ES2856403T3 - Composiciones a base de rAAV y procedimientos para el tratamiento de la esclerosis lateral amiotrofica - Google Patents
Composiciones a base de rAAV y procedimientos para el tratamiento de la esclerosis lateral amiotrofica Download PDFInfo
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- ES2856403T3 ES2856403T3 ES15764861T ES15764861T ES2856403T3 ES 2856403 T3 ES2856403 T3 ES 2856403T3 ES 15764861 T ES15764861 T ES 15764861T ES 15764861 T ES15764861 T ES 15764861T ES 2856403 T3 ES2856403 T3 ES 2856403T3
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- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
- C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
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- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
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| US9217155B2 (en) | 2008-05-28 | 2015-12-22 | University Of Massachusetts | Isolation of novel AAV'S and uses thereof |
| SG171914A1 (en) | 2008-12-02 | 2011-07-28 | Chiralgen Ltd | Method for the synthesis of phosphorus atom modified nucleic acids |
| WO2010138263A2 (en) | 2009-05-28 | 2010-12-02 | University Of Massachusetts | Novel aav 's and uses thereof |
| IN2012DN00720A (OSRAM) | 2009-07-06 | 2015-06-19 | Ontorii Inc | |
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| WO2011133874A1 (en) | 2010-04-23 | 2011-10-27 | University Of Massachusetts | Multicistronic expression constructs |
| DK2826860T3 (en) | 2010-04-23 | 2018-12-03 | Univ Massachusetts | CNS targeting AAV vectors and methods for their use |
| WO2012039448A1 (ja) | 2010-09-24 | 2012-03-29 | 株式会社キラルジェン | 不斉補助基 |
| EP3318635A1 (en) | 2011-04-21 | 2018-05-09 | University of Massachusetts | Raav-based compositions and methods for treating alpha-1 anti-trypsin deficiencies |
| MX347361B (es) | 2011-07-19 | 2017-04-12 | Wave Life Sciences Ltd | Metodos para la sintesis de acidos nucleicos funcionalizados. |
| EP2872485B1 (en) | 2012-07-13 | 2020-12-16 | Wave Life Sciences Ltd. | Asymmetric auxiliary group |
| CN106068325B (zh) | 2014-01-16 | 2021-07-09 | 波涛生命科学有限公司 | 手性设计 |
| WO2015127128A2 (en) | 2014-02-19 | 2015-08-27 | University Of Massachusetts | Recombinant aavs having useful transcytosis properties |
| EP4410805A3 (en) | 2014-03-18 | 2024-11-27 | University of Massachusetts | Raav-based compositions and methods for treating amyotrophic lateral sclerosis |
| WO2015164786A1 (en) | 2014-04-25 | 2015-10-29 | University Of Massachusetts | Recombinant aav vectors useful for reducing immunity against transgene products |
| WO2015187825A2 (en) | 2014-06-03 | 2015-12-10 | University Of Massachusetts | Compositions and methods for modulating dysferlin expression |
| WO2016054554A1 (en) | 2014-10-03 | 2016-04-07 | University Of Massachusetts | Heterologous targeting peptide grafted aavs |
| EP3209311B1 (en) | 2014-10-21 | 2024-03-06 | University of Massachusetts | Recombinant aav variants and uses thereof |
| MX2017006217A (es) | 2014-11-14 | 2018-05-02 | Voyager Therapeutics Inc | Polinucleotidos moduladores. |
| US10597660B2 (en) | 2014-11-14 | 2020-03-24 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (ALS) |
| US10584321B2 (en) | 2015-02-13 | 2020-03-10 | University Of Massachusetts | Compositions and methods for transient delivery of nucleases |
| EP3285780A4 (en) | 2015-04-24 | 2018-12-19 | University of Massachusetts | Modified aav constructions and uses thereof |
| US10285388B2 (en) | 2015-05-29 | 2019-05-14 | Regeneron Pharmaceuticals, Inc. | Non-human animals having a disruption in a C9ORF72 locus |
| US11253576B2 (en) | 2015-10-22 | 2022-02-22 | University Of Massachusetts | Methods and compositions for treating metabolic imbalance in neurodegenerative disease |
| EP3364996B1 (en) | 2015-10-22 | 2021-08-25 | University of Massachusetts | Prostate-targeting adeno-associated virus serotype vectors |
| WO2017136536A1 (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 |
| EP4094780A3 (en) | 2016-02-12 | 2023-02-08 | University of Massachusetts | Anti-angiogenic mirna therapeutics for inhibiting corneal neovascularization |
| MA44490A (fr) | 2016-03-22 | 2019-01-30 | Harvard College | Adhésifs biocompatibles et leurs procédés d'utilisation |
| EP4008356A1 (en) * | 2016-03-31 | 2022-06-08 | University of Cincinnati | Methods and compositions for the treatment of als |
| EP4335502A3 (en) | 2016-04-02 | 2024-07-03 | Research Institute at Nationwide Children's Hospital | Modified u6 promoter system for tissue specific expression |
| US11207426B2 (en) | 2016-04-05 | 2021-12-28 | University Of Massachusetts | Compositions and methods for selective inhibition of grainyhead-like protein expression |
| WO2017181105A1 (en) | 2016-04-15 | 2017-10-19 | University Of Massachusetts | Methods and compositions for treating metabolic imbalance |
| KR20240056729A (ko) | 2016-05-18 | 2024-04-30 | 보이저 테라퓨틱스, 인크. | 조절성 폴리뉴클레오티드 |
| MX2018014152A (es) * | 2016-05-18 | 2019-03-28 | Voyager Therapeutics Inc | Composiciones y métodos para tratar la enfermedad de huntington. |
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| US11882815B2 (en) | 2016-06-15 | 2024-01-30 | University Of Massachusetts | Recombinant adeno-associated viruses for delivering gene editing molecules to embryonic cells |
| US10457940B2 (en) | 2016-09-22 | 2019-10-29 | University Of Massachusetts | AAV treatment of Huntington's disease |
| EP3518667B1 (en) * | 2016-09-30 | 2024-10-30 | Regeneron Pharmaceuticals, Inc. | Rodents having a hexanucleotide repeat expansion in a c9orf72 locus |
| AU2017341849B2 (en) | 2016-10-13 | 2024-03-21 | University Of Massachusetts | AAV capsid designs |
| JP6730525B2 (ja) | 2016-11-21 | 2020-07-29 | ナノストリング テクノロジーズ，インコーポレイティド | 化学組成物とそれを利用する方法 |
| EP3619310A4 (en) * | 2017-05-05 | 2021-01-27 | Voyager Therapeutics, Inc. | Modulatory polynucleotides |
| AU2018261790B2 (en) | 2017-05-05 | 2024-10-03 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (ALS) |
| EP3622073A4 (en) | 2017-05-09 | 2021-01-06 | University of Massachusetts | Methods of treating amyotrophic lateral sclerosis (als) |
| WO2019051025A2 (en) * | 2017-09-06 | 2019-03-14 | The Trustees Of Columbia University In The City Of New York | TREATMENT OF AGGRESSIVE CANCERS BY TARGETING C9ORF72 |
| WO2019060686A1 (en) * | 2017-09-22 | 2019-03-28 | University Of Massachusetts | NEW DUAL EXPRESSION VECTORS OF SOD1 AND USES THEREOF |
| WO2019079240A1 (en) | 2017-10-16 | 2019-04-25 | Voyager Therapeutics, Inc. | TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS) |
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| US20230070049A1 (en) * | 2020-02-03 | 2023-03-09 | Rutgers, The State University Of New Jersey | Microrna-7 compositions for promoting functional recovery following spinal cord injury and methods of use thereof |
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