CL2012002969A1 - Vector viral adenoasociado recombinante (raav) que comprende promotor de rodopsina quinasa humana especifica del fotorreceptor, o promotor de beta-actina de pollo cmv quimerico truncado ubicuo unido a al menos un adn que codifica una guanilato ciclasa; virion o particula viral infecciosa que comprende el vector; celula huesped; composicion que comprende el vector. - Google Patents
Vector viral adenoasociado recombinante (raav) que comprende promotor de rodopsina quinasa humana especifica del fotorreceptor, o promotor de beta-actina de pollo cmv quimerico truncado ubicuo unido a al menos un adn que codifica una guanilato ciclasa; virion o particula viral infecciosa que comprende el vector; celula huesped; composicion que comprende el vector.Info
- Publication number
- CL2012002969A1 CL2012002969A1 CL2012002969A CL2012002969A CL2012002969A1 CL 2012002969 A1 CL2012002969 A1 CL 2012002969A1 CL 2012002969 A CL2012002969 A CL 2012002969A CL 2012002969 A CL2012002969 A CL 2012002969A CL 2012002969 A1 CL2012002969 A1 CL 2012002969A1
- Authority
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- Chile
- Prior art keywords
- vector
- raav
- virion
- photoreceptor
- host cell
- Prior art date
Links
- 239000013598 vector Substances 0.000 title abstract 4
- 102000004437 G-Protein-Coupled Receptor Kinase 1 Human genes 0.000 title abstract 2
- 101000834253 Gallus gallus Actin, cytoplasmic 1 Proteins 0.000 title abstract 2
- 101000611338 Homo sapiens Rhodopsin Proteins 0.000 title abstract 2
- RQFCJASXJCIDSX-UUOKFMHZSA-N guanosine 5'-monophosphate Chemical compound C1=2NC(N)=NC(=O)C=2N=CN1[C@@H]1O[C@H](COP(O)(O)=O)[C@@H](O)[C@H]1O RQFCJASXJCIDSX-UUOKFMHZSA-N 0.000 title abstract 2
- 208000015181 infectious disease Diseases 0.000 title abstract 2
- 230000002458 infectious effect Effects 0.000 title abstract 2
- 239000002245 particle Substances 0.000 title abstract 2
- 108091008695 photoreceptors Proteins 0.000 title abstract 2
- 239000013603 viral vector Substances 0.000 title abstract 2
- 210000002845 virion Anatomy 0.000 title abstract 2
- 230000003612 virological effect Effects 0.000 title abstract 2
- 125000004122 cyclic group Chemical group 0.000 title 1
- 108060002020 cyanase Proteins 0.000 abstract 1
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Abstract
Vector viral adenoasociado recombinante (RAAV) que comprende promotor de rodopsina quinasa humana específica del fotorreceptor, o promotor de beta-actina de pollo CMV quimérico truncado ubicuo unido a al menos un ADN que codifica una guanilato ciclasa; virión o partícula viral infecciosa que comprende el vector; célula huésped; composición que comprende el vector.
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US32752110P | 2010-04-23 | 2010-04-23 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CL2012002969A1 true CL2012002969A1 (es) | 2013-05-31 |
Family
ID=44533055
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CL2012002969A CL2012002969A1 (es) | 2010-04-23 | 2012-10-23 | Vector viral adenoasociado recombinante (raav) que comprende promotor de rodopsina quinasa humana especifica del fotorreceptor, o promotor de beta-actina de pollo cmv quimerico truncado ubicuo unido a al menos un adn que codifica una guanilato ciclasa; virion o particula viral infecciosa que comprende el vector; celula huesped; composicion que comprende el vector. |
Country Status (28)
| Country | Link |
|---|---|
| US (3) | US9816108B2 (es) |
| EP (3) | EP4056700A1 (es) |
| JP (4) | JP5897549B2 (es) |
| KR (3) | KR101984145B1 (es) |
| CN (2) | CN102918152B (es) |
| AU (1) | AU2011242527B2 (es) |
| BR (1) | BR112012026730A8 (es) |
| CA (1) | CA2796399C (es) |
| CL (1) | CL2012002969A1 (es) |
| CO (1) | CO6640236A2 (es) |
| CY (1) | CY1123008T1 (es) |
| DK (2) | DK3486320T3 (es) |
| EA (2) | EA202091105A1 (es) |
| ES (2) | ES2911185T3 (es) |
| HR (1) | HRP20190144T1 (es) |
| HU (1) | HUE041571T2 (es) |
| IL (2) | IL222651B (es) |
| LT (1) | LT2561067T (es) |
| MX (1) | MX351865B (es) |
| NZ (1) | NZ602897A (es) |
| PL (2) | PL3486320T3 (es) |
| PT (2) | PT2561067T (es) |
| RS (1) | RS58434B1 (es) |
| SG (4) | SG10201800541SA (es) |
| SI (1) | SI2561067T1 (es) |
| SM (1) | SMT201900097T1 (es) |
| TR (1) | TR201901377T4 (es) |
| WO (1) | WO2011133933A2 (es) |
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| US20120322861A1 (en) | 2007-02-23 | 2012-12-20 | Barry John Byrne | Compositions and Methods for Treating Diseases |
| WO2013075008A1 (en) | 2011-11-16 | 2013-05-23 | University Of Florida Research Foundation Inc. | Aav dual vector systems for gene therapy |
| HUE051632T2 (hu) | 2012-06-19 | 2021-03-29 | Univ Florida | Betegségek kezelésére szolgáló készítmények és eljárások |
| FR3008618A1 (fr) * | 2013-07-19 | 2015-01-23 | Univ Paris Curie | Utilisation de composes pour restaurer la reponse a la lumiere des cellules de la retine |
| MX2016001044A (es) | 2013-07-22 | 2016-09-09 | The Children's Hospital Of Philadelphia | Aav variante y composiciones, metodos y usos para transferencia genica a celulas, organos y tejidos. |
| EP3030665B9 (en) | 2013-08-05 | 2023-10-04 | Academisch Ziekenhuis Leiden H.O.D.N. Leids Universitair Medisch Centrum | Recombinant aav-crumbs homologue composition and methods for treating lca-8 and progressive rp |
| GB201420139D0 (en) | 2014-11-12 | 2014-12-24 | Ucl Business Plc | Factor IX gene therapy |
| PE20180675A1 (es) | 2015-06-23 | 2018-04-19 | Childrens Hospital Philadelphia | Factor ix modificado, y composiciones, metodos y usos para la transferencia de genes a celulas, organos y tejidos |
| KR102526506B1 (ko) * | 2016-07-08 | 2023-05-03 | 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 | Rdh12가 연루된 장애 및 질환의 치료를 위한 방법 및 조성물 |
| AU2018261769B2 (en) * | 2017-05-05 | 2024-03-14 | The Regents Of The University Of California | Compositions and methods for expressing Otoferlin |
| WO2019046341A2 (en) | 2017-08-28 | 2019-03-07 | University Of Florida Research Foundation, Incorporated | METHODS AND COMPOSITIONS FOR TREATING RETINAL DYSTROPHY OF CONES AND STICKS |
| CN117752822A (zh) * | 2017-11-15 | 2024-03-26 | 密歇根大学董事会 | 包括rdh12编码区的病毒载体和治疗视网膜营养不良的方法 |
| JP7287611B2 (ja) * | 2018-02-07 | 2023-06-06 | 学校法人日本医科大学 | 改良型アデノ随伴ウイルスベクター |
| CN119410636A (zh) | 2018-04-27 | 2025-02-11 | 分贝治疗公司 | 肌球蛋白15启动子及其用途 |
| US11660353B2 (en) | 2018-04-27 | 2023-05-30 | Decibel Therapeutics, Inc. | Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems |
| CA3103676A1 (en) * | 2018-07-11 | 2020-01-16 | Cyclerion Therapeutics, Inc. | Use of sgc stimulators for the treatment of mitochonrial disorders |
| WO2020093018A1 (en) | 2018-11-01 | 2020-05-07 | University Of Florida Research Foundation, Incorporated | A codon optimized otoferlin aav dual vector gene therapy |
| US20220133909A1 (en) * | 2019-01-23 | 2022-05-05 | University Of Florida Research Foundation, Incorporated | Highly efficient transduction and lateral spread in the retina by a novel aav virus enhanced by rational design |
| EP3921032A4 (en) | 2019-02-08 | 2022-11-30 | Decibel Therapeutics, Inc. | Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems |
| WO2020163761A1 (en) | 2019-02-08 | 2020-08-13 | Decibel Therapeutics, Inc. | Myosin 15 promoters and uses thereof |
| EP3924470A4 (en) | 2019-02-15 | 2022-12-14 | Exhaura, Ltd. | DUAL LEUCINE ZIPPER KINASE INHIBITORS FOR GENE THERAPY |
| CN115666658A (zh) | 2020-04-01 | 2023-01-31 | 佛罗里达州大学研究基金会 | 具有提高的治疗ush1b的安全性的双aav-myo7a载体 |
| US12297444B2 (en) | 2020-04-14 | 2025-05-13 | University Of Florida Research Foundation, Incorporated | AAV-based gene therapies for treatment of autoimmune diseases |
| US20230233708A1 (en) * | 2020-04-14 | 2023-07-27 | University Of Florida Research Foundation, Incorporated | Enhancing aav-mediated delivery and transduction with polyvinyl alcohol |
| JP2024016295A (ja) * | 2020-11-30 | 2024-02-07 | タカラバイオ株式会社 | 二価の陽イオンを利用した非エンベロープウイルスの製造方法 |
| CN112852976B (zh) * | 2021-03-17 | 2023-10-31 | 湖北省农业科学院畜牧兽医研究所 | 蛋鸡ncs1基因中与后期产蛋性状相关的分子标记及其应用 |
| IL310017A (en) * | 2021-07-14 | 2024-03-01 | Meiragtx Uk Ii Ltd | Retgc gene therapy |
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