JP2003501068A5 - - Google Patents
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- JP2003501068A5 JP2003501068A5 JP2001501645A JP2001501645A JP2003501068A5 JP 2003501068 A5 JP2003501068 A5 JP 2003501068A5 JP 2001501645 A JP2001501645 A JP 2001501645A JP 2001501645 A JP2001501645 A JP 2001501645A JP 2003501068 A5 JP2003501068 A5 JP 2003501068A5
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- describe
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- agent
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- trifluoromethyl
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- 238000000034 method Methods 0.000 description 23
- 125000000217 alkyl group Chemical group 0.000 description 19
- 239000003795 chemical substances by application Substances 0.000 description 15
- 210000004027 cell Anatomy 0.000 description 10
- 229910052736 halogen Inorganic materials 0.000 description 10
- 150000002367 halogens Chemical class 0.000 description 10
- 125000002023 trifluoromethyl group Chemical group FC(F)(F)* 0.000 description 10
- 125000003545 alkoxy group Chemical group 0.000 description 9
- 125000003342 alkenyl group Chemical group 0.000 description 8
- 125000000304 alkynyl group Chemical group 0.000 description 8
- 239000003814 drug Substances 0.000 description 8
- 229910052739 hydrogen Inorganic materials 0.000 description 8
- 239000001257 hydrogen Substances 0.000 description 8
- 125000001589 carboacyl group Chemical group 0.000 description 7
- 241000700605 Viruses Species 0.000 description 6
- 229940024606 amino acid Drugs 0.000 description 6
- 235000001014 amino acid Nutrition 0.000 description 6
- 150000001413 amino acids Chemical class 0.000 description 6
- 229940079593 drug Drugs 0.000 description 6
- 125000002485 formyl group Chemical group [H]C(*)=O 0.000 description 6
- 125000004435 hydrogen atom Chemical group [H]* 0.000 description 6
- 125000001584 benzyloxycarbonyl group Chemical group C(=O)(OCC1=CC=CC=C1)* 0.000 description 5
- 210000004962 mammalian cell Anatomy 0.000 description 5
- UYWQUFXKFGHYNT-UHFFFAOYSA-N phenylmethyl ester of formic acid Chemical group O=COCC1=CC=CC=C1 UYWQUFXKFGHYNT-UHFFFAOYSA-N 0.000 description 5
- DHMQDGOQFOQNFH-UHFFFAOYSA-N Glycine Chemical group NCC(O)=O DHMQDGOQFOQNFH-UHFFFAOYSA-N 0.000 description 4
- 125000002777 acetyl group Chemical group [H]C([H])([H])C(*)=O 0.000 description 4
- 125000003118 aryl group Chemical group 0.000 description 4
- 125000003178 carboxy group Chemical group [H]OC(*)=O 0.000 description 4
- 125000000753 cycloalkyl group Chemical group 0.000 description 4
- 229960000310 isoleucine Drugs 0.000 description 4
- AGPKZVBTJJNPAG-UHFFFAOYSA-N isoleucine Chemical group CCC(C)C(N)C(O)=O AGPKZVBTJJNPAG-UHFFFAOYSA-N 0.000 description 4
- 150000003839 salts Chemical class 0.000 description 4
- 230000026683 transduction Effects 0.000 description 4
- 238000010361 transduction Methods 0.000 description 4
- SNDPXSYFESPGGJ-BYPYZUCNSA-N L-2-aminopentanoic acid Chemical group CCC[C@H](N)C(O)=O SNDPXSYFESPGGJ-BYPYZUCNSA-N 0.000 description 3
- AGPKZVBTJJNPAG-WHFBIAKZSA-N L-isoleucine Chemical group CC[C@H](C)[C@H](N)C(O)=O AGPKZVBTJJNPAG-WHFBIAKZSA-N 0.000 description 3
- SNDPXSYFESPGGJ-UHFFFAOYSA-N L-norVal-OH Chemical group CCCC(N)C(O)=O SNDPXSYFESPGGJ-UHFFFAOYSA-N 0.000 description 3
- LRQKBLKVPFOOQJ-YFKPBYRVSA-N L-norleucine Chemical group CCCC[C@H]([NH3+])C([O-])=O LRQKBLKVPFOOQJ-YFKPBYRVSA-N 0.000 description 3
- 125000001429 N-terminal alpha-amino-acid group Chemical group 0.000 description 3
- 125000000959 isobutyl group Chemical group [H]C([H])([H])C([H])(C([H])([H])[H])C([H])([H])* 0.000 description 3
- 229910052760 oxygen Inorganic materials 0.000 description 3
- 108090000765 processed proteins & peptides Proteins 0.000 description 3
- 229910052717 sulfur Inorganic materials 0.000 description 3
- 239000004475 Arginine Chemical group 0.000 description 2
- 241000702421 Dependoparvovirus Species 0.000 description 2
- 239000004471 Glycine Chemical group 0.000 description 2
- ODKSFYDXXFIFQN-BYPYZUCNSA-P L-argininium(2+) Chemical group NC(=[NH2+])NCCC[C@H]([NH3+])C(O)=O ODKSFYDXXFIFQN-BYPYZUCNSA-P 0.000 description 2
- ROHFNLRQFUQHCH-YFKPBYRVSA-N L-leucine Chemical group CC(C)C[C@H](N)C(O)=O ROHFNLRQFUQHCH-YFKPBYRVSA-N 0.000 description 2
- KZSNJWFQEVHDMF-BYPYZUCNSA-N L-valine Chemical group CC(C)[C@H](N)C(O)=O KZSNJWFQEVHDMF-BYPYZUCNSA-N 0.000 description 2
- ROHFNLRQFUQHCH-UHFFFAOYSA-N Leucine Chemical group CC(C)CC(N)C(O)=O ROHFNLRQFUQHCH-UHFFFAOYSA-N 0.000 description 2
- 102000044159 Ubiquitin Human genes 0.000 description 2
- 108090000848 Ubiquitin Proteins 0.000 description 2
- KZSNJWFQEVHDMF-UHFFFAOYSA-N Valine Chemical group CC(C)C(N)C(O)=O KZSNJWFQEVHDMF-UHFFFAOYSA-N 0.000 description 2
- 235000004279 alanine Nutrition 0.000 description 2
- ODKSFYDXXFIFQN-UHFFFAOYSA-N arginine Chemical group OC(=O)C(N)CCCNC(N)=N ODKSFYDXXFIFQN-UHFFFAOYSA-N 0.000 description 2
- 230000015572 biosynthetic process Effects 0.000 description 2
- 230000000903 blocking effect Effects 0.000 description 2
- 125000000484 butyl group Chemical group [H]C([*])([H])C([H])([H])C([H])([H])C([H])([H])[H] 0.000 description 2
- 150000001875 compounds Chemical class 0.000 description 2
- 239000012528 membrane Substances 0.000 description 2
- -1 methyl Chemical group 0.000 description 2
- 125000001436 propyl group Chemical group [H]C([*])([H])C([H])([H])C([H])([H])[H] 0.000 description 2
- 108090000623 proteins and genes Proteins 0.000 description 2
- 238000003786 synthesis reaction Methods 0.000 description 2
- 239000004474 valine Chemical group 0.000 description 2
- 230000003612 virological effect Effects 0.000 description 2
- 125000001433 C-terminal amino-acid group Chemical group 0.000 description 1
- 101000749287 Clitocybe nebularis Clitocypin Proteins 0.000 description 1
- 101000767029 Clitocybe nebularis Clitocypin-1 Proteins 0.000 description 1
- 229940094664 Cysteine protease inhibitor Drugs 0.000 description 1
- FRJIAZKQGSCKPQ-FSPLSTOPSA-N His-Ala Chemical compound OC(=O)[C@H](C)NC(=O)[C@@H](N)CC1=CN=CN1 FRJIAZKQGSCKPQ-FSPLSTOPSA-N 0.000 description 1
- UFHFLCQGNIYNRP-UHFFFAOYSA-N Hydrogen Chemical compound [H][H] UFHFLCQGNIYNRP-UHFFFAOYSA-N 0.000 description 1
- QNAYBMKLOCPYGJ-REOHCLBHSA-N L-alanine Chemical compound C[C@H](N)C(O)=O QNAYBMKLOCPYGJ-REOHCLBHSA-N 0.000 description 1
- HSQGMTRYSIHDAC-BQBZGAKWSA-N Leu-Ala Chemical group CC(C)C[C@H](N)C(=O)N[C@@H](C)C(O)=O HSQGMTRYSIHDAC-BQBZGAKWSA-N 0.000 description 1
- 241001529936 Murinae Species 0.000 description 1
- 241000283973 Oryctolagus cuniculus Species 0.000 description 1
- 229940124158 Protease/peptidase inhibitor Drugs 0.000 description 1
- 102000006275 Ubiquitin-Protein Ligases Human genes 0.000 description 1
- 108010083111 Ubiquitin-Protein Ligases Proteins 0.000 description 1
- 102100020696 Ubiquitin-conjugating enzyme E2 K Human genes 0.000 description 1
- 230000004913 activation Effects 0.000 description 1
- 125000002252 acyl group Chemical group 0.000 description 1
- 125000003295 alanine group Chemical group N[C@@H](C)C(=O)* 0.000 description 1
- 150000001408 amides Chemical class 0.000 description 1
- 125000003277 amino group Chemical group 0.000 description 1
- 230000000694 effects Effects 0.000 description 1
- DEFVIWRASFVYLL-UHFFFAOYSA-N ethylene glycol bis(2-aminoethyl)tetraacetic acid Chemical group OC(=O)CN(CC(O)=O)CCOCCOCCN(CC(O)=O)CC(O)=O DEFVIWRASFVYLL-UHFFFAOYSA-N 0.000 description 1
- 210000003494 hepatocyte Anatomy 0.000 description 1
- 210000005260 human cell Anatomy 0.000 description 1
- 150000002431 hydrogen Chemical group 0.000 description 1
- 125000002887 hydroxy group Chemical group [H]O* 0.000 description 1
- 125000000741 isoleucyl group Chemical group [H]N([H])C(C(C([H])([H])[H])C([H])([H])C([H])([H])[H])C(=O)O* 0.000 description 1
- 210000004072 lung Anatomy 0.000 description 1
- 238000004519 manufacturing process Methods 0.000 description 1
- 239000003550 marker Substances 0.000 description 1
- 125000002496 methyl group Chemical group [H]C([H])([H])* 0.000 description 1
- 239000000137 peptide hydrolase inhibitor Substances 0.000 description 1
- 125000001997 phenyl group Chemical group [H]C1=C([H])C([H])=C(*)C([H])=C1[H] 0.000 description 1
- 230000001225 therapeutic effect Effects 0.000 description 1
- 125000004044 trifluoroacetyl group Chemical group FC(C(=O)*)(F)F 0.000 description 1
- 108010084736 ubiquitin carrier proteins Proteins 0.000 description 1
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US13818899P | 1999-06-08 | 1999-06-08 | |
| US60/138,188 | 1999-06-08 | ||
| US20108900P | 2000-05-02 | 2000-05-02 | |
| US60/201,089 | 2000-05-02 | ||
| PCT/US2000/015700 WO2000075365A2 (en) | 1999-06-08 | 2000-06-08 | Compounds and methods to enhance raav transduction |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2003501068A JP2003501068A (ja) | 2003-01-14 |
| JP2003501068A5 true JP2003501068A5 (enExample) | 2007-08-09 |
| JP4969002B2 JP4969002B2 (ja) | 2012-07-04 |
Family
ID=26835948
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2001501645A Expired - Lifetime JP4969002B2 (ja) | 1999-06-08 | 2000-06-08 | ｒＡＡＶ形質導入を増加するための化合物および方法 |
Country Status (6)
| Country | Link |
|---|---|
| US (2) | US8846030B2 (enExample) |
| EP (1) | EP1190249A2 (enExample) |
| JP (1) | JP4969002B2 (enExample) |
| AU (1) | AU782966B2 (enExample) |
| CA (1) | CA2376400A1 (enExample) |
| WO (1) | WO2000075365A2 (enExample) |
Families Citing this family (36)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6924128B2 (en) | 1994-12-06 | 2005-08-02 | Targeted Genetics Corporation | Packaging cell lines for generation of high titers of recombinant AAV vectors |
| US6989264B2 (en) | 1997-09-05 | 2006-01-24 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| US6346415B1 (en) | 1997-10-21 | 2002-02-12 | Targeted Genetics Corporation | Transcriptionally-activated AAV inverted terminal repeats (ITRS) for use with recombinant AAV vectors |
| US6436392B1 (en) | 1998-05-20 | 2002-08-20 | University Of Iowa Research Foundation | Adeno-associated virus vectors |
| US6893865B1 (en) | 1999-04-28 | 2005-05-17 | Targeted Genetics Corporation | Methods, compositions, and cells for encapsidating recombinant vectors in AAV particles |
| WO2000075365A2 (en) | 1999-06-08 | 2000-12-14 | University Of Iowa Research Foundation | Compounds and methods to enhance raav transduction |
| US7241447B1 (en) * | 1999-10-07 | 2007-07-10 | University Of Iowa Research Foundation | Adeno-associated virus vectors and uses thereof |
| US8241622B2 (en) | 2001-07-13 | 2012-08-14 | University Of Iowa Research Foundation | Adeno-associated virus vectors with intravector heterologous terminal palindromic sequences |
| AU2004227915A1 (en) * | 2003-03-31 | 2004-10-21 | John F. Engelhardt | Pharmico-gene theraphy of epithelial sodium channel associated disoders |
| JP2009535034A (ja) * | 2006-04-28 | 2009-10-01 | ユニバーシテイ・オブ・アイオワ・リサーチ・フアウンデーシヨン | 哺乳動物細胞のパルボウイルス形質導入を調節若しくはウイルス感染を変えるための方法および化合物、ウイルス受容体若しくは共受容体の同定方法 |
| CA2788682C (en) * | 2010-02-05 | 2019-03-05 | The University Of North Carolina At Chapel Hill | Compositions and methods for enhanced parvovirus transduction |
| EP2550021A2 (en) * | 2010-03-22 | 2013-01-30 | Association Institut de Myologie | Methods of increasing efficiency of vector penetration of target tissue |
| WO2015191508A1 (en) | 2014-06-09 | 2015-12-17 | Voyager Therapeutics, Inc. | Chimeric capsids |
| KR102584655B1 (ko) | 2014-11-14 | 2023-10-06 | 보이저 테라퓨틱스, 인크. | 조절성 폴리뉴클레오티드 |
| MX2017006216A (es) | 2014-11-14 | 2018-08-29 | Voyager Therapeutics Inc | Composiciones y métodos para tratar la esclerosis lateral amiotrófica (ela). |
| US11697825B2 (en) | 2014-12-12 | 2023-07-11 | Voyager Therapeutics, Inc. | Compositions and methods for the production of scAAV |
| US11702672B2 (en) | 2016-02-08 | 2023-07-18 | University Of Iowa Research Foundation | Methods to produce chimeric adeno-associated virus/bocavirus parvovirus |
| EP3426787A1 (en) | 2016-03-07 | 2019-01-16 | University of Iowa Research Foundation | Aav-mediated expression using a synthetic promoter and enhancer |
| US11326182B2 (en) | 2016-04-29 | 2022-05-10 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| WO2017189964A2 (en) | 2016-04-29 | 2017-11-02 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| AU2017268382B2 (en) | 2016-05-18 | 2023-09-28 | Voyager Therapeutics, Inc. | Compositions and methods of treating Huntington's disease |
| RU2758488C2 (ru) | 2016-05-18 | 2021-10-28 | Вояджер Терапьютикс, Инк. | Модулирующие полинуклеотиды |
| WO2017205739A1 (en) | 2016-05-26 | 2017-11-30 | University Of Iowa Research Foundation | cis AND trans REQUIREMENTS FOR TERMINAL RESOLUTION OF HUMAN BOCAVIRUS 1 |
| JP2019531787A (ja) | 2016-08-30 | 2019-11-07 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア | 生物医学的ターゲティング及びデリバリーの方法並びにそれを実行するための装置及びシステム |
| US11142775B2 (en) | 2017-01-13 | 2021-10-12 | University Of Iowa Research Foundation | Bocaparvovirus small noncoding RNA and uses thereof |
| AU2018261790B2 (en) | 2017-05-05 | 2024-10-03 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (ALS) |
| WO2018204803A1 (en) | 2017-05-05 | 2018-11-08 | Voyager Therapeutics, Inc. | Compositions and methods of treating huntington's disease |
| CN111132626B (zh) | 2017-07-17 | 2024-01-30 | 沃雅戈治疗公司 | 轨迹阵列引导系统 |
| EP3662060A2 (en) | 2017-08-03 | 2020-06-10 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of aav |
| AU2018320849A1 (en) * | 2017-08-25 | 2020-03-05 | Ovid Therapeutics Inc. | Recombinant adeno-associated vectors |
| EP3687582A4 (en) | 2017-09-29 | 2021-07-14 | Voyager Therapeutics, Inc. | RESCUE OF CENTRAL AND PERIPHERAL NEUROLOGICAL PHENOTYPE OF FRIEDREICH ATAXIA BY INTRAVENOUS ADMINISTRATION |
| US11434502B2 (en) | 2017-10-16 | 2022-09-06 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (ALS) |
| EP4454654A3 (en) | 2017-10-16 | 2025-02-19 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (als) |
| SG11202103151RA (en) | 2018-09-28 | 2021-04-29 | Voyager Therapeutics Inc | Frataxin expression constructs having engineered promoters and methods of use thereof |
| MX2022013477A (es) | 2020-04-29 | 2022-11-30 | Bristol Myers Squibb Co | Distrofinas miniaturizadas con dominios de fusion de espectrina y usos de las mismas. |
| WO2025222013A1 (en) * | 2024-04-17 | 2025-10-23 | Solid Biosciences Inc. | Recombinant aav production process |
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| 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 |
| US20020128203A1 (en) * | 2000-09-20 | 2002-09-12 | Laurent Schild | Methods of identifying inhibitory compounds and uses thereof |
| AU2001289438A1 (en) | 2000-09-22 | 2002-04-02 | Galephar M/F | Pharmaceutical semi-solid composition of isotretinoin |
| US20020115619A1 (en) * | 2000-10-04 | 2002-08-22 | Rubenstein Ronald C. | Compositions and methods for treatment of cystic fibrosis |
| WO2002074156A2 (en) * | 2001-02-02 | 2002-09-26 | Corixa Corporation | Compositions and methods for the therapy and diagnosis of colon cancer |
| US20030013137A1 (en) | 2001-03-13 | 2003-01-16 | Barak Larry S. | Automated methods of detecting receptor activity |
| WO2002085367A1 (en) * | 2001-04-19 | 2002-10-31 | Uab Research Foundation | Methods of treatment using an epithelial sodium channel blocker and an inhibitor of the mineralocorticoid receptor |
| WO2002087306A2 (en) | 2001-05-01 | 2002-11-07 | Senomyx, Inc. | High throughput cell-based assay for monitoring sodium channel activity and discovery of salty taste modulating compounds |
| WO2002101012A2 (en) * | 2001-06-08 | 2002-12-19 | Children's Hospital Research Foundation | Regulation of transgene expression following aav transduction |
| EP1419245A4 (en) * | 2001-07-13 | 2006-04-05 | Univ Iowa Res Found | ADENO-ASSOCIATED PSEUDOTYPE VIRUSES AND USES THEREOF |
| US8241622B2 (en) * | 2001-07-13 | 2012-08-14 | University Of Iowa Research Foundation | Adeno-associated virus vectors with intravector heterologous terminal palindromic sequences |
| AU2002364612A1 (en) | 2001-12-31 | 2003-07-24 | Algos Therapeutics, Inc. | METHODS AND MATERIALS FOR MODULATING ENaC-BETA |
| DE60323091D1 (de) | 2002-05-13 | 2008-10-02 | Arexis Ab | Autoimmunerkrankungen und nadph-oxidase-defekte |
| AU2004227915A1 (en) | 2003-03-31 | 2004-10-21 | John F. Engelhardt | Pharmico-gene theraphy of epithelial sodium channel associated disoders |
| EP1486567A1 (en) | 2003-06-11 | 2004-12-15 | Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts | Improved adeno-associated virus (AAV) vector for gene therapy |
| PL3235827T3 (pl) | 2003-06-19 | 2021-07-05 | Genzyme Corporation | Wiriony AAV o zmniejszonej immunoreaktywności i ich zastosowania |
| US7655467B2 (en) | 2003-11-14 | 2010-02-02 | The University Of Washington | Compositions and methods for systemic nucleic acid sequence delivery |
| WO2005116224A2 (en) | 2004-05-18 | 2005-12-08 | Children's Memorial Hospital | Tetracycline-regulated adeno-associated viral (aav) vectors for gene delivery to the nervous system |
| EP1602926A1 (en) | 2004-06-04 | 2005-12-07 | University of Geneva | Novel means and methods for the treatment of hearing loss and phantom hearing |
| JP2009535034A (ja) * | 2006-04-28 | 2009-10-01 | ユニバーシテイ・オブ・アイオワ・リサーチ・フアウンデーシヨン | 哺乳動物細胞のパルボウイルス形質導入を調節若しくはウイルス感染を変えるための方法および化合物、ウイルス受容体若しくは共受容体の同定方法 |
| EP2184073A4 (en) * | 2007-08-24 | 2010-10-20 | Kyowa Hakko Kirin Co Ltd | AGAINST PROTEASE HEMMER RESISTANT CANCER THERAPEUTICS |
-
2000
- 2000-06-08 WO PCT/US2000/015700 patent/WO2000075365A2/en not_active Ceased
- 2000-06-08 CA CA002376400A patent/CA2376400A1/en not_active Abandoned
- 2000-06-08 AU AU58694/00A patent/AU782966B2/en not_active Expired
- 2000-06-08 JP JP2001501645A patent/JP4969002B2/ja not_active Expired - Lifetime
- 2000-06-08 EP EP00944624A patent/EP1190249A2/en not_active Withdrawn
-
2005
- 2005-12-13 US US11/301,601 patent/US8846030B2/en not_active Expired - Fee Related
-
2007
- 2007-08-07 US US11/890,777 patent/US20080213221A1/en not_active Abandoned
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