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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- 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
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
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- Molecular Biology (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 (enExample) |
| EP (2) | EP2717922B1 (enExample) |
| JP (2) | JP6143231B2 (enExample) |
| KR (2) | KR20150029756A (enExample) |
| CN (1) | CN103717240A (enExample) |
| AU (1) | AU2012267512B2 (enExample) |
| CA (1) | CA2838749A1 (enExample) |
| DK (1) | DK2717922T3 (enExample) |
| ES (1) | ES2710452T3 (enExample) |
| MX (1) | MX2013014544A (enExample) |
| PL (1) | PL2717922T3 (enExample) |
| PT (1) | PT2717922T (enExample) |
| RU (1) | RU2014100160A (enExample) |
| TR (1) | TR201819015T4 (enExample) |
| WO (1) | WO2012170911A2 (enExample) |
| ZA (1) | ZA201309515B (enExample) |
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 (40)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2012267512B2 (en) | 2011-06-10 | 2014-11-20 | Bluebird Bio, Inc. | Gene therapy vectors for adrenoleukodystrophy and adrenomyeloneuropathy |
| AU2013352156B2 (en) | 2012-11-27 | 2018-12-06 | Children's Medical Center Corporation | Targeting BCL11A distal regulatory elements for fetal hemoglobin reinduction |
| GB201318804D0 (en) * | 2013-10-24 | 2013-12-11 | Adaptimmune Ltd | Vectors for transgene expression |
| IL296691B2 (en) * | 2014-04-25 | 2023-11-01 | 2Seventy Bio Inc | MND promoter chimeric antigen receptors |
| EP3738597A1 (en) | 2014-04-25 | 2020-11-18 | Bluebird Bio, Inc. | Improved methods for manufacturing adoptive cell therapies |
| ES2744186T3 (es) | 2014-04-25 | 2020-02-24 | Childrens Medical Center | Composiciones y métodos para tratar las hemoglobinopatías |
| CN106793780B (zh) | 2014-06-06 | 2020-05-26 | 蓝鸟生物公司 | 改善的t细胞组合物 |
| MX2017001079A (es) | 2014-07-24 | 2017-09-12 | Bluebird Bio Inc | Receptores de antígeno quiméricos de antígeno de maduración de células b (bcma). |
| EP3971210A1 (en) * | 2014-12-12 | 2022-03-23 | 2seventy bio, Inc. | Composition for use in the treatment of b cell related conditions in a subject in need thereof comprisine an effector cell comrising a bcma chimeric antigen receptors |
| EP3270960A4 (en) * | 2015-03-20 | 2018-08-08 | Bluebird Bio, Inc. | Vector formulations |
| CN104857014B (zh) * | 2015-04-21 | 2017-12-08 | 武汉大学 | 2‑羟丙基‑β‑环化糊精在制备治疗X‑连锁肾上腺脑白质退化症的药物中的应用 |
| ES2835861T5 (en) | 2015-05-08 | 2025-02-18 | Childrens Medical Ct Corp | Targeting bcl11a enhancer functional regions for fetal hemoglobin reinduction |
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Cited By (1)
| Publication number | Priority date | Publication date | Assignee | Title |
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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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| KR20140039035A (ko) | 2014-03-31 |
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| ES2710452T3 (es) | 2019-04-25 |
| US20130004471A1 (en) | 2013-01-03 |
| EP2717922A4 (en) | 2015-01-07 |
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| JP2015107134A (ja) | 2015-06-11 |
| AU2012267512B2 (en) | 2014-11-20 |
| PT2717922T (pt) | 2018-12-18 |
| WO2012170911A3 (en) | 2013-04-11 |
| KR101528440B1 (ko) | 2015-06-26 |
| CN103717240A (zh) | 2014-04-09 |
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