RU2014100160A - Векторы генной терапии адренолейкодистрофии и адреномиелонейропатии - Google Patents
Векторы генной терапии адренолейкодистрофии и адреномиелонейропатии Download PDFInfo
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- RU2014100160A RU2014100160A RU2014100160/10A RU2014100160A RU2014100160A RU 2014100160 A RU2014100160 A RU 2014100160A RU 2014100160/10 A RU2014100160/10 A RU 2014100160/10A RU 2014100160 A RU2014100160 A RU 2014100160A RU 2014100160 A RU2014100160 A RU 2014100160A
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- vector
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- 239000013598 vector Substances 0.000 title claims abstract 64
- 201000011452 Adrenoleukodystrophy Diseases 0.000 title claims 7
- 208000018126 Adrenomyeloneuropathy Diseases 0.000 title claims 3
- 238000001415 gene therapy Methods 0.000 title claims 3
- 241000700605 Viruses Species 0.000 claims abstract 11
- 230000001105 regulatory effect Effects 0.000 claims abstract 11
- 108091033319 polynucleotide Proteins 0.000 claims abstract 10
- 102000040430 polynucleotide Human genes 0.000 claims abstract 10
- 239000002157 polynucleotide Substances 0.000 claims abstract 10
- 230000001177 retroviral effect Effects 0.000 claims abstract 9
- 229920001184 polypeptide Polymers 0.000 claims abstract 8
- 108090000765 processed proteins & peptides Proteins 0.000 claims abstract 8
- 102000004196 processed proteins & peptides Human genes 0.000 claims abstract 8
- 241000555745 Sciuridae Species 0.000 claims abstract 7
- 108091093126 WHP Posttrascriptional Response Element Proteins 0.000 claims abstract 7
- 208000006454 hepatitis Diseases 0.000 claims abstract 7
- 231100000283 hepatitis Toxicity 0.000 claims abstract 7
- 230000001124 posttranscriptional effect Effects 0.000 claims abstract 7
- 241000701022 Cytomegalovirus Species 0.000 claims abstract 6
- 241000713666 Lentivirus Species 0.000 claims abstract 6
- 108010049137 Member 1 Subfamily D ATP Binding Cassette Transporter Proteins 0.000 claims abstract 6
- 102100024643 ATP-binding cassette sub-family D member 1 Human genes 0.000 claims abstract 4
- 108020004414 DNA Proteins 0.000 claims abstract 4
- 108010006533 ATP-Binding Cassette Transporters Proteins 0.000 claims abstract 3
- 238000012217 deletion Methods 0.000 claims abstract 3
- 230000037430 deletion Effects 0.000 claims abstract 3
- 210000000274 microglia Anatomy 0.000 claims abstract 3
- 241000282693 Cercopithecidae Species 0.000 claims abstract 2
- 206010039491 Sarcoma Diseases 0.000 claims abstract 2
- 230000004048 modification Effects 0.000 claims abstract 2
- 238000012986 modification Methods 0.000 claims abstract 2
- 210000004027 cell Anatomy 0.000 claims 10
- 101000760602 Homo sapiens ATP-binding cassette sub-family D member 1 Proteins 0.000 claims 7
- 241000713772 Human immunodeficiency virus 1 Species 0.000 claims 7
- 230000008488 polyadenylation Effects 0.000 claims 7
- 102000009030 Member 1 Subfamily D ATP Binding Cassette Transporter Human genes 0.000 claims 5
- 102000047168 human ABCD1 Human genes 0.000 claims 5
- 239000002299 complementary DNA Substances 0.000 claims 4
- 102100021519 Hemoglobin subunit beta Human genes 0.000 claims 3
- 108091005904 Hemoglobin subunit beta Proteins 0.000 claims 3
- 241000283973 Oryctolagus cuniculus Species 0.000 claims 3
- 238000000034 method Methods 0.000 claims 3
- 102000005416 ATP-Binding Cassette Transporters Human genes 0.000 claims 2
- 210000001671 embryonic stem cell Anatomy 0.000 claims 2
- 210000003958 hematopoietic stem cell Anatomy 0.000 claims 2
- 238000004806 packaging method and process Methods 0.000 claims 2
- 238000012856 packing Methods 0.000 claims 2
- 210000001988 somatic stem cell Anatomy 0.000 claims 2
- 210000000130 stem cell Anatomy 0.000 claims 2
- 241000725303 Human immunodeficiency virus Species 0.000 claims 1
- 241000713883 Myeloproliferative sarcoma virus Species 0.000 claims 1
- 108010076504 Protein Sorting Signals Proteins 0.000 claims 1
- KDCGOANMDULRCW-UHFFFAOYSA-N Purine Natural products N1=CNC2=NC=NC2=C1 KDCGOANMDULRCW-UHFFFAOYSA-N 0.000 claims 1
- 108010006025 bovine growth hormone Proteins 0.000 claims 1
- 230000003828 downregulation Effects 0.000 claims 1
- 239000003623 enhancer Substances 0.000 claims 1
- 239000012634 fragment Substances 0.000 claims 1
- 210000004962 mammalian cell Anatomy 0.000 claims 1
- 239000002245 particle Substances 0.000 claims 1
- 230000002265 prevention Effects 0.000 claims 1
- 125000000561 purinyl group Chemical group N1=C(N=C2N=CNC2=C1)* 0.000 claims 1
- 238000006467 substitution reaction Methods 0.000 claims 1
- 239000013603 viral vector Substances 0.000 claims 1
- 102000043966 ABC-type transporter activity proteins Human genes 0.000 abstract 1
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- 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
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- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
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- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
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- Medicinal Chemistry (AREA)
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- General Engineering & Computer Science (AREA)
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- Animal Behavior & Ethology (AREA)
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- Gastroenterology & Hepatology (AREA)
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Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201161495857P | 2011-06-10 | 2011-06-10 | |
| US61/495,857 | 2011-06-10 | ||
| PCT/US2012/041693 WO2012170911A2 (en) | 2011-06-10 | 2012-06-08 | Gene therapy vectors for adrenoleukodystrophy and adrenomyeloneuropathy |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| RU2014100160A true RU2014100160A (ru) | 2015-07-20 |
Family
ID=47296778
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| RU2014100160/10A RU2014100160A (ru) | 2011-06-10 | 2012-06-08 | Векторы генной терапии адренолейкодистрофии и адреномиелонейропатии |
Country Status (16)
| Country | Link |
|---|---|
| US (3) | US8858928B2 (cg-RX-API-DMAC7.html) |
| EP (2) | EP3492108A1 (cg-RX-API-DMAC7.html) |
| JP (2) | JP6143231B2 (cg-RX-API-DMAC7.html) |
| KR (2) | KR20150029756A (cg-RX-API-DMAC7.html) |
| CN (1) | CN103717240A (cg-RX-API-DMAC7.html) |
| AU (1) | AU2012267512B2 (cg-RX-API-DMAC7.html) |
| CA (1) | CA2838749A1 (cg-RX-API-DMAC7.html) |
| DK (1) | DK2717922T3 (cg-RX-API-DMAC7.html) |
| ES (1) | ES2710452T3 (cg-RX-API-DMAC7.html) |
| MX (1) | MX2013014544A (cg-RX-API-DMAC7.html) |
| PL (1) | PL2717922T3 (cg-RX-API-DMAC7.html) |
| PT (1) | PT2717922T (cg-RX-API-DMAC7.html) |
| RU (1) | RU2014100160A (cg-RX-API-DMAC7.html) |
| TR (1) | TR201819015T4 (cg-RX-API-DMAC7.html) |
| WO (1) | WO2012170911A2 (cg-RX-API-DMAC7.html) |
| ZA (1) | ZA201309515B (cg-RX-API-DMAC7.html) |
Cited By (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| RU2739384C2 (ru) * | 2015-11-05 | 2020-12-23 | Дзе Дженерал Хоспитал Корпорейшн | Интратекальная доставка последовательностей нуклеиновых кислот, кодирующих abcd1, для лечения адреномиелоневропатии |
Families Citing this family (42)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| DK2717922T3 (en) | 2011-06-10 | 2019-01-07 | Bluebird Bio Inc | GENERATIVE VECTORS FOR ADDRENOLEUCODYSTROPHY AND ADDRENOMYELONE EUROPE |
| ES2708948T3 (es) | 2012-11-27 | 2019-04-12 | Childrens Medical Ct Corp | Elementos reguladores distales de bcl11a como dianas para la reinducción de la hemoglobina fetal |
| GB201318804D0 (en) † | 2013-10-24 | 2013-12-11 | Adaptimmune Ltd | Vectors for transgene expression |
| CN106794260B (zh) | 2014-04-25 | 2021-02-23 | 儿童医疗中心有限公司 | 治疗血红蛋白病的组合物和方法 |
| WO2015164759A2 (en) * | 2014-04-25 | 2015-10-29 | Bluebird Bio, Inc. | Mnd promoter chimeric antigen receptors |
| CN106456670B (zh) | 2014-04-25 | 2020-01-07 | 蓝鸟生物公司 | 制备过继性细胞疗法的改善方法 |
| RS61406B1 (sr) | 2014-06-06 | 2021-03-31 | Bluebird Bio Inc | Poboljšane kompozicije t ćelija |
| EP3172231B1 (en) | 2014-07-24 | 2021-05-05 | Bluebird Bio, Inc. | Bcma chimeric antigen receptors |
| US10383929B2 (en) | 2014-12-12 | 2019-08-20 | Bluebird Bio, Inc. | BCMA chimeric antigen receptors |
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| RU2739384C2 (ru) * | 2015-11-05 | 2020-12-23 | Дзе Дженерал Хоспитал Корпорейшн | Интратекальная доставка последовательностей нуклеиновых кислот, кодирующих abcd1, для лечения адреномиелоневропатии |
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| DK2717922T3 (en) | 2019-01-07 |
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| CA2838749A1 (en) | 2012-12-13 |
| TR201819015T4 (tr) | 2019-01-21 |
| AU2012267512B2 (en) | 2014-11-20 |
| EP2717922A2 (en) | 2014-04-16 |
| US9789139B2 (en) | 2017-10-17 |
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| US20150064150A1 (en) | 2015-03-05 |
| WO2012170911A3 (en) | 2013-04-11 |
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