JP2019506445A5 - - Google Patents
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- JP2019506445A5 JP2019506445A5 JP2018549419A JP2018549419A JP2019506445A5 JP 2019506445 A5 JP2019506445 A5 JP 2019506445A5 JP 2018549419 A JP2018549419 A JP 2018549419A JP 2018549419 A JP2018549419 A JP 2018549419A JP 2019506445 A5 JP2019506445 A5 JP 2019506445A5
- Authority
- JP
- Japan
- Prior art keywords
- capsid protein
- amino acid
- disease
- viral vector
- parvovirus
- 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
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- 108090000565 Capsid Proteins Proteins 0.000 claims 37
- 102100023321 Ceruloplasmin Human genes 0.000 claims 37
- 239000013603 viral vector Substances 0.000 claims 14
- 241000700605 Viruses Species 0.000 claims 12
- 125000000539 amino acid group Chemical group 0.000 claims 12
- 239000013598 vector Substances 0.000 claims 11
- 241000125945 Protoparvovirus Species 0.000 claims 10
- 102000039446 nucleic acids Human genes 0.000 claims 6
- 108020004707 nucleic acids Proteins 0.000 claims 6
- 150000007523 nucleic acids Chemical class 0.000 claims 6
- 239000008194 pharmaceutical composition Substances 0.000 claims 5
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims 4
- 229920002971 Heparan sulfate Polymers 0.000 claims 4
- HTTJABKRGRZYRN-UHFFFAOYSA-N Heparin Chemical compound OC1C(NC(=O)C)C(O)OC(COS(O)(=O)=O)C1OC1C(OS(O)(=O)=O)C(O)C(OC2C(C(OS(O)(=O)=O)C(OC3C(C(O)C(O)C(O3)C(O)=O)OS(O)(=O)=O)C(CO)O2)NS(O)(=O)=O)C(C(O)=O)O1 HTTJABKRGRZYRN-UHFFFAOYSA-N 0.000 claims 4
- 125000003275 alpha amino acid group Chemical group 0.000 claims 4
- 210000000234 capsid Anatomy 0.000 claims 4
- 208000011580 syndromic disease Diseases 0.000 claims 4
- 230000010415 tropism Effects 0.000 claims 4
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 3
- 238000000034 method Methods 0.000 claims 3
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims 2
- 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 claims 2
- 206010019280 Heart failures Diseases 0.000 claims 2
- OIRDTQYFTABQOQ-KQYNXXCUSA-N adenosine Chemical compound C1=NC=2C(N)=NC=NC=2N1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1O OIRDTQYFTABQOQ-KQYNXXCUSA-N 0.000 claims 2
- 206010003246 arthritis Diseases 0.000 claims 2
- 230000007812 deficiency Effects 0.000 claims 2
- 208000019622 heart disease Diseases 0.000 claims 2
- 210000004185 liver Anatomy 0.000 claims 2
- 238000012986 modification Methods 0.000 claims 2
- 230000004048 modification Effects 0.000 claims 2
- 210000003205 muscle Anatomy 0.000 claims 2
- 201000006938 muscular dystrophy Diseases 0.000 claims 2
- 210000002027 skeletal muscle Anatomy 0.000 claims 2
- 230000009885 systemic effect Effects 0.000 claims 2
- 239000013607 AAV vector Substances 0.000 claims 1
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 1
- 208000024827 Alzheimer disease Diseases 0.000 claims 1
- 208000031277 Amaurotic familial idiocy Diseases 0.000 claims 1
- 206010003591 Ataxia Diseases 0.000 claims 1
- 208000023275 Autoimmune disease Diseases 0.000 claims 1
- 201000006935 Becker muscular dystrophy Diseases 0.000 claims 1
- 239000002126 C01EB10 - Adenosine Substances 0.000 claims 1
- 206010007559 Cardiac failure congestive Diseases 0.000 claims 1
- 102100022641 Coagulation factor IX Human genes 0.000 claims 1
- 102100026735 Coagulation factor VIII Human genes 0.000 claims 1
- 206010010356 Congenital anomaly Diseases 0.000 claims 1
- 201000003883 Cystic fibrosis Diseases 0.000 claims 1
- 241000702421 Dependoparvovirus Species 0.000 claims 1
- 208000024720 Fabry Disease Diseases 0.000 claims 1
- 201000003542 Factor VIII deficiency Diseases 0.000 claims 1
- 208000015872 Gaucher disease Diseases 0.000 claims 1
- 208000010055 Globoid Cell Leukodystrophy Diseases 0.000 claims 1
- WHUUTDBJXJRKMK-UHFFFAOYSA-N Glutamic acid Natural products OC(=O)C(N)CCC(O)=O WHUUTDBJXJRKMK-UHFFFAOYSA-N 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
- 208000009292 Hemophilia A Diseases 0.000 claims 1
- 101000911390 Homo sapiens Coagulation factor VIII Proteins 0.000 claims 1
- 208000023105 Huntington disease Diseases 0.000 claims 1
- 208000015178 Hurler syndrome Diseases 0.000 claims 1
- 206010020575 Hyperammonaemia Diseases 0.000 claims 1
- 208000028226 Krabbe disease Diseases 0.000 claims 1
- 108010001831 LDL receptors Proteins 0.000 claims 1
- 102100024640 Low-density lipoprotein receptor Human genes 0.000 claims 1
- 102100033448 Lysosomal alpha-glucosidase Human genes 0.000 claims 1
- 206010056886 Mucopolysaccharidosis I Diseases 0.000 claims 1
- 206010028095 Mucopolysaccharidosis IV Diseases 0.000 claims 1
- 206010056893 Mucopolysaccharidosis VII Diseases 0.000 claims 1
- 206010028289 Muscle atrophy Diseases 0.000 claims 1
- 206010028980 Neoplasm Diseases 0.000 claims 1
- 208000002537 Neuronal Ceroid-Lipofuscinoses Diseases 0.000 claims 1
- 208000018737 Parkinson disease Diseases 0.000 claims 1
- 208000005764 Peripheral Arterial Disease Diseases 0.000 claims 1
- 208000030831 Peripheral arterial occlusive disease Diseases 0.000 claims 1
- 201000011252 Phenylketonuria Diseases 0.000 claims 1
- 201000001828 Sly syndrome Diseases 0.000 claims 1
- 208000002903 Thalassemia Diseases 0.000 claims 1
- 229960005305 adenosine Drugs 0.000 claims 1
- 208000007502 anemia Diseases 0.000 claims 1
- 210000004556 brain Anatomy 0.000 claims 1
- 201000011510 cancer Diseases 0.000 claims 1
- 230000000747 cardiac effect Effects 0.000 claims 1
- 238000012217 deletion Methods 0.000 claims 1
- 230000037430 deletion Effects 0.000 claims 1
- 206010012601 diabetes mellitus Diseases 0.000 claims 1
- 208000035475 disorder Diseases 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 206010015037 epilepsy Diseases 0.000 claims 1
- 210000001508 eye Anatomy 0.000 claims 1
- 235000013922 glutamic acid Nutrition 0.000 claims 1
- 239000004220 glutamic acid Substances 0.000 claims 1
- 201000004502 glycogen storage disease II Diseases 0.000 claims 1
- 208000009429 hemophilia B Diseases 0.000 claims 1
- 229910052739 hydrogen Inorganic materials 0.000 claims 1
- 239000001257 hydrogen Substances 0.000 claims 1
- 206010020718 hyperplasia Diseases 0.000 claims 1
- 238000000338 in vitro Methods 0.000 claims 1
- 208000017476 juvenile neuronal ceroid lipofuscinosis Diseases 0.000 claims 1
- 208000002780 macular degeneration Diseases 0.000 claims 1
- 208000030159 metabolic disease Diseases 0.000 claims 1
- 201000002273 mucopolysaccharidosis II Diseases 0.000 claims 1
- 208000022018 mucopolysaccharidosis type 2 Diseases 0.000 claims 1
- 208000010978 mucopolysaccharidosis type 4 Diseases 0.000 claims 1
- 208000025919 mucopolysaccharidosis type 7 Diseases 0.000 claims 1
- 201000006417 multiple sclerosis Diseases 0.000 claims 1
- 201000000585 muscular atrophy Diseases 0.000 claims 1
- 210000004165 myocardium Anatomy 0.000 claims 1
- 208000015122 neurodegenerative disease Diseases 0.000 claims 1
- 201000007607 neuronal ceroid lipofuscinosis 3 Diseases 0.000 claims 1
- 201000001119 neuropathy Diseases 0.000 claims 1
- 230000007823 neuropathy Effects 0.000 claims 1
- 208000030613 peripheral artery disease Diseases 0.000 claims 1
- 208000033808 peripheral neuropathy Diseases 0.000 claims 1
- 102000040430 polynucleotide Human genes 0.000 claims 1
- 108091033319 polynucleotide Proteins 0.000 claims 1
- 239000002157 polynucleotide Substances 0.000 claims 1
- 230000002207 retinal effect Effects 0.000 claims 1
- 238000006467 substitution reaction Methods 0.000 claims 1
- 210000001519 tissue Anatomy 0.000 claims 1
- 230000026683 transduction Effects 0.000 claims 1
- 238000010361 transduction Methods 0.000 claims 1
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2022016322A JP2022065003A (ja) | 2015-12-14 | 2022-02-04 | パルボウイルスベクターの高められた送達のための改変キャプシドタンパク質 |
| JP2024003324A JP2024045227A (ja) | 2015-12-14 | 2024-01-12 | パルボウイルスベクターの高められた送達のための改変キャプシドタンパク質 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562266941P | 2015-12-14 | 2015-12-14 | |
| US62/266,941 | 2015-12-14 | ||
| PCT/US2016/066466 WO2017106236A1 (en) | 2015-12-14 | 2016-12-14 | Modified capsid proteins for enhanced delivery of parvovirus vectors |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022016322A Division JP2022065003A (ja) | 2015-12-14 | 2022-02-04 | パルボウイルスベクターの高められた送達のための改変キャプシドタンパク質 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2019506445A JP2019506445A (ja) | 2019-03-07 |
| JP2019506445A5 true JP2019506445A5 (enExample) | 2020-01-30 |
| JP7406783B2 JP7406783B2 (ja) | 2023-12-28 |
Family
ID=59057532
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018549419A Active JP7406783B2 (ja) | 2015-12-14 | 2016-12-14 | パルボウイルスベクターの高められた送達のための改変キャプシドタンパク質 |
| JP2022016322A Pending JP2022065003A (ja) | 2015-12-14 | 2022-02-04 | パルボウイルスベクターの高められた送達のための改変キャプシドタンパク質 |
| JP2024003324A Pending JP2024045227A (ja) | 2015-12-14 | 2024-01-12 | パルボウイルスベクターの高められた送達のための改変キャプシドタンパク質 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022016322A Pending JP2022065003A (ja) | 2015-12-14 | 2022-02-04 | パルボウイルスベクターの高められた送達のための改変キャプシドタンパク質 |
| JP2024003324A Pending JP2024045227A (ja) | 2015-12-14 | 2024-01-12 | パルボウイルスベクターの高められた送達のための改変キャプシドタンパク質 |
Country Status (12)
| Country | Link |
|---|---|
| US (3) | US11124546B2 (enExample) |
| EP (2) | EP4659816A3 (enExample) |
| JP (3) | JP7406783B2 (enExample) |
| CN (2) | CN116478254A (enExample) |
| AU (2) | AU2016371779C1 (enExample) |
| CA (1) | CA3008013A1 (enExample) |
| DK (1) | DK3390429T3 (enExample) |
| ES (1) | ES3040934T3 (enExample) |
| MX (2) | MX395524B (enExample) |
| PL (1) | PL3390429T3 (enExample) |
| RU (1) | RU2751953C2 (enExample) |
| WO (1) | WO2017106236A1 (enExample) |
Families Citing this family (47)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US10577627B2 (en) | 2014-06-09 | 2020-03-03 | Voyager Therapeutics, Inc. | Chimeric capsids |
| EP3215191B1 (en) | 2014-11-05 | 2024-08-14 | Voyager Therapeutics, Inc. | Aadc polynucleotides for the treatment of parkinson's disease |
| CN114717264A (zh) | 2014-11-14 | 2022-07-08 | 沃雅戈治疗公司 | 治疗肌萎缩性侧索硬化(als)的组合物和方法 |
| KR20230145206A (ko) | 2014-11-14 | 2023-10-17 | 보이저 테라퓨틱스, 인크. | 조절성 폴리뉴클레오티드 |
| US11697825B2 (en) | 2014-12-12 | 2023-07-11 | Voyager Therapeutics, Inc. | Compositions and methods for the production of scAAV |
| GB201508026D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Capsid |
| GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
| IL314171A (en) | 2015-09-28 | 2024-09-01 | Univ Florida | Methods and compositions for antibody-evading virus vectors |
| AU2016362477A1 (en) | 2015-12-02 | 2018-06-14 | Voyager Therapeutics, Inc. | Assays for the detection of AAV neutralizing antibodies |
| US11124546B2 (en) * | 2015-12-14 | 2021-09-21 | The University Of North Carolina At Chapel Hill | Modified capsid proteins for enhanced delivery of parvovirus vectors |
| IL297576B2 (en) | 2016-05-18 | 2024-02-01 | Voyager Therapeutics Inc | Compositions and methods of treating huntington's disease |
| US10584337B2 (en) | 2016-05-18 | 2020-03-10 | Voyager Therapeutics, Inc. | Modulatory polynucleotides |
| EP3585883A4 (en) * | 2017-02-21 | 2021-04-14 | University of Florida Research Foundation, Incorporated | MODIFIED AAV CAPSID PROTEINS AND USES THEREOF |
| MX2019013172A (es) | 2017-05-05 | 2020-09-07 | Voyager Therapeutics Inc | Composiciones y metodos para tratar la enfermedad de huntington. |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| MX2020003042A (es) | 2017-09-29 | 2020-11-18 | Voyager Therapeutics Inc | Rescate del fenotipo neurológico central y periférico de la ataxia de friedreich mediante administración intravenosa. |
| WO2019191716A1 (en) | 2018-03-29 | 2019-10-03 | University Of Florida Research Foundation, Incorporated | Aav6 variants |
| EP3781187A4 (en) * | 2018-03-29 | 2021-12-22 | Asklepios Biopharmaceutical, Inc. | TROPICAL RECOMBINANT AAV6 LIVER VECTORS TO Bypass NEUTRALIZATION |
| CA3095179A1 (en) | 2018-04-03 | 2019-10-10 | Stridebio, Inc. | Antibody-evading virus vectors |
| BR112020020266A2 (pt) | 2018-04-03 | 2021-01-19 | Stridebio, Inc. | Vetores de vírus com evasão de anticorpos |
| MX2020010465A (es) | 2018-04-03 | 2021-01-08 | Vectores de virus para direccionamiento a tejidos oftalmicos. | |
| CN112424359A (zh) | 2018-05-15 | 2021-02-26 | 沃雅戈治疗公司 | 用于治疗帕金森氏病的组合物和方法 |
| WO2019221992A1 (en) | 2018-05-15 | 2019-11-21 | President And Fellows Of Harvard College | Viral vectors exhibiting improved gene delivery properties |
| US12281321B2 (en) | 2018-09-28 | 2025-04-22 | Voyager Therapeutics, Inc. | Frataxin expression constructs having engineered promoters and methods of use thereof |
| WO2020102645A1 (en) * | 2018-11-16 | 2020-05-22 | Asklepios Biopharmaceutical, Inc. | Therapeutic adeno-associated virus for treating pompe disease |
| CN113727992A (zh) | 2019-03-21 | 2021-11-30 | 斯特里迪比奥公司 | 重组腺相关病毒载体 |
| AU2020263392A1 (en) * | 2019-04-26 | 2021-10-28 | The University Of North Carolina At Chapel Hill | Methods and compositions for dual glycan binding AAV2.5 vector |
| AU2020299026A1 (en) * | 2019-07-04 | 2022-01-20 | Children's Medical Research Institute | Methods and AAV vectors for in vivo transduction |
| EP4010357A2 (en) | 2019-08-09 | 2022-06-15 | University of Florida Research Foundation, Incorporated | Aav capsid variants for targeting human glioblastoma cells |
| MX2022004353A (es) | 2019-10-17 | 2022-07-19 | Casete de transferencia de aav. | |
| CN114787180A (zh) | 2019-10-17 | 2022-07-22 | 斯特里迪比奥公司 | 用于治疗c型尼曼-匹克病的腺相关病毒载体 |
| EP4055030A4 (en) | 2019-11-08 | 2024-05-22 | President And Fellows Of Harvard College | VIRAL CAPSID POLYPEPTIDES |
| EP4055034A4 (en) | 2019-11-08 | 2024-05-29 | President And Fellows Of Harvard College | VIRAL CAPSID POLYPEPTIDES |
| CN110950934B (zh) * | 2019-12-31 | 2022-11-04 | 复旦大学 | 一种腺相关病毒衣壳蛋白、载体及其构建方法与应用 |
| FI4103214T3 (fi) | 2020-02-13 | 2026-07-03 | Tenaya Therapeutics Inc | Geeniterapian vektorit sydänsairauden hoitoon |
| JP2023542614A (ja) | 2020-08-19 | 2023-10-11 | サレプタ セラピューティクス， インコーポレイテッド | レット症候群の治療のためのアデノ随伴ウイルスベクター |
| US20230374542A1 (en) | 2020-10-07 | 2023-11-23 | Asklepios Biopharmaceutical, Inc. | Therapeutic adeno-associated virus delivery of fukutin related protein (fkrp) for treating dystroglycanopathy. disorders including limb girdle 21 (lgmd21) |
| AU2021410043A1 (en) * | 2020-12-23 | 2023-06-22 | Synteny Therapeutics, Inc. | Protoparvovirus and tetraparvovirus compositions and methods for gene therapy |
| TW202317768A (zh) | 2021-07-08 | 2023-05-01 | 美商特納亞治療股份有限公司 | 用於基因療法的優化表現卡匣 |
| WO2024054864A1 (en) | 2022-09-06 | 2024-03-14 | Tenaya Therapeutics, Inc. | Cardioprotective heart disease therapies |
| EP4623087A2 (en) * | 2022-11-21 | 2025-10-01 | Shape Therapeutics Inc. | Systems for amplification of aav cap protein |
| US12383615B2 (en) | 2023-03-23 | 2025-08-12 | Carbon Biosciences, Inc. | Protoparvovirus compositions comprising a protoparvovirus variant VP1 capsid polypeptide and related methods |
| EP4695396A1 (en) | 2023-04-10 | 2026-02-18 | Tenaya Therapeutics, Inc. | Guide rnas, vectors, and virions for targeting mutations in the pln gene |
| WO2024215655A1 (en) | 2023-04-10 | 2024-10-17 | Tenaya Therapeutics, Inc. | Cardioprotective bag3 therapies |
| US20260234665A1 (en) * | 2023-04-17 | 2026-08-13 | The University Of North Carolina At Chapel Hill | Modified capsid proteins for enhanced intra-articular delivery of adenovirus-associated virus (aav) vectors |
| CN121311595A (zh) | 2023-04-17 | 2026-01-09 | 阿斯克生物公司 | 转基因在灵长类动物中可调节的受控表达 |
| EP4786601A1 (en) | 2025-01-31 | 2026-08-05 | AskBio Inc. | Helper plasmids and uses thereof |
Family Cites Families (41)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4501729A (en) | 1982-12-13 | 1985-02-26 | Research Corporation | Aerosolized amiloride treatment of retained pulmonary secretions |
| AU7966987A (en) | 1986-09-08 | 1988-04-07 | Applied Biotechnology, Inc. | Empty viral capsid vaccines |
| US4968603A (en) | 1986-12-31 | 1990-11-06 | The Regents Of The University Of California | Determination of status in neoplastic disease |
| EP0442926A1 (en) | 1988-11-10 | 1991-08-28 | Imperial Cancer Research Technology Limited | Polypeptides |
| US5916563A (en) | 1988-11-14 | 1999-06-29 | United States Of America | Parvovirus protein presenting capsids |
| US5399346A (en) | 1989-06-14 | 1995-03-21 | The United States Of America As Represented By The Department Of Health And Human Services | Gene therapy |
| ES2026826A6 (es) | 1991-03-26 | 1992-05-01 | Ercros Sa | Procedimiento para la produccion de una vacuna subunidad contra el parvovirus canino y otros virus relacionados. |
| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| US5869248A (en) | 1994-03-07 | 1999-02-09 | Yale University | Targeted cleavage of RNA using ribonuclease P targeting and cleavage sequences |
| US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
| US5599706A (en) | 1994-09-23 | 1997-02-04 | Stinchcomb; Dan T. | Ribozymes targeted to apo(a) mRNA |
| IL122206A0 (en) | 1995-06-07 | 1998-04-05 | Univ North Carolina | Adeno-associated virus vectors for gene expression |
| US6040183A (en) | 1995-06-07 | 2000-03-21 | University Of North Carloina At Chapel Hill | Helper virus-free AAV production |
| US6093570A (en) | 1995-06-07 | 2000-07-25 | The University Of North Carolina At Chapel Hill | Helper virus-free AAV production |
| EP0883344B2 (en) | 1995-12-15 | 2010-06-09 | VIRxSYS Corporation | Therapeutic molecules generated by trans-splicing |
| US6083702A (en) | 1995-12-15 | 2000-07-04 | Intronn Holdings Llc | Methods and compositions for use in spliceosome mediated RNA trans-splicing |
| AU4645697A (en) | 1996-09-11 | 1998-04-02 | Government Of The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services, The | Aav4 vector and uses thereof |
| US6156303A (en) | 1997-06-11 | 2000-12-05 | University Of Washington | Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom |
| DE69939169D1 (de) | 1998-05-28 | 2008-09-04 | Us Gov Health & Human Serv | Aav5 vektoren und deren verwendung |
| AU774706B2 (en) | 1998-09-22 | 2004-07-08 | Johns Hopkins University, The | Methods for large-scale production of recombinant AAV vectors |
| DE69936104T2 (de) | 1998-11-05 | 2008-01-17 | The Trustees Of The University Of Pennsylvania | Nukleinsäuresequenzen des adeno-assoziierten virus des serotyps i, und vektoren und wirtszellen, die diese enthalten |
| EP1135468B1 (en) | 1998-11-10 | 2010-01-06 | University Of North Carolina At Chapel Hill | Virus vectors and methods of making and administering the same |
| US7465583B2 (en) | 2000-06-01 | 2008-12-16 | The University Of North Carolina At Chapel Hill | Duplexed parvovirus vectors |
| AU6814901A (en) | 2000-06-01 | 2001-12-11 | Univ North Carolina | Methods and compounds for controlled release of recombinant parvovirus vectors |
| US6623729B2 (en) | 2001-07-09 | 2003-09-23 | Korea Advanced Institute Of Science And Technology | Process for preparing sustained release micelle employing conjugate of anticancer drug and biodegradable polymer |
| US7071172B2 (en) | 2002-04-30 | 2006-07-04 | The University Of North Carolina At Chapel Hill | Secretion signal vectors |
| ITRM20020253A1 (it) | 2002-05-08 | 2003-11-10 | Univ Roma | Molecole chimeriche di snrna con sequenze antisenso per le giunzioni di splicing del gene della distrofina e applicazioni terapeutiche. |
| FR2874384B1 (fr) | 2004-08-17 | 2010-07-30 | Genethon | Vecteur viral adeno-associe pour realiser du saut d'exons dans un gene codant une proteine a domaines dispensables |
| EP1828390B1 (en) * | 2004-12-15 | 2012-06-13 | The University Of North Carolina At Chapel Hill | Chimeric vectors |
| DE23193606T1 (de) | 2005-04-07 | 2024-02-15 | The Trustees Of The University Of Pennsylvania | Verfahren zur erhöhung der funktion eines aav-vektors |
| WO2007084773A2 (en) * | 2006-01-20 | 2007-07-26 | University Of North Carolina At Chapel Hill | Enhanced production of infectious parvovirus vectors in insect cells |
| US7867484B2 (en) * | 2006-01-27 | 2011-01-11 | University Of North Carolina At Chapel Hill | Heparin and heparan sulfate binding chimeric vectors |
| JP2009535339A (ja) * | 2006-04-28 | 2009-10-01 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | 低下されたキャプシド免疫原性を有する改変ａａｖベクターおよびその使用 |
| WO2008088895A2 (en) | 2007-01-18 | 2008-07-24 | University Of Missouri-Columbia | Synthetic mini/micro-dystrophin genes to restore nnos to the sarcolemma |
| EP2396343B1 (en) * | 2009-02-11 | 2017-05-17 | The University of North Carolina At Chapel Hill | Modified virus vectors and methods of making and using the same |
| PL2900686T3 (pl) | 2012-09-28 | 2021-01-25 | The University Of North Carolina At Chapel Hill | Wektory aav ukierunkowane na oligodendrocyty |
| KR102298579B1 (ko) * | 2013-05-21 | 2021-09-07 | 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 | 캡시드-변형된 raav3 벡터 조성물, 및 인간 간암의 유전자 요법에서의 용도 |
| EP3049527A4 (en) * | 2013-09-26 | 2017-08-16 | University of Florida Research Foundation, Inc. | Synthetic combinatorial aav capsid library for targeted gene therapy |
| US10081659B2 (en) * | 2015-04-06 | 2018-09-25 | The United States Of America, As Represented By The Secretary, Dept. Of Health And Human Services | Adeno-associated vectors for enhanced transduction and reduced immunogenicity |
| IL314171A (en) * | 2015-09-28 | 2024-09-01 | Univ Florida | Methods and compositions for antibody-evading virus vectors |
| US11124546B2 (en) * | 2015-12-14 | 2021-09-21 | The University Of North Carolina At Chapel Hill | Modified capsid proteins for enhanced delivery of parvovirus vectors |
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2016
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