологический институт МЗ РФ | Способ химиотерапии опухолей центральной нервной системы |
| CA2518407A1 (en) * | 2003-03-12 | 2004-09-23 | Samaritan Pharmaceuticals, Inc. | Animal model simulating neurologic disease |
| US20040258666A1 (en) | 2003-05-01 | 2004-12-23 | Passini Marco A. | Gene therapy for neurometabolic disorders |
| CA2525236C (en) * | 2003-06-20 | 2015-03-24 | Biomarin Pharmaceutical Inc. | Delivery of therapeutic compounds to the brain and other tissues |
| US20050026823A1 (en) * | 2003-06-20 | 2005-02-03 | Biomarin Pharmaceutical Inc. | Use of the chaperone receptor-associated protein (RAP) for the delivery of therapeutic compounds to the brain and other tissues |
| US7442372B2 (en) * | 2003-08-29 | 2008-10-28 | Biomarin Pharmaceutical Inc. | Delivery of therapeutic compounds to the brain and other tissues |
| US20050208090A1 (en) * | 2004-03-18 | 2005-09-22 | Medtronic, Inc. | Methods and systems for treatment of neurological diseases of the central nervous system |
| US20080233655A1 (en) * | 2004-03-31 | 2008-09-25 | Children, Youth And Women's Health Service | Screening For Lysosomal Storage Disease Status |
| AR059089A1 (es) | 2006-01-20 | 2008-03-12 | Genzyme Corp | Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomal |
| AR059088A1 (es) * | 2006-01-20 | 2008-03-12 | Genzyme Corp | Administracion intraventricular de una proteina para esclerosis lateral amiotrofica |
| DK1988823T3 (en) | 2006-02-09 | 2018-12-03 | Genzyme Corp | SLOW INTRAVENTRICULAR ADMINISTRATION |
| PL3252161T3 (pl) | 2007-06-06 | 2022-03-07 | Genzyme Corporation | Terapia genowa dla lizosomalnych chorób spichrzeniowych |
-
2007
- 2007-02-08 DK DK07750236.7T patent/DK1988823T3/en active
- 2007-02-08 EP EP07750236.7A patent/EP1988823B1/en active Active
- 2007-02-08 PT PT07750236T patent/PT1988823T/pt unknown
- 2007-02-08 AR ARP070100529A patent/AR059372A1/es not_active Application Discontinuation
- 2007-02-08 CA CA3168881A patent/CA3168881C/en active Active
- 2007-02-08 EP EP18188582.3A patent/EP3459441B1/en active Active
- 2007-02-08 PL PL07750236T patent/PL1988823T3/pl unknown
- 2007-02-08 WO PCT/US2007/003382 patent/WO2007095056A2/en not_active Ceased
- 2007-02-08 RU RU2008136196/14A patent/RU2452368C2/ru not_active IP Right Cessation
- 2007-02-08 SI SI200732069T patent/SI1988823T1/sl unknown
- 2007-02-08 MX MX2018015511A patent/MX392030B/es unknown
- 2007-02-08 MX MX2008010125A patent/MX358492B/es active IP Right Grant
- 2007-02-08 ES ES07750236T patent/ES2697502T3/es active Active
- 2007-02-08 LT LTEP07750236.7T patent/LT1988823T/lt unknown
- 2007-02-08 ES ES18188582T patent/ES3054134T3/es active Active
- 2007-02-08 CN CNA2007800098972A patent/CN101404927A/zh active Pending
- 2007-02-08 BR BRPI0707900-1A patent/BRPI0707900A2/pt not_active IP Right Cessation
- 2007-02-08 CA CA2641359A patent/CA2641359C/en active Active
- 2007-02-08 JP JP2008554340A patent/JP2009526066A/ja active Pending
- 2007-02-08 HU HUE07750236A patent/HUE040490T2/hu unknown
- 2007-02-08 MX MX2013013333A patent/MX361816B/es unknown
-
2008
- 2008-08-05 IL IL193242A patent/IL193242A/en not_active IP Right Cessation
- 2008-08-07 US US12/187,896 patent/US20090123451A1/en not_active Abandoned
- 2008-08-07 MX MX2022005303A patent/MX2022005303A/es unknown
-
2013
- 2013-08-21 JP JP2013171131A patent/JP6129685B2/ja active Active
-
2015
- 2015-09-25 JP JP2015188744A patent/JP6408970B2/ja active Active
-
2016
- 2016-10-25 IL IL248492A patent/IL248492A0/en unknown
-
2017
- 2017-08-08 JP JP2017152871A patent/JP6617125B2/ja active Active
-
2018
- 2018-11-15 CY CY20181101209T patent/CY1121822T1/el unknown
-
2019
- 2019-02-21 US US16/281,961 patent/US11253485B2/en active Active
-
2022
- 2022-01-10 US US17/572,330 patent/US20220370348A1/en not_active Abandoned
Patent Citations (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5720720A (en) * | 1993-08-27 | 1998-02-24 | The United States Of America As Represented By The Department Of Health And Human Services | Convection-enhanced drug delivery |
| US20030022879A1 (en) * | 2001-03-02 | 2003-01-30 | Hesson David P. | Treatment of neurological disease |
| US20030163181A1 (en) * | 2001-12-07 | 2003-08-28 | Neuron Therapeutics, Inc. | Protection of neurological tissue by direct CNS perfusion cooling |
Cited By (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US8865182B2 (en) | 2009-07-31 | 2014-10-21 | Paxvax, Inc. | Adenoviral-based vectors |
| CN106139133A (zh) * | 2010-06-25 | 2016-11-23 | 夏尔人类遗传性治疗公司 | 艾杜糖醛酸‑2‑硫酸酯酶的cns递送的方法和组合物 |
Also Published As
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| US20220370348A1 (en) | Slow intraventricular delivery | |
| EP3252161B1 (en) | Gene therapy for lysosomal storage diseases | |
| AU734290B2 (en) | Compositions and methods for treating lysosomal storage disease | |
| US20040204379A1 (en) | Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases | |
| JP6312771B2 (ja) | 神経代謝性疾患についての遺伝子治療 | |
| Belur et al. | Comparative effectiveness of intracerebroventricular, intrathecal, and intranasal routes of AAV9 vector administration for genetic therapy of neurologic disease in murine mucopolysaccharidosis type I | |
| US20020095135A1 (en) | Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases | |
| US20100216709A1 (en) | Systemic insulin-like growth factor-1 therapy reduces diabetic peripheral neuropathy and improves renal function in diabetic nephropathy | |
| Gøtzsche et al. | Lalitha R. Belur1*, Megan Romero1, Junggu Lee1, Kelly M. Podetz-Pedersen1, Zhenhong Nan2, Maureen S. Riedl3, Lucy Vulchanova3, Kelley F. Kitto4, Carolyn A. Fairbanks4, Karen F. Kozarsky5†, Paul J. Orchard6, William H. Frey II7, Walter C. Low2 and R. Scott McIvor1 | |
| EP1658857A1 (en) | Compositions and methods for treating lysosomal storage disease | |
| Kang et al. | 864. AAV-2-Mediated Expression of [alpha]-N-Acetyl-Glucosaminidase in Peripheral Tissues and the CNS in Mucopolysaccharidosis IIIB Mice by Combined Intravenous and Intracisternal Vector Delivery | |
| Wilber et al. | 908. Evaluation of the Sleeping Beauty Transposon System in OTC Deficient Spf-Ash Mi A Non-Viral Approach to a Cell Autonomous Metabolic Disease | |
| CN101405008A (zh) | A型尼曼-皮克病的基因治疗 |
Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| C06 | Publication | ||
| PB01 | Publication | ||
| C10 | Entry into substantive examination | ||
| SE01 | Entry into force of request for substantive examination | ||
| C02 | Deemed withdrawal of patent application after publication (patent law 2001) | ||
| WD01 | Invention patent application deemed withdrawn after publication |
Application publication date: 20090408 |