JP2020114235A5 - - Google Patents
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- JP2020114235A5 JP2020114235A5 JP2020054236A JP2020054236A JP2020114235A5 JP 2020114235 A5 JP2020114235 A5 JP 2020114235A5 JP 2020054236 A JP2020054236 A JP 2020054236A JP 2020054236 A JP2020054236 A JP 2020054236A JP 2020114235 A5 JP2020114235 A5 JP 2020114235A5
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- Prior art keywords
- sequence
- nucleic acid
- hotc
- seq
- acid sequence
- 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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- 150000007523 nucleic acids Chemical class 0.000 claims 18
- 239000013612 plasmid Substances 0.000 claims 14
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 13
- 102100021244 Integral membrane protein GPR180 Human genes 0.000 claims 6
- 102000002248 Thyroxine-Binding Globulin Human genes 0.000 claims 4
- 108010000259 Thyroxine-Binding Globulin Proteins 0.000 claims 4
- 108020004707 nucleic acids Proteins 0.000 claims 4
- 102000039446 nucleic acids Human genes 0.000 claims 4
- 102000004169 proteins and genes Human genes 0.000 claims 4
- 108090000623 proteins and genes Proteins 0.000 claims 4
- XUIIKFGFIJCVMT-UHFFFAOYSA-N thyroxine-binding globulin Natural products IC1=CC(CC([NH3+])C([O-])=O)=CC(I)=C1OC1=CC(I)=C(O)C(I)=C1 XUIIKFGFIJCVMT-UHFFFAOYSA-N 0.000 claims 4
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 3
- 239000003623 enhancer Substances 0.000 claims 3
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims 2
- 210000004185 liver Anatomy 0.000 claims 2
- 238000004806 packaging method and process Methods 0.000 claims 2
- 241000202702 Adeno-associated virus - 3 Species 0.000 claims 1
- 102000003966 Alpha-1-microglobulin Human genes 0.000 claims 1
- 101800001761 Alpha-1-microglobulin Proteins 0.000 claims 1
- 241000702421 Dependoparvovirus Species 0.000 claims 1
- 101800001691 Inter-alpha-trypsin inhibitor light chain Proteins 0.000 claims 1
- 102000007981 Ornithine carbamoyltransferase Human genes 0.000 claims 1
- 101710198224 Ornithine carbamoyltransferase, mitochondrial Proteins 0.000 claims 1
- 102100032859 Protein AMBP Human genes 0.000 claims 1
- 230000015556 catabolic process Effects 0.000 claims 1
- 238000006731 degradation reaction Methods 0.000 claims 1
- 230000001124 posttranscriptional effect Effects 0.000 claims 1
- 230000001105 regulatory effect Effects 0.000 claims 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201461950157P | 2014-03-09 | 2014-03-09 | |
| US61/950,157 | 2014-03-09 | ||
| JP2016556768A JP6822841B2 (ja) | 2014-03-09 | 2015-03-09 | オルニチントランスカルバミラーゼ（ｏｔｃ）欠損症の処置において有用な組成物 |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016556768A Division JP6822841B2 (ja) | 2014-03-09 | 2015-03-09 | オルニチントランスカルバミラーゼ（ｏｔｃ）欠損症の処置において有用な組成物 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2020114235A JP2020114235A (ja) | 2020-07-30 |
| JP2020114235A5 true JP2020114235A5 (enExample) | 2021-03-25 |
| JP6920500B2 JP6920500B2 (ja) | 2021-08-18 |
Family
ID=52737408
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016556768A Active JP6822841B2 (ja) | 2014-03-09 | 2015-03-09 | オルニチントランスカルバミラーゼ（ｏｔｃ）欠損症の処置において有用な組成物 |
| JP2020054236A Active JP6920500B2 (ja) | 2014-03-09 | 2020-03-25 | オルニチントランスカルバミラーゼ（ｏｔｃ）欠損症の処置において有用な組成物 |
Family Applications Before (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016556768A Active JP6822841B2 (ja) | 2014-03-09 | 2015-03-09 | オルニチントランスカルバミラーゼ（ｏｔｃ）欠損症の処置において有用な組成物 |
Country Status (22)
| Country | Link |
|---|---|
| US (6) | US9890365B2 (enExample) |
| EP (2) | EP3778627A1 (enExample) |
| JP (2) | JP6822841B2 (enExample) |
| KR (1) | KR102390075B1 (enExample) |
| AU (2) | AU2015229750A1 (enExample) |
| BR (2) | BR112016020688B1 (enExample) |
| CA (1) | CA2939950C (enExample) |
| CL (2) | CL2016002235A1 (enExample) |
| CY (1) | CY1123378T1 (enExample) |
| DK (1) | DK3116900T3 (enExample) |
| ES (1) | ES2821938T3 (enExample) |
| HR (1) | HRP20201544T1 (enExample) |
| HU (1) | HUE051311T2 (enExample) |
| IL (2) | IL247329B (enExample) |
| LT (1) | LT3116900T (enExample) |
| MX (1) | MX377804B (enExample) |
| PL (1) | PL3116900T3 (enExample) |
| PT (1) | PT3116900T (enExample) |
| RS (1) | RS60902B1 (enExample) |
| SI (1) | SI3116900T1 (enExample) |
| SM (1) | SMT202000531T1 (enExample) |
| WO (2) | WO2015138357A2 (enExample) |
Families Citing this family (41)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN103501812A (zh) | 2011-04-29 | 2014-01-08 | 西莱克塔生物科技公司 | 用于过敏症治疗的致耐受性合成纳米载体 |
| CA2910584A1 (en) | 2013-05-03 | 2014-11-06 | Selecta Biosciences, Inc. | Use of immunosuppressants attached to synthetic nanocarriers to enhance levels of cd4+ regulatory t cells |
| WO2015017519A1 (en) | 2013-07-30 | 2015-02-05 | Phaserx, Inc. | Block copolymers and their conjugates or complexes with oligonucleotides |
| RS60902B1 (sr) | 2014-03-09 | 2020-11-30 | Univ Pennsylvania | Kompozicije korisne u lečenju nedostatka ornitinske transkarbamilaze (otc) |
| WO2016037163A1 (en) | 2014-09-07 | 2016-03-10 | Selecta Biosciences, Inc. | Methods and compositions for attenuating gene therapy anti-viral transfer vector immune responses |
| CN107106689A (zh) | 2014-11-05 | 2017-08-29 | 沃雅戈治疗公司 | 用于治疗帕金森病的aadc多核苷酸 |
| CN114642735A (zh) | 2015-01-21 | 2022-06-21 | 菲泽尔克斯公司 | 用于将治疗剂和诊断剂递送到细胞中的方法、组合物和系统 |
| WO2017059902A1 (en) | 2015-10-07 | 2017-04-13 | Biontech Rna Pharmaceuticals Gmbh | 3' utr sequences for stabilization of rna |
| EP3452101A2 (en) | 2016-05-04 | 2019-03-13 | CureVac AG | Rna encoding a therapeutic protein |
| IL263801B2 (en) | 2016-07-26 | 2024-01-01 | Biomarin Pharm Inc | Novel adeno-associated virus capsid proteins |
| IL264872B2 (en) | 2016-08-18 | 2025-02-01 | Univ California | CRISPR-CAS genome engineering using a modular AAV delivery system |
| US11400109B2 (en) | 2016-11-10 | 2022-08-02 | Translate Bio, Inc. | Subcutaneous delivery of messenger RNA |
| WO2018126084A1 (en) | 2016-12-30 | 2018-07-05 | Phaserx, Inc. | Branched peg molecules and related compositions and methods |
| WO2018126112A1 (en) * | 2016-12-30 | 2018-07-05 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating phenylketonuria |
| US11104887B2 (en) | 2017-01-03 | 2021-08-31 | Ethris Gmbh | Ornithine transcarbamylase coding polyribonucleotides and formulations thereof |
| US11938197B2 (en) | 2017-01-10 | 2024-03-26 | The Sydney Children's Hospitals Network (Randwick And Westmead (Incorporating The Royal Alexandra Hospital For Children) | Polynucleotides and vectors for the expression of transgenes |
| EP3592389B1 (en) | 2017-03-11 | 2025-05-07 | Cartesian Therapeutics, Inc. | Methods and compositions related to combined treatment with anti-inflammatories and synthetic nanocarriers comprising an immunosuppressant |
| AU2018275549B2 (en) * | 2017-05-31 | 2024-12-19 | The University Of North Carolina At Chapel Hill | Optimized human clotting factor IX gene expression cassettes and their use |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| JP7374883B2 (ja) | 2017-08-09 | 2023-11-07 | バイオベラティブ セラピューティクス インコーポレイテッド | 核酸分子およびその使用 |
| WO2019104152A1 (en) * | 2017-11-22 | 2019-05-31 | Modernatx, Inc. | Polynucleotides encoding ornithine transcarbamylase for the treatment of urea cycle disorders |
| WO2019217513A2 (en) | 2018-05-09 | 2019-11-14 | Biomarin Pharmaceutical Inc. | Methods of treating phenylketonuria |
| TW202005978A (zh) | 2018-05-14 | 2020-02-01 | 美商拜奧馬林製藥公司 | 新穎肝靶向腺相關病毒載體 |
| JP7448488B2 (ja) * | 2018-05-15 | 2024-03-12 | トランスレイト バイオ， インコーポレイテッド | メッセンジャーｒｎａの皮下送達 |
| MX2020012077A (es) | 2018-05-15 | 2021-03-09 | Voyager Therapeutics Inc | Composiciones y metodos para el tratamiento de la enfermedad de parkinson. |
| CN112771070A (zh) * | 2018-07-16 | 2021-05-07 | 西莱克塔生物科技公司 | Otc构建体和载体的方法和组合物 |
| WO2020016655A2 (en) | 2018-07-17 | 2020-01-23 | Helixmith Co., Ltd. | Treatment of neuropathy with dna constructs expressing igf-1 isoforms |
| CN119955796A (zh) * | 2018-08-09 | 2025-05-09 | 比奥维拉迪维治疗股份有限公司 | 核酸分子及其用于非病毒基因疗法的用途 |
| SG11202103151RA (en) | 2018-09-28 | 2021-04-29 | Voyager Therapeutics Inc | Frataxin expression constructs having engineered promoters and methods of use thereof |
| AU2019394996B2 (en) * | 2018-12-06 | 2025-11-06 | Arcturus Therapeutics, Inc. | Compositions and methods for treating ornithine transcarbamylase deficiency |
| US20230193315A1 (en) | 2019-01-31 | 2023-06-22 | Oregon Health & Science University | Methods for using transcription-dependent directed evolution of aav capsids |
| WO2021178510A1 (en) * | 2020-03-03 | 2021-09-10 | Arcturus Therapeutics, Inc. | Compositions and methods for the treatment of ornithine transcarbamylase deficiency |
| CA3169529A1 (en) * | 2020-03-04 | 2021-09-10 | Jingjing Jiang | Compositions and methods for the treatment of metabolic liver disorders |
| EP4121544A1 (en) * | 2020-03-19 | 2023-01-25 | Ultragenyx Pharmaceutical Inc. | Compositions and methods for reducing reverse packaging of cap and rep sequences in recombinant aav |
| AU2021270447A1 (en) | 2020-05-12 | 2023-01-05 | The Trustees Of The University Of Pennsylvania | Compositions for DRG-specific reduction of transgene expression |
| EP4284335A1 (en) | 2021-02-01 | 2023-12-06 | RegenxBio Inc. | Gene therapy for neuronal ceroid lipofuscinoses |
| US20250339480A1 (en) * | 2021-04-21 | 2025-11-06 | Jichi Medical University | Adeno-associated virus virion for treating ornithine transcarbamylase deficiency |
| IL310963A (en) * | 2021-08-23 | 2024-04-01 | Bioverativ Therapeutics Inc | Production of closed DNA with inverted repeat sequences |
| US20250295807A1 (en) | 2021-11-15 | 2025-09-25 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
| IL314257A (en) * | 2022-01-21 | 2024-09-01 | Univ Pennsylvania | Treatment methods for ornithine transcarbamylase deficiency (OTC) |
| JP2025534674A (ja) * | 2022-10-11 | 2025-10-17 | パシフィック・ノースウェスト・リサーチ・インスティテュート | 遺伝性障害に関する構築物 |
Family Cites Families (81)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US1016745A (en) | 1911-04-11 | 1912-02-06 | Edwin C Henrikson | Table-caster. |
| US5139941A (en) | 1985-10-31 | 1992-08-18 | University Of Florida Research Foundation, Inc. | AAV transduction vectors |
| US5436146A (en) | 1989-09-07 | 1995-07-25 | The Trustees Of Princeton University | Helper-free stocks of recombinant adeno-associated virus vectors |
| US6268213B1 (en) | 1992-06-03 | 2001-07-31 | Richard Jude Samulski | Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy |
| US5869305A (en) | 1992-12-04 | 1999-02-09 | The University Of Pittsburgh | Recombinant viral vector system |
| US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
| US5856152A (en) | 1994-10-28 | 1999-01-05 | The Trustees Of The University Of Pennsylvania | Hybrid adenovirus-AAV vector and methods of use therefor |
| US5652224A (en) | 1995-02-24 | 1997-07-29 | The Trustees Of The University Of Pennsylvania | Methods and compositions for gene therapy for the treatment of defects in lipoprotein metabolism |
| US5741683A (en) | 1995-06-07 | 1998-04-21 | The Research Foundation Of State University Of New York | In vitro packaging of adeno-associated virus DNA |
| US6093570A (en) | 1995-06-07 | 2000-07-25 | The University Of North Carolina At Chapel Hill | Helper virus-free AAV production |
| US20020037867A1 (en) | 1999-02-26 | 2002-03-28 | James M. Wilson | Method for recombinant adeno-associated virus-directed gene therapy |
| US5866552A (en) | 1996-09-06 | 1999-02-02 | The Trustees Of The University Of Pennsylvania | Method for expressing a gene in the absence of an immune response |
| ATE465267T1 (de) | 1996-09-06 | 2010-05-15 | Univ Pennsylvania | Rekombinante aav zur herstellung eines medikaments für die gentherapie von muskelzellen |
| AU722375B2 (en) | 1996-09-06 | 2000-08-03 | Trustees Of The University Of Pennsylvania, The | Methods using cre-lox for production of recombinant adeno-associated viruses |
| EP0931158A1 (en) | 1996-09-06 | 1999-07-28 | The Trustees Of The University Of Pennsylvania | An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase |
| EP0932694A2 (en) | 1996-09-11 | 1999-08-04 | THE UNITED STATES GOVERNMENT as represented by THE DEPARTMENT OF HEALTH AND HUMAN SERVICES | Aav4 vector and uses thereof |
| ES2215222T3 (es) | 1996-12-05 | 2004-10-01 | Crucell Holland B.V. | Modificacion genetica de celulas cepa de repoblacion hematopoyeticas de primates. |
| ATE364718T1 (de) | 1997-04-01 | 2007-07-15 | Solexa Ltd | Verfahren zur vervielfältigung von nukleinsäure |
| DE69824859T2 (de) | 1997-04-14 | 2005-08-04 | Cell Genesys, Inc., Foster City | Methoden zur erhöhung der effizienz rekombinanter aav produkte |
| US6156303A (en) | 1997-06-11 | 2000-12-05 | University Of Washington | Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom |
| US6251677B1 (en) | 1997-08-25 | 2001-06-26 | The Trustees Of The University Of Pennsylvania | Hybrid adenovirus-AAV virus and methods of use thereof |
| AU9319198A (en) | 1997-09-19 | 1999-04-05 | Trustees Of The University Of Pennsylvania, The | Methods and vector constructs useful for production of recombinant aav |
| JP2001517454A (ja) | 1997-09-19 | 2001-10-09 | 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 | 組換えアデノ随伴ウイルスの産生に有用な方法および細胞株 |
| CA2304131A1 (en) | 1997-09-19 | 1999-04-01 | James M. Wilson | Method for gene transfer using bcl2 and compositions useful therein |
| EP1064393B1 (en) | 1998-03-20 | 2004-12-29 | The Trustees Of The University Of Pennsylvania | Compositions and methods for helper-free production of recombinant adeno-associated viruses |
| US6953690B1 (en) | 1998-03-20 | 2005-10-11 | The Trustees Of The University Of Pennsylvania | Compositions and methods for helper-free production of recombinant adeno-associated viruses |
| US6146874A (en) | 1998-05-27 | 2000-11-14 | University Of Florida | Method of preparing recombinant adeno-associated virus compositions |
| ES2313784T3 (es) | 1998-05-28 | 2009-03-01 | The Government Of The Usa, As Represented By The Secretary, Department Of Health And Human Services | Vector aav5 y usos del mismo. |
| US6759237B1 (en) | 1998-11-05 | 2004-07-06 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same |
| ATE362542T1 (de) | 1998-11-05 | 2007-06-15 | Univ Pennsylvania | Nukleinsäuresequenzen des adeno-assoziierten virus des serotyps i, und vektoren und wirtszellen, die diese enthalten |
| AU780231B2 (en) | 1998-11-10 | 2005-03-10 | University Of North Carolina At Chapel Hill, The | Virus vectors and methods of making and administering the same |
| US6387368B1 (en) | 1999-02-08 | 2002-05-14 | The Trustees Of The University Of Pennsylvania | Hybrid adenovirus-AAV virus and methods of use thereof |
| JP4693244B2 (ja) | 1999-03-18 | 2011-06-01 | 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 | 組換えアデノ随伴ウイルスのヘルパー無しの生産のための組成物および方法 |
| US6258595B1 (en) | 1999-03-18 | 2001-07-10 | The Trustees Of The University Of Pennsylvania | Compositions and methods for helper-free production of recombinant adeno-associated viruses |
| JP2003501067A (ja) | 1999-06-02 | 2003-01-14 | 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 | ヘルパーウイルスを要求する組み換えウイルス産生のために有用な組成物及び方法 |
| DE60039766D1 (de) | 1999-08-09 | 2008-09-18 | Targeted Genetics Corp | Terologen nukleotidsequenz von einem rekombinanten viralen vektor durch ausgestaltung der sequenz in einer art und weise, dass basenpaarungen innerhalb der sequenz entstehen |
| EP1218035A2 (en) | 1999-09-29 | 2002-07-03 | The Trustees Of The University Of Pennsylvania | Rapid peg-modification of viral vectors |
| US6365394B1 (en) | 1999-09-29 | 2002-04-02 | The Trustees Of The University Of Pennsylvania | Cell lines and constructs useful in production of E1-deleted adenoviruses in absence of replication competent adenovirus |
| CA2385823A1 (en) | 1999-10-01 | 2001-04-12 | Haifeng Chen | Production of recombinant aav using adenovirus comprising aav rep/cap genes |
| US7115391B1 (en) | 1999-10-01 | 2006-10-03 | Genovo, Inc. | Production of recombinant AAV using adenovirus comprising AAV rep/cap genes |
| JP2003523180A (ja) | 1999-12-03 | 2003-08-05 | 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 | 多重のパッケージングシグナルを用いる組み換えアデノウイルスのパッケージングおよび収量を増加するための組成物および方法 |
| US6821512B1 (en) | 1999-12-03 | 2004-11-23 | The Trustees Of The University Of Pennsylvania | Compositions and methods for increasing packaging and yield of recombinant adenoviruses using multiple packaging signals |
| US6855314B1 (en) | 2000-03-22 | 2005-02-15 | The United States Of America As Represented By The Department Of Health And Human Services | AAV5 vector for transducing brain cells and lung cells |
| US6468524B1 (en) | 2000-03-22 | 2002-10-22 | The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services | AAV4 vector and uses thereof |
| CA2406743A1 (en) | 2000-04-28 | 2001-11-08 | The Trustees Of The University Of Pennsylvania | Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids |
| WO2001091802A1 (en) * | 2000-05-30 | 2001-12-06 | Baylor College Of Medicine | Chimeric viral vectors for gene therapy |
| AU6814901A (en) | 2000-06-01 | 2001-12-11 | Univ North Carolina | Methods and compounds for controlled release of recombinant parvovirus vectors |
| WO2002018659A2 (en) | 2000-08-30 | 2002-03-07 | Haplogen, Llc | Method for determining alleles |
| EP1392868B2 (en) | 2001-05-18 | 2013-09-04 | Wisconsin Alumni Research Foundation | Method for the synthesis of dna sequences using photo-labile linkers |
| AU2002324700A1 (en) | 2001-08-14 | 2003-03-03 | Bayer Ag | Nucleic acid and amino acid sequences involved in pain |
| AU2002347813A1 (en) * | 2001-10-04 | 2003-04-14 | Carlos Estuardo Aguilar-Cordova | Chimeric viral vectors for gene therapy |
| CA2915124C (en) * | 2001-11-13 | 2018-08-14 | The Trustees Of The University Of Pennsylvania | A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby |
| JP4769417B2 (ja) | 2001-12-17 | 2011-09-07 | 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 | アデノ随伴ウイルス（ａａｖ）血清型９の配列、それを含むベクターおよびその使用 |
| ES2602352T3 (es) | 2001-12-17 | 2017-02-20 | The Trustees Of The University Of Pennsylvania | Secuencias de serotipo 8 de virus adenoasociado (VAA), vectores que las contienen y usos de las mismas |
| WO2003104392A2 (en) | 2001-12-18 | 2003-12-18 | University Of North Carolina At Chapel Hill | Improved reagents and methods for producing parvoviruses |
| WO2003104413A2 (en) | 2002-06-05 | 2003-12-18 | University Of Florida | Production of pseudotyped recombinant aav virions |
| EP2298926A1 (en) | 2003-09-30 | 2011-03-23 | The Trustees of The University of Pennsylvania | Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof |
| EP2357010B1 (en) | 2005-04-07 | 2013-06-12 | The Trustees of The University of Pennsylvania | Method of increasing the function of an AAV vector |
| US7456683B2 (en) | 2005-06-09 | 2008-11-25 | Panasonic Corporation | Amplitude error compensating device and quadrature skew error compensating device |
| EP2007795B1 (en) | 2006-03-30 | 2016-11-16 | The Board Of Trustees Of The Leland Stanford Junior University | Aav capsid proteins |
| DK2094086T3 (da) | 2006-11-08 | 2013-11-25 | Veritas Bio LLC | Indgivelse in vivo af dobbeltstrenget rna til en målcelle |
| EP2058401A1 (en) | 2007-10-05 | 2009-05-13 | Genethon | Widespread gene delivery to motor neurons using peripheral injection of AAV vectors |
| JP5797551B2 (ja) * | 2008-04-22 | 2015-10-21 | フエー・イー・ベー・フエー・ゼツト・ウエー | 肝特異的核酸調節要素ならびにその方法および用途 |
| CA3065577C (en) | 2008-05-13 | 2022-05-31 | Phaserx, Inc. | Diblock copolymers and polynucleotide complexes thereof for delivery into cells |
| JP5766611B2 (ja) | 2008-11-06 | 2015-08-19 | ユニヴァーシティ オブ ワシントン | マルチブロック共重合体 |
| JP2012507581A (ja) | 2008-11-06 | 2012-03-29 | ユニヴァーシティ オブ ワシントン | 二重特異性細胞内送達媒体 |
| MX353900B (es) | 2008-11-07 | 2018-02-01 | Massachusetts Inst Technology | Lipidoides de aminoalcohol y usos de los mismos. |
| WO2011011584A1 (en) | 2009-07-24 | 2011-01-27 | Immune Design Corp | Lentiviral vectors pseudotyped with a sindbis virus envelope glycoprotein |
| EP2281579A1 (en) | 2009-08-05 | 2011-02-09 | BioNTech AG | Vaccine composition comprising 5'-Cap modified RNA |
| SG10201502270TA (en) | 2010-03-29 | 2015-05-28 | Univ Pennsylvania | Pharmacologically induced transgene ablation system |
| US9315825B2 (en) | 2010-03-29 | 2016-04-19 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
| CN103534417B (zh) | 2011-05-12 | 2016-03-23 | 日立建机株式会社 | 工程机械 |
| US20140206753A1 (en) | 2011-06-08 | 2014-07-24 | Shire Human Genetic Therapies, Inc. | Lipid nanoparticle compositions and methods for mrna delivery |
| FR2977562B1 (fr) | 2011-07-06 | 2016-12-23 | Gaztransport Et Technigaz | Cuve etanche et thermiquement isolante integree dans une structure porteuse |
| PE20181541A1 (es) | 2011-10-27 | 2018-09-26 | Massachusetts Inst Technology | Derivados de aminoacidos funcionalizados en la terminal n capaces de formar microesferas encapsuladoras de farmaco |
| WO2013143555A1 (en) | 2012-03-26 | 2013-10-03 | Biontech Ag | Rna formulation for immunotherapy |
| DE18200782T1 (de) | 2012-04-02 | 2021-10-21 | Modernatx, Inc. | Modifizierte polynukleotide zur herstellung von proteinen im zusammenhang mit erkrankungen beim menschen |
| AU2013243949A1 (en) * | 2012-04-02 | 2014-10-30 | Moderna Therapeutics, Inc. | Modified polynucleotides for the production of biologics and proteins associated with human disease |
| WO2013182683A1 (en) | 2012-06-08 | 2013-12-12 | Ethris Gmbh | Pulmonary delivery of messenger rna |
| WO2015017519A1 (en) * | 2013-07-30 | 2015-02-05 | Phaserx, Inc. | Block copolymers and their conjugates or complexes with oligonucleotides |
| RS60902B1 (sr) | 2014-03-09 | 2020-11-30 | Univ Pennsylvania | Kompozicije korisne u lečenju nedostatka ornitinske transkarbamilaze (otc) |
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