JP2012090630A5 - - Google Patents
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- Publication number
- JP2012090630A5 JP2012090630A5 JP2011235922A JP2011235922A JP2012090630A5 JP 2012090630 A5 JP2012090630 A5 JP 2012090630A5 JP 2011235922 A JP2011235922 A JP 2011235922A JP 2011235922 A JP2011235922 A JP 2011235922A JP 2012090630 A5 JP2012090630 A5 JP 2012090630A5
- Authority
- JP
- Japan
- Prior art keywords
- sulfatase
- composition
- fge
- cell
- arylsulfatase
- 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.)
- Granted
Links
- 108060007951 sulfatase Proteins 0.000 claims 90
- 102000005262 Sulfatase Human genes 0.000 claims 89
- 210000004027 cell Anatomy 0.000 claims 48
- 239000000203 mixture Substances 0.000 claims 46
- 150000001413 amino acids Chemical class 0.000 claims 25
- 229920001184 polypeptide Polymers 0.000 claims 19
- 102000004196 processed proteins & peptides Human genes 0.000 claims 19
- 108090000765 processed proteins & peptides Proteins 0.000 claims 19
- 210000004962 mammalian cell Anatomy 0.000 claims 16
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims 15
- 150000007523 nucleic acids Chemical class 0.000 claims 14
- UGJBHEZMOKVTIM-UHFFFAOYSA-N N-formylglycine Chemical compound OC(=O)CNC=O UGJBHEZMOKVTIM-UHFFFAOYSA-N 0.000 claims 13
- 239000013598 vector Substances 0.000 claims 13
- 235000001014 amino acid Nutrition 0.000 claims 12
- 230000007812 deficiency Effects 0.000 claims 12
- 208000022018 mucopolysaccharidosis type 2 Diseases 0.000 claims 12
- 108020004707 nucleic acids Proteins 0.000 claims 11
- 102000039446 nucleic acids Human genes 0.000 claims 11
- 230000000694 effects Effects 0.000 claims 9
- 238000000034 method Methods 0.000 claims 9
- IAJILQKETJEXLJ-UHFFFAOYSA-N Galacturonsaeure Natural products O=CC(O)C(O)C(O)C(O)C(O)=O IAJILQKETJEXLJ-UHFFFAOYSA-N 0.000 claims 8
- AEMOLEFTQBMNLQ-HNFCZKTMSA-N L-idopyranuronic acid Chemical group OC1O[C@@H](C(O)=O)[C@@H](O)[C@H](O)[C@H]1O AEMOLEFTQBMNLQ-HNFCZKTMSA-N 0.000 claims 8
- 201000011442 Metachromatic leukodystrophy Diseases 0.000 claims 8
- 230000014509 gene expression Effects 0.000 claims 8
- 201000002273 mucopolysaccharidosis II Diseases 0.000 claims 8
- 239000008194 pharmaceutical composition Substances 0.000 claims 8
- 230000001105 regulatory effect Effects 0.000 claims 8
- XMTCKNXTTXDPJX-UHFFFAOYSA-N 3-oxoalanine Chemical compound O=CC(N)C(O)=O XMTCKNXTTXDPJX-UHFFFAOYSA-N 0.000 claims 7
- 102100022146 Arylsulfatase A Human genes 0.000 claims 7
- 108010036867 Cerebroside-Sulfatase Proteins 0.000 claims 7
- 208000002678 Mucopolysaccharidoses Diseases 0.000 claims 6
- 238000001415 gene therapy Methods 0.000 claims 6
- 206010028093 mucopolysaccharidosis Diseases 0.000 claims 6
- 208000012253 mucopolysaccharidosis IVA Diseases 0.000 claims 6
- 208000011580 syndromic disease Diseases 0.000 claims 6
- 102100031491 Arylsulfatase B Human genes 0.000 claims 5
- 101710115233 Arylsulfatase D Proteins 0.000 claims 5
- 102100033887 Arylsulfatase D Human genes 0.000 claims 5
- 102100033886 Arylsulfatase F Human genes 0.000 claims 5
- 101710115249 Arylsulfatase F Proteins 0.000 claims 5
- 102100033890 Arylsulfatase G Human genes 0.000 claims 5
- 101710115232 Arylsulfatase G Proteins 0.000 claims 5
- 108010027520 N-Acetylgalactosamine-4-Sulfatase Proteins 0.000 claims 5
- 101710099863 N-acetylgalactosamine-6-sulfatase Proteins 0.000 claims 5
- 102100031688 N-acetylgalactosamine-6-sulfatase Human genes 0.000 claims 5
- 108010023320 N-acetylglucosamine-6-sulfatase Proteins 0.000 claims 5
- 102100023282 N-acetylglucosamine-6-sulfatase Human genes 0.000 claims 5
- 108010006140 N-sulfoglucosamine sulfohydrolase Proteins 0.000 claims 5
- 102100027661 N-sulphoglucosamine sulphohydrolase Human genes 0.000 claims 5
- 108010087999 Steryl-Sulfatase Proteins 0.000 claims 5
- 102100038021 Steryl-sulfatase Human genes 0.000 claims 5
- 239000013603 viral vector Substances 0.000 claims 5
- 102100023943 Arylsulfatase L Human genes 0.000 claims 4
- 101710115246 Arylsulfatase L Proteins 0.000 claims 4
- 206010028095 Mucopolysaccharidosis IV Diseases 0.000 claims 4
- 208000001001 X-linked ichthyosis Diseases 0.000 claims 4
- 208000005340 mucopolysaccharidosis III Diseases 0.000 claims 4
- 108091033319 polynucleotide Proteins 0.000 claims 4
- 102000040430 polynucleotide Human genes 0.000 claims 4
- 239000002157 polynucleotide Substances 0.000 claims 4
- 208000026079 recessive X-linked ichthyosis Diseases 0.000 claims 4
- 230000003612 virological effect Effects 0.000 claims 4
- 241000702421 Dependoparvovirus Species 0.000 claims 3
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 3
- 235000018417 cysteine Nutrition 0.000 claims 3
- XUJNEKJLAYXESH-UHFFFAOYSA-N cysteine Natural products SCC(N)C(O)=O XUJNEKJLAYXESH-UHFFFAOYSA-N 0.000 claims 3
- 239000000758 substrate Substances 0.000 claims 3
- 208000025797 Mucopolysaccharidosis type 4A Diseases 0.000 claims 2
- 125000003172 aldehyde group Chemical group 0.000 claims 2
- 230000015572 biosynthetic process Effects 0.000 claims 2
- 210000004978 chinese hamster ovary cell Anatomy 0.000 claims 2
- 208000017568 chondrodysplasia Diseases 0.000 claims 2
- 239000012634 fragment Substances 0.000 claims 2
- 108020001507 fusion proteins Proteins 0.000 claims 2
- 102000037865 fusion proteins Human genes 0.000 claims 2
- 210000005260 human cell Anatomy 0.000 claims 2
- 206010021198 ichthyosis Diseases 0.000 claims 2
- 208000000690 mucopolysaccharidosis VI Diseases 0.000 claims 2
- 208000010978 mucopolysaccharidosis type 4 Diseases 0.000 claims 2
- 230000035772 mutation Effects 0.000 claims 2
- 241000701161 unidentified adenovirus Species 0.000 claims 2
- 241001430294 unidentified retrovirus Species 0.000 claims 2
- MTCFGRXMJLQNBG-REOHCLBHSA-N (2S)-2-Amino-3-hydroxypropansäure Chemical compound OC[C@H](N)C(O)=O MTCFGRXMJLQNBG-REOHCLBHSA-N 0.000 claims 1
- 102000009133 Arylsulfatases Human genes 0.000 claims 1
- XUJNEKJLAYXESH-REOHCLBHSA-N L-Cysteine Chemical compound SC[C@H](N)C(O)=O XUJNEKJLAYXESH-REOHCLBHSA-N 0.000 claims 1
- MTCFGRXMJLQNBG-UHFFFAOYSA-N Serine Natural products OCC(N)C(O)=O MTCFGRXMJLQNBG-UHFFFAOYSA-N 0.000 claims 1
- 230000003213 activating effect Effects 0.000 claims 1
- 235000009697 arginine Nutrition 0.000 claims 1
- 125000000637 arginyl group Chemical class N[C@@H](CCCNC(N)=N)C(=O)* 0.000 claims 1
- 210000000601 blood cell Anatomy 0.000 claims 1
- 210000002798 bone marrow cell Anatomy 0.000 claims 1
- 238000012258 culturing Methods 0.000 claims 1
- 125000000151 cysteine group Chemical group N[C@@H](CS)C(=O)* 0.000 claims 1
- 239000003814 drug Substances 0.000 claims 1
- 210000002889 endothelial cell Anatomy 0.000 claims 1
- 210000002919 epithelial cell Anatomy 0.000 claims 1
- 210000002950 fibroblast Anatomy 0.000 claims 1
- 210000002510 keratinocyte Anatomy 0.000 claims 1
- 210000004698 lymphocyte Anatomy 0.000 claims 1
- 238000004519 manufacturing process Methods 0.000 claims 1
- 210000000663 muscle cell Anatomy 0.000 claims 1
- 210000004498 neuroglial cell Anatomy 0.000 claims 1
- 210000002569 neuron Anatomy 0.000 claims 1
- 210000000130 stem cell Anatomy 0.000 claims 1
- 125000004055 thiomethyl group Chemical group [H]SC([H])([H])* 0.000 claims 1
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US44774703P | 2003-02-11 | 2003-02-11 | |
| US60/447,747 | 2003-02-11 |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2006503413A Division JP5241101B2 (ja) | 2003-02-11 | 2004-02-10 | 多重スルファターゼ欠損症およびその他のスルファターゼ欠損症の診断および治療 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2014036642A Division JP6007203B2 (ja) | 2003-02-11 | 2014-02-27 | 多重スルファターゼ欠損症およびその他のスルファターゼ欠損症の診断および治療 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2012090630A JP2012090630A (ja) | 2012-05-17 |
| JP2012090630A5 true JP2012090630A5 (enExample) | 2013-01-24 |
| JP5527854B2 JP5527854B2 (ja) | 2014-06-25 |
Family
ID=32869644
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2006503413A Expired - Lifetime JP5241101B2 (ja) | 2003-02-11 | 2004-02-10 | 多重スルファターゼ欠損症およびその他のスルファターゼ欠損症の診断および治療 |
| JP2011235922A Expired - Lifetime JP5527854B2 (ja) | 2003-02-11 | 2011-10-27 | 多重スルファターゼ欠損症およびその他のスルファターゼ欠損症の診断および治療 |
| JP2014036642A Expired - Lifetime JP6007203B2 (ja) | 2003-02-11 | 2014-02-27 | 多重スルファターゼ欠損症およびその他のスルファターゼ欠損症の診断および治療 |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2006503413A Expired - Lifetime JP5241101B2 (ja) | 2003-02-11 | 2004-02-10 | 多重スルファターゼ欠損症およびその他のスルファターゼ欠損症の診断および治療 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2014036642A Expired - Lifetime JP6007203B2 (ja) | 2003-02-11 | 2014-02-27 | 多重スルファターゼ欠損症およびその他のスルファターゼ欠損症の診断および治療 |
Country Status (17)
| Country | Link |
|---|---|
| US (4) | US8227212B2 (enExample) |
| EP (3) | EP2325302B1 (enExample) |
| JP (3) | JP5241101B2 (enExample) |
| CN (4) | CN1759176B (enExample) |
| AT (1) | ATE534730T1 (enExample) |
| AU (2) | AU2004210936C1 (enExample) |
| CA (1) | CA2515708A1 (enExample) |
| CY (1) | CY1117346T1 (enExample) |
| DK (2) | DK2325302T3 (enExample) |
| ES (3) | ES2555056T3 (enExample) |
| HU (1) | HUE027210T2 (enExample) |
| MX (2) | MXPA05008533A (enExample) |
| NZ (4) | NZ570201A (enExample) |
| PT (1) | PT1592786E (enExample) |
| SI (1) | SI2325302T1 (enExample) |
| WO (1) | WO2004072275A2 (enExample) |
| ZA (1) | ZA200506378B (enExample) |
Families Citing this family (50)
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| HUE027210T2 (en) * | 2003-02-11 | 2016-10-28 | Shire Human Genetic Therapies | Cells expressing the enzyme producing sulfatase and C-formylglycine together and their uses |
| US8741260B2 (en) * | 2005-10-07 | 2014-06-03 | Armagen Technologies, Inc. | Fusion proteins for delivery of GDNF to the CNS |
| US7741446B2 (en) | 2006-08-18 | 2010-06-22 | Armagen Technologies, Inc. | Fusion antibodies that cross the blood-brain barrier in both directions |
| US7985783B2 (en) * | 2006-09-21 | 2011-07-26 | The Regents Of The University Of California | Aldehyde tags, uses thereof in site-specific protein modification |
| US8974791B2 (en) | 2007-07-27 | 2015-03-10 | Armagen Technologies, Inc. | Methods and compositions for increasing α-L-iduronidase activity in the CNS |
| WO2009045370A2 (en) | 2007-09-28 | 2009-04-09 | Intrexon Corporation | Therapeutic gene-switch constructs and bioreactors for the expression of biotherapeutic molecules, and uses thereof |
| JP5687069B2 (ja) * | 2008-01-18 | 2015-03-18 | バイオマリン ファーマシューティカル インコーポレイテッド | 高度にリン酸化された活性なヒトリソソームスルファターゼ酵素の製造およびそれの使用 |
| US7722865B2 (en) | 2008-01-18 | 2010-05-25 | Biomarin Pharmaceutical Inc. | Manufacture of active highly phosphorylated human lysosomal sulfatase enzymes and uses thereof |
| WO2009120611A2 (en) * | 2008-03-27 | 2009-10-01 | The Regents Of The University Of California | Aldehyde tags, uses thereof in site-specific protein modification |
| US9238878B2 (en) | 2009-02-17 | 2016-01-19 | Redwood Bioscience, Inc. | Aldehyde-tagged protein-based drug carriers and methods of use |
| EP2408474B1 (en) | 2009-03-18 | 2019-06-26 | Armagen, Inc. | Compositions and methods for blood-brain barrier delivery of igg-decoy receptor fusion proteins |
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| KR101380740B1 (ko) | 2012-06-29 | 2014-04-11 | 쉐어 휴먼 제네텍 세러피스, 인코포레이티드 | 이듀로네이트-2-설파타제의 정제 |
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| JP6692293B2 (ja) * | 2013-07-22 | 2020-05-13 | アーマジェン・インコーポレイテッドＡｒｍａｇｅｎ， Ｉｎｃ． | Ｃｎｓにおける酵素活性を増大するための方法および組成物 |
| US20170191041A1 (en) * | 2014-07-11 | 2017-07-06 | Biostrategies LC | Materials and methods for treating disorders associated with sulfatase enzymes |
| MA41198A (fr) | 2014-12-18 | 2017-10-24 | Shire Human Genetic Therapies | Essais d'activité enzymatique pour i2s |
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| JPWO2016121715A1 (ja) * | 2015-01-26 | 2017-11-02 | 国立大学法人名古屋大学 | 肺がん患者の予後を評価するための情報を提供する方法、肺がん患者の予後予測方法、内部標準、抗体、肺がん患者の予後予測装置、予後予測装置のプログラム及び記録媒体 |
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2004
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