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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- Prior art keywords
- describe
- alkyl
- agent
- cells
- trifluoromethyl
- Prior art date
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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 (cg-RX-API-DMAC7.html) | 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 (cg-RX-API-DMAC7.html) |
| EP (1) | EP1190249A2 (cg-RX-API-DMAC7.html) |
| JP (1) | JP4969002B2 (cg-RX-API-DMAC7.html) |
| AU (1) | AU782966B2 (cg-RX-API-DMAC7.html) |
| CA (1) | CA2376400A1 (cg-RX-API-DMAC7.html) |
| WO (1) | WO2000075365A2 (cg-RX-API-DMAC7.html) |
Families Citing this family (36)
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|---|---|---|---|---|
| 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 |
| US20050095225A1 (en) * | 2003-03-31 | 2005-05-05 | Engelhardt John F. | Compounds and methods for pharmico-gene therapy of epithelial sodium channel associated disorders |
| WO2007127464A2 (en) * | 2006-04-28 | 2007-11-08 | University Of Iowa Research Foundation | Methods and compounds to modulate parvovirus transduction of mammalian cells or to alter virus infection, method to identify a viral receptor or co-receptor |
| CA2788682C (en) * | 2010-02-05 | 2019-03-05 | The University Of North Carolina At Chapel Hill | Compositions and methods for enhanced parvovirus transduction |
| WO2011117258A2 (en) * | 2010-03-22 | 2011-09-29 | Association Institut De Myologie | Methods of increasing efficiency of vector penetration of target tissue |
| US10577627B2 (en) | 2014-06-09 | 2020-03-03 | Voyager Therapeutics, Inc. | Chimeric capsids |
| CN112375760A (zh) | 2014-11-14 | 2021-02-19 | 沃雅戈治疗公司 | 调节性多核苷酸 |
| WO2016077687A1 (en) | 2014-11-14 | 2016-05-19 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
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| US11702672B2 (en) | 2016-02-08 | 2023-07-18 | University Of Iowa Research Foundation | Methods to produce chimeric adeno-associated virus/bocavirus parvovirus |
| MX2018010842A (es) | 2016-03-07 | 2019-07-04 | Univ Iowa Res Found | Expresion mediada por el virus adeno-asociado (aav) usando un promotor y pontenciador sintetico. |
| US11299751B2 (en) | 2016-04-29 | 2022-04-12 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| WO2017189959A1 (en) | 2016-04-29 | 2017-11-02 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| SG11201809643UA (en) | 2016-05-18 | 2018-12-28 | Voyager Therapeutics Inc | Compositions and methods of treating huntington's disease |
| IL302748B1 (en) | 2016-05-18 | 2025-09-01 | Voyager Therapeutics Inc | Modulatory polynucleotides |
| US12173305B2 (en) | 2016-05-26 | 2024-12-24 | 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 |
| US11752181B2 (en) | 2017-05-05 | 2023-09-12 | Voyager Therapeutics, Inc. | Compositions and methods of treating Huntington's disease |
| EP3618839A4 (en) | 2017-05-05 | 2021-06-09 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| EP3654860A1 (en) | 2017-07-17 | 2020-05-27 | Voyager Therapeutics, Inc. | Trajectory array guide system |
| CN111448308A (zh) | 2017-08-03 | 2020-07-24 | 沃雅戈治疗公司 | 递送aav的组合物和方法 |
| US20190085358A1 (en) * | 2017-08-25 | 2019-03-21 | Ovid Therapeutics Inc. | Recombinant adeno-associated vectors |
| WO2019067840A1 (en) | 2017-09-29 | 2019-04-04 | Voyager Therapeutics, Inc. | RESTRICTING CENTRAL AND PERIPHERAL NEUROLOGICAL PHENOTYPE FROM FRIEDREICH ATAXIA BY INTRAVENOUS ADMINISTRATION |
| WO2019079242A1 (en) | 2017-10-16 | 2019-04-25 | Voyager Therapeutics, Inc. | TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
| CN111479924B (zh) | 2017-10-16 | 2024-06-14 | 沃雅戈治疗公司 | 肌萎缩性侧索硬化症(als)的治疗 |
| EP3856762A1 (en) | 2018-09-28 | 2021-08-04 | Voyager Therapeutics, Inc. | Frataxin expression constructs having engineered promoters and methods of use thereof |
| IL297753A (en) | 2020-04-29 | 2022-12-01 | Bristol Myers Squibb Co | Minimized dystrophins with spectrin fusion domains and uses thereof |
| WO2025222013A1 (en) * | 2024-04-17 | 2025-10-23 | Solid Biosciences Inc. | Recombinant aav production process |
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| WO2002028348A2 (en) * | 2000-10-04 | 2002-04-11 | The Children's Hospital Of Philadelphia | 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 |
| CA2482124C (en) * | 2001-04-19 | 2008-10-07 | Uab Research Foundation | Methods of treatment using an epithelial sodium channel blocker and an inhibitor of the mineralocorticoid receptor |
| US20040072254A1 (en) | 2001-05-01 | 2004-04-15 | Senomyx, Inc. | High throughput cell-based assay for monitoring sodium channel activity and discovery of salty taste modulating compounds |
| US20030003583A1 (en) * | 2001-06-08 | 2003-01-02 | Raphael Hirsch | Regulation of transgene expression following AAV transduction |
| WO2003006616A2 (en) * | 2001-07-13 | 2003-01-23 | University Of Iowa Research Foundation | Pseudotyped adeno-associated 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 |
| EP1478655A4 (en) | 2001-12-31 | 2006-05-10 | Algos Therapeutics Inc | METHODS AND SUBSTANCES FOR ENAC-BETA MODULATION |
| WO2003095667A2 (en) | 2002-05-13 | 2003-11-20 | Arexis Ab | Autoimmune conditions and nadph oxidase defects |
| US20050095225A1 (en) * | 2003-03-31 | 2005-05-05 | Engelhardt John F. | Compounds and methods for pharmico-gene therapy of epithelial sodium channel associated disorders |
| EP1486567A1 (en) | 2003-06-11 | 2004-12-15 | Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts | Improved adeno-associated virus (AAV) vector for gene therapy |
| WO2004112727A2 (en) | 2003-06-19 | 2004-12-29 | Avigen, Inc. | Aav virions with decreased immunoreactivity and uses therefor |
| 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 |
| WO2007127464A2 (en) | 2006-04-28 | 2007-11-08 | University Of Iowa Research Foundation | Methods and compounds to modulate parvovirus transduction of mammalian cells or to alter virus infection, method to identify a viral receptor or co-receptor |
| JPWO2009028387A1 (ja) * | 2007-08-24 | 2010-12-02 | 協和発酵キリン株式会社 | プロテアーゼ阻害剤耐性を有する癌の治療薬 |
-
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 EP EP00944624A patent/EP1190249A2/en not_active Withdrawn
- 2000-06-08 JP JP2001501645A patent/JP4969002B2/ja not_active Expired - Lifetime
- 2000-06-08 AU AU58694/00A patent/AU782966B2/en not_active Expired
-
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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