JP2013531471A5 - - Google Patents
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- JP2013531471A5 JP2013531471A5 JP2013506330A JP2013506330A JP2013531471A5 JP 2013531471 A5 JP2013531471 A5 JP 2013531471A5 JP 2013506330 A JP2013506330 A JP 2013506330A JP 2013506330 A JP2013506330 A JP 2013506330A JP 2013531471 A5 JP2013531471 A5 JP 2013531471A5
- Authority
- JP
- Japan
- Prior art keywords
- subject
- raav
- administration
- cns
- vector
- 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.)
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- 210000003169 central nervous system Anatomy 0.000 claims 32
- 239000013598 vector Substances 0.000 claims 29
- 210000001519 tissue Anatomy 0.000 claims 21
- 239000008194 pharmaceutical composition Substances 0.000 claims 19
- 241000702421 Dependoparvovirus Species 0.000 claims 16
- 108020004707 nucleic acids Proteins 0.000 claims 15
- 102000039446 nucleic acids Human genes 0.000 claims 15
- 150000007523 nucleic acids Chemical class 0.000 claims 15
- 108090000623 proteins and genes Proteins 0.000 claims 13
- 108700019146 Transgenes Proteins 0.000 claims 12
- 108090000565 Capsid Proteins Proteins 0.000 claims 10
- 102100023321 Ceruloplasmin Human genes 0.000 claims 10
- 108091070501 miRNA Proteins 0.000 claims 9
- 239000002679 microRNA Substances 0.000 claims 9
- 108010005939 Ciliary Neurotrophic Factor Proteins 0.000 claims 8
- 102100031614 Ciliary neurotrophic factor Human genes 0.000 claims 8
- 102000036509 GTP Cyclohydrolase Human genes 0.000 claims 8
- 108010023555 GTP Cyclohydrolase Proteins 0.000 claims 8
- 241000699666 Mus <mouse, genus> Species 0.000 claims 8
- 108010025020 Nerve Growth Factor Proteins 0.000 claims 8
- 102000015336 Nerve Growth Factor Human genes 0.000 claims 8
- 229940053128 nerve growth factor Drugs 0.000 claims 8
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims 6
- 241000700159 Rattus Species 0.000 claims 5
- 150000001413 amino acids Chemical class 0.000 claims 5
- 230000002518 glial effect Effects 0.000 claims 5
- 230000002401 inhibitory effect Effects 0.000 claims 5
- 108020004999 messenger RNA Proteins 0.000 claims 5
- 241001164823 Adeno-associated virus - 7 Species 0.000 claims 4
- 229940088872 Apoptosis inhibitor Drugs 0.000 claims 4
- 108090000715 Brain-derived neurotrophic factor Proteins 0.000 claims 4
- 102000004219 Brain-derived neurotrophic factor Human genes 0.000 claims 4
- 208000022526 Canavan disease Diseases 0.000 claims 4
- 102000034615 Glial cell line-derived neurotrophic factor Human genes 0.000 claims 4
- 108091010837 Glial cell line-derived neurotrophic factor Proteins 0.000 claims 4
- 102000008221 Superoxide Dismutase-1 Human genes 0.000 claims 4
- 108010021188 Superoxide Dismutase-1 Proteins 0.000 claims 4
- 206010002026 amyotrophic lateral sclerosis Diseases 0.000 claims 4
- 239000000158 apoptosis inhibitor Substances 0.000 claims 4
- 108010046910 brain-derived growth factor Proteins 0.000 claims 4
- 239000003102 growth factor Substances 0.000 claims 4
- 108091006086 inhibitor proteins Proteins 0.000 claims 4
- 238000007913 intrathecal administration Methods 0.000 claims 4
- 210000004962 mammalian cell Anatomy 0.000 claims 4
- 239000000203 mixture Substances 0.000 claims 4
- 230000009223 neuronal apoptosis Effects 0.000 claims 4
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims 3
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims 3
- 241000282553 Macaca Species 0.000 claims 3
- 238000007914 intraventricular administration Methods 0.000 claims 3
- OUYCCCASQSFEME-UHFFFAOYSA-N tyrosine Natural products OC(=O)C(N)CC1=CC=C(O)C=C1 OUYCCCASQSFEME-UHFFFAOYSA-N 0.000 claims 3
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 2
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims 2
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 2
- 108020005544 Antisense RNA Proteins 0.000 claims 2
- 101100222092 Caenorhabditis elegans csp-3 gene Proteins 0.000 claims 2
- 102000004190 Enzymes Human genes 0.000 claims 2
- 108090000790 Enzymes Proteins 0.000 claims 2
- 241000282326 Felis catus Species 0.000 claims 2
- 108010025815 Kanamycin Kinase Proteins 0.000 claims 2
- 108091027967 Small hairpin RNA Proteins 0.000 claims 2
- 210000004027 cell Anatomy 0.000 claims 2
- 239000003184 complementary RNA Substances 0.000 claims 2
- 230000006870 function Effects 0.000 claims 2
- 238000000185 intracerebroventricular administration Methods 0.000 claims 2
- 241001515942 marmosets Species 0.000 claims 2
- 102000004169 proteins and genes Human genes 0.000 claims 2
- 239000004055 small Interfering RNA Substances 0.000 claims 2
- 210000000278 spinal cord Anatomy 0.000 claims 2
- 101800000263 Acidic protein Proteins 0.000 claims 1
- 108091010877 Allograft inflammatory factor 1 Proteins 0.000 claims 1
- 102100040121 Allograft inflammatory factor 1 Human genes 0.000 claims 1
- 108700023155 Aspartoacylases Proteins 0.000 claims 1
- 108010035563 Chloramphenicol O-acetyltransferase Proteins 0.000 claims 1
- 102100039289 Glial fibrillary acidic protein Human genes 0.000 claims 1
- 101710193519 Glial fibrillary acidic protein Proteins 0.000 claims 1
- 108060001084 Luciferase Proteins 0.000 claims 1
- 239000005089 Luciferase Substances 0.000 claims 1
- 102000004317 Lyases Human genes 0.000 claims 1
- 108090000856 Lyases Proteins 0.000 claims 1
- 101710160107 Outer membrane protein A Proteins 0.000 claims 1
- 108091000117 Tyrosine 3-Monooxygenase Proteins 0.000 claims 1
- 102000048218 Tyrosine 3-monooxygenases Human genes 0.000 claims 1
- 102000006646 aminoglycoside phosphotransferase Human genes 0.000 claims 1
- 239000003708 ampul Substances 0.000 claims 1
- 102000018919 aspartoacylase Human genes 0.000 claims 1
- 210000000133 brain stem Anatomy 0.000 claims 1
- 208000015114 central nervous system disease Diseases 0.000 claims 1
- 210000001638 cerebellum Anatomy 0.000 claims 1
- 239000003814 drug Substances 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 108091006047 fluorescent proteins Proteins 0.000 claims 1
- 102000034287 fluorescent proteins Human genes 0.000 claims 1
- 210000005046 glial fibrillary acidic protein Anatomy 0.000 claims 1
- 210000001320 hippocampus Anatomy 0.000 claims 1
- 210000003016 hypothalamus Anatomy 0.000 claims 1
- 238000007918 intramuscular administration Methods 0.000 claims 1
- 238000007912 intraperitoneal administration Methods 0.000 claims 1
- 230000003907 kidney function Effects 0.000 claims 1
- 210000005230 lumbar spinal cord Anatomy 0.000 claims 1
- 210000004705 lumbosacral region Anatomy 0.000 claims 1
- 230000001537 neural effect Effects 0.000 claims 1
- 210000004129 prosencephalon Anatomy 0.000 claims 1
- 108010045647 puromycin N-acetyltransferase Proteins 0.000 claims 1
- 239000013608 rAAV vector Substances 0.000 claims 1
- 210000005084 renal tissue Anatomy 0.000 claims 1
- 230000002207 retinal effect Effects 0.000 claims 1
- 210000001103 thalamus Anatomy 0.000 claims 1
- 210000000115 thoracic cavity Anatomy 0.000 claims 1
- 230000002861 ventricular Effects 0.000 claims 1
- 230000004304 visual acuity Effects 0.000 claims 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US32762710P | 2010-04-23 | 2010-04-23 | |
| US61/327,627 | 2010-04-23 | ||
| PCT/US2011/033616 WO2011133890A1 (en) | 2010-04-23 | 2011-04-22 | Cns targeting aav vectors and methods of use thereof |
Related Child Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016000309A Division JP6422897B2 (ja) | 2010-04-23 | 2016-01-04 | Ｃｎｓターゲティングａａｖベクターおよびその使用方法 |
| JP2016000310A Division JP6235619B2 (ja) | 2010-04-23 | 2016-01-04 | Ｃｎｓターゲティングａａｖベクターおよびその使用方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2013531471A JP2013531471A (ja) | 2013-08-08 |
| JP2013531471A5 true JP2013531471A5 (enExample) | 2014-08-21 |
| JP5963743B2 JP5963743B2 (ja) | 2016-08-03 |
Family
ID=44834531
Family Applications (6)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2013506330A Active JP5963743B2 (ja) | 2010-04-23 | 2011-04-22 | Ｃｎｓターゲティングａａｖベクターおよびその使用方法 |
| JP2016000310A Active JP6235619B2 (ja) | 2010-04-23 | 2016-01-04 | Ｃｎｓターゲティングａａｖベクターおよびその使用方法 |
| JP2016000309A Active JP6422897B2 (ja) | 2010-04-23 | 2016-01-04 | Ｃｎｓターゲティングａａｖベクターおよびその使用方法 |
| JP2018195573A Active JP6879988B2 (ja) | 2010-04-23 | 2018-10-17 | Ｃｎｓターゲティングａａｖベクターおよびその使用方法 |
| JP2019005016A Active JP6941632B2 (ja) | 2010-04-23 | 2019-01-16 | Ｃｎｓターゲティングａａｖベクターおよびその使用方法 |
| JP2021077161A Pending JP2021138702A (ja) | 2010-04-23 | 2021-04-30 | Ｃｎｓターゲティングａａｖベクターおよびその使用方法 |
Family Applications After (5)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016000310A Active JP6235619B2 (ja) | 2010-04-23 | 2016-01-04 | Ｃｎｓターゲティングａａｖベクターおよびその使用方法 |
| JP2016000309A Active JP6422897B2 (ja) | 2010-04-23 | 2016-01-04 | Ｃｎｓターゲティングａａｖベクターおよびその使用方法 |
| JP2018195573A Active JP6879988B2 (ja) | 2010-04-23 | 2018-10-17 | Ｃｎｓターゲティングａａｖベクターおよびその使用方法 |
| JP2019005016A Active JP6941632B2 (ja) | 2010-04-23 | 2019-01-16 | Ｃｎｓターゲティングａａｖベクターおよびその使用方法 |
| JP2021077161A Pending JP2021138702A (ja) | 2010-04-23 | 2021-04-30 | Ｃｎｓターゲティングａａｖベクターおよびその使用方法 |
Country Status (7)
| Country | Link |
|---|---|
| US (8) | US9102949B2 (enExample) |
| EP (6) | EP2561073B1 (enExample) |
| JP (6) | JP5963743B2 (enExample) |
| CA (3) | CA3066596A1 (enExample) |
| DK (2) | DK2561073T3 (enExample) |
| ES (2) | ES2698203T3 (enExample) |
| WO (1) | WO2011133890A1 (enExample) |
Families Citing this family (233)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN103589692B (zh) | 2001-11-13 | 2018-07-24 | 宾夕法尼亚大学托管会 | 检测和/或鉴定腺伴随病毒(aav)序列以及分离所鉴定的新型序列的方法 |
| CN101528916B (zh) | 2006-04-28 | 2013-09-04 | 宾夕法尼亚大学托管会 | 规模可调的aav生产方法 |
| DK2623605T3 (en) | 2007-04-09 | 2019-03-25 | Univ Florida | RAAV VECTOR COMPOSITIONS WITH TYROSIN MODIFIED CAPSIDE PROTEINS AND PROCEDURES FOR USE THEREOF |
| US9725485B2 (en) * | 2012-05-15 | 2017-08-08 | University Of Florida Research Foundation, Inc. | AAV vectors with high transduction efficiency and uses thereof for gene therapy |
| US9217155B2 (en) * | 2008-05-28 | 2015-12-22 | University Of Massachusetts | Isolation of novel AAV'S and uses thereof |
| US11219696B2 (en) | 2008-12-19 | 2022-01-11 | Nationwide Children's Hospital | Delivery of polynucleotides using recombinant AAV9 |
| US8734809B2 (en) | 2009-05-28 | 2014-05-27 | University Of Massachusetts | AAV's and uses thereof |
| CA2833912C (en) | 2010-04-23 | 2021-09-21 | University Of Massachusetts | Aav-based treatment of cholesterol-related disorders |
| DK2561073T3 (en) * | 2010-04-23 | 2016-12-12 | Univ Massachusetts | Aav vectors targeted to central nervous system and methods of use thereof |
| CA3050894C (en) | 2010-04-23 | 2022-10-18 | University Of Massachusetts | Multicistronic expression constructs |
| WO2012145624A2 (en) | 2011-04-21 | 2012-10-26 | University Of Massachusetts | Raav-based compositions and methods for treating alpha-1 anti-trypsin deficiencies |
| US20130039888A1 (en) | 2011-06-08 | 2013-02-14 | Nationwide Children's Hospital Inc. | Products and methods for delivery of polynucleotides by adeno-associated virus for lysosomal storage disorders |
| WO2013006514A2 (en) * | 2011-07-01 | 2013-01-10 | University Of South Florida | Recombinant adeno-associated virus-mediated expression of fractalkine for treatment of neuroinflammatory and neurodegenerative diseases |
| BR112014020325A2 (pt) * | 2012-02-17 | 2017-08-08 | Childrens Hospital Philadelphia | composições de vetor do aav e métodos para a transferência de gene para as células, órgãos e tecidos |
| WO2013151664A1 (en) | 2012-04-02 | 2013-10-10 | modeRNA Therapeutics | Modified polynucleotides for the production of proteins |
| US10501513B2 (en) | 2012-04-02 | 2019-12-10 | Modernatx, Inc. | Modified polynucleotides for the production of oncology-related proteins and peptides |
| US10501512B2 (en) | 2012-04-02 | 2019-12-10 | Modernatx, Inc. | Modified polynucleotides |
| US12358954B2 (en) | 2012-05-15 | 2025-07-15 | University Of Florida Research Foundation, Incorporated | Capsid-modified rAAV vector compositions and methods therefor |
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| CA2885216A1 (en) | 2012-09-17 | 2014-03-20 | The Research Institute At Nationwide Children's Hospital | Compositions and methods for treating amyotrophic lateral sclerosis |
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| DK2956056T3 (da) | 2013-03-14 | 2019-09-16 | Univ Laval | Elektroretinografisystem (erg) til vurdering af psykiatriske lidelser |
| BR112015021036A2 (pt) | 2013-03-15 | 2017-10-10 | Univ Pennsylvania | composições e métodos para tratar mps1 |
| JP2016523835A (ja) * | 2013-05-15 | 2016-08-12 | リージェンツ オブ ザ ユニバーシティ オブ ミネソタ | 中枢神経系へのアデノ随伴ウイルスを介した遺伝子導入 |
| NZ716102A (en) | 2013-07-22 | 2021-12-24 | The Children’S Hospital Of Philadelphia | Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues |
| RU2664471C2 (ru) * | 2013-07-26 | 2018-08-17 | Юниверсити Оф Айова Рисерч Фаундейшн | Способы и композиции для лечения болезней мозга |
| RS63942B1 (sr) | 2013-08-27 | 2023-02-28 | Res Inst Nationwide Childrens Hospital | Proizvodi i postupci za lečenje amiotrofične lateralne skleroze |
| EP3564379A1 (en) | 2013-09-13 | 2019-11-06 | California Institute of Technology | Selective recovery |
| WO2015060722A1 (en) * | 2013-10-24 | 2015-04-30 | Uniqure Ip B.V. | Aav-5 pseudotyped vector for gene therapy for neurological diseases |
| US9725719B2 (en) | 2013-11-05 | 2017-08-08 | The Research Institute At Nationwide Children's Hospital | Compositions and methods for inhibiting NF-κB and SOD-1 to treat amyotrophic lateral sclerosis |
| CN105765066B (zh) | 2013-11-29 | 2018-12-14 | 宝生物工程株式会社 | 用于定量腺伴随病毒的方法 |
| AU2014362722B2 (en) * | 2013-12-11 | 2021-06-10 | Riken | Method for manufacturing ciliary marginal zone-like structure |
| GB201403684D0 (en) | 2014-03-03 | 2014-04-16 | King S College London | Vector |
| WO2015127128A2 (en) | 2014-02-19 | 2015-08-27 | University Of Massachusetts | Recombinant aavs having useful transcytosis properties |
| JP6756700B2 (ja) | 2014-03-18 | 2020-09-16 | ユニバーシティ オブ マサチューセッツ | 筋萎縮性側索硬化症を処置するためのｒＡＡＶベースの組成物および方法 |
| WO2015164786A1 (en) | 2014-04-25 | 2015-10-29 | University Of Massachusetts | Recombinant aav vectors useful for reducing immunity against transgene products |
| WO2015164723A1 (en) * | 2014-04-25 | 2015-10-29 | The Trustees Of The University Of Pennsylvania | Methods and compositions for treating metastatic breast cancer and other cancers in the brain |
| LT3137497T (lt) | 2014-05-02 | 2021-07-26 | Genzyme Corporation | Aav vektoriai, skirti tinklainės ir cns genų terapijai |
| US10689653B2 (en) | 2014-06-03 | 2020-06-23 | University Of Massachusetts | Compositions and methods for modulating dysferlin expression |
| US10577627B2 (en) | 2014-06-09 | 2020-03-03 | Voyager Therapeutics, Inc. | Chimeric capsids |
| US11008561B2 (en) | 2014-06-30 | 2021-05-18 | Bioverativ Therapeutics Inc. | Optimized factor IX gene |
| US10370432B2 (en) | 2014-10-03 | 2019-08-06 | University Of Massachusetts | Heterologous targeting peptide grafted AAVS |
| WO2016054557A1 (en) | 2014-10-03 | 2016-04-07 | University Of Massachusetts | Novel high efficiency library-identified aav vectors |
| WO2016065001A1 (en) | 2014-10-21 | 2016-04-28 | University Of Massachusetts | Recombinant aav variants and uses thereof |
| EP3215191B1 (en) | 2014-11-05 | 2024-08-14 | Voyager Therapeutics, Inc. | Aadc polynucleotides for the treatment of parkinson's disease |
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| EP3256170B1 (en) | 2015-02-13 | 2020-09-23 | University of Massachusetts | Compositions and methods for transient delivery of nucleases |
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| WO2017060510A1 (en) * | 2015-10-09 | 2017-04-13 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Methods and pharmaceutical compositions for the treatment of alzheimer's disease |
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| EP3387138B2 (en) | 2015-12-11 | 2026-05-27 | The Trustees Of The University Of Pennsylvania | Scalable purification method for aav9 |
| KR20180118659A (ko) | 2016-02-01 | 2018-10-31 | 바이오버라티브 테라퓨틱스 인크. | 최적화된 viii 인자 유전자 |
| CA3011939A1 (en) | 2016-02-02 | 2017-08-10 | University Of Massachusetts | Method to enhance the efficiency of systemic aav gene delivery to the central nervous system |
| MA43968A (fr) | 2016-02-03 | 2018-12-12 | Univ Pennsylvania | Thérapie génique pour traiter la mucopolysaccharidose de type i |
| CA3012344A1 (en) | 2016-02-12 | 2017-08-17 | University Of Massachusetts | Anti-angiogenic mirna therapeutics for inhibiting corneal neovascularization |
| WO2017173234A1 (en) * | 2016-03-31 | 2017-10-05 | University Of Cincinnati | Methods and compositions for the treatment of als |
| EP3440210A4 (en) | 2016-04-05 | 2019-11-27 | University of Massachusetts | COMPOSITIONS AND METHOD FOR SELECTIVELY INHIBITING THE EXPRESSION OF GRAINYHEAD LIKE PROTEIN |
| US11253612B2 (en) | 2016-04-15 | 2022-02-22 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating mucopolysaccharidosis type II |
| US11413356B2 (en) | 2016-04-15 | 2022-08-16 | University Of Massachusetts | Methods and compositions for treating metabolic imbalance |
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