BR112012026730A8 - vetor viral adeno-associado recombinante, vírion, pluralidade de partículas virais infecciosas, célula hospedeira de mamífero, kit, uso de composição, métodos para prevenir, tratar ou melhorar doença, disfunção, distúrbio, deficiência ou condição anormal em mamífero de um ou mais sintomas dos mesmos, para prover quantidade terapeuticamente eficaz de peptídeo, polipeptídeo ou proteína de guanilato ciclase a mamífero biologicamente ativo em necessidade da mesma, para aumentar o nível de proteína retgc1 biologicamente ativa em uma ou mais células retinais de mamífero e para tratar ou melhorar um ou mais sintomas de distrofia retinal em mamífero - Google Patents
vetor viral adeno-associado recombinante, vírion, pluralidade de partículas virais infecciosas, célula hospedeira de mamífero, kit, uso de composição, métodos para prevenir, tratar ou melhorar doença, disfunção, distúrbio, deficiência ou condição anormal em mamífero de um ou mais sintomas dos mesmos, para prover quantidade terapeuticamente eficaz de peptídeo, polipeptídeo ou proteína de guanilato ciclase a mamífero biologicamente ativo em necessidade da mesma, para aumentar o nível de proteína retgc1 biologicamente ativa em uma ou mais células retinais de mamífero e para tratar ou melhorar um ou mais sintomas de distrofia retinal em mamífero Download PDFInfo
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Abstract
vetor viral adeno-associado recombinante, pluralidade de partículas virais infecciosas, células hospedeira de mamífero, kit, uso de composição, métodos para prevenir, tratar ou melhorar doença, disfunção, distúrbio, deficiência ou condição anormal em manífero de um ou mais sintomas dos mesmos, para prover quantidade terapeuticamente eficaz de peptídeo, polipeptídeo ou proteína de guanilato ciclase a mamífero biologicamente ativo em necessidade da mesma, para aumentar o nível de proteína retgc1 biologicamente em um ou mais células retinais de mamífero e para tratar ou melhorar um ou mais sintomas de distrofia retinal em mamífero são descritas composições de vetores compreendendo sequências de polinucleotídeo que expressam uma ou mais proteínas de mamíferps guanilato cinase biologicamente ativas. também são descritos métodos para seu uso na prevenção, tratamento e/ou melhora de pelo menos um ou mais sintomas de uma doença, distúrbios, condição anormal, ou disfunção resultando pelo menos em parte, de uma deficiência de guanilato ciclase in vivo. em modalidades particulares, o uso dos vetores virais adeno-associados recombinantes (raav) para tratar ou melhorar os sintomas de amaurose congênita de leber, bem como outras condições causadas por uma ausência ou redução na expressão de um guanilato ciclase 1 específico de retina funcional (retgc1).
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US32752110P | 2010-04-23 | 2010-04-23 | |
PCT/US2011/033669 WO2011133933A2 (en) | 2010-04-23 | 2011-04-22 | Raav-guanylate cyclase compositions and methods for treating leber's congenital amaurosis-1 (lca1) |
Publications (2)
Publication Number | Publication Date |
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BR112012026730A2 BR112012026730A2 (pt) | 2015-09-22 |
BR112012026730A8 true BR112012026730A8 (pt) | 2018-07-03 |
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Application Number | Title | Priority Date | Filing Date |
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BR112012026730A BR112012026730A8 (pt) | 2010-04-23 | 2011-04-22 | vetor viral adeno-associado recombinante, vírion, pluralidade de partículas virais infecciosas, célula hospedeira de mamífero, kit, uso de composição, métodos para prevenir, tratar ou melhorar doença, disfunção, distúrbio, deficiência ou condição anormal em mamífero de um ou mais sintomas dos mesmos, para prover quantidade terapeuticamente eficaz de peptídeo, polipeptídeo ou proteína de guanilato ciclase a mamífero biologicamente ativo em necessidade da mesma, para aumentar o nível de proteína retgc1 biologicamente ativa em uma ou mais células retinais de mamífero e para tratar ou melhorar um ou mais sintomas de distrofia retinal em mamífero |
Country Status (27)
Country | Link |
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US (3) | US9816108B2 (pt) |
EP (3) | EP3486320B1 (pt) |
JP (4) | JP5897549B2 (pt) |
KR (3) | KR101984145B1 (pt) |
CN (2) | CN105821079B (pt) |
AU (1) | AU2011242527B2 (pt) |
BR (1) | BR112012026730A8 (pt) |
CA (1) | CA2796399C (pt) |
CL (1) | CL2012002969A1 (pt) |
CO (1) | CO6640236A2 (pt) |
CY (1) | CY1123008T1 (pt) |
DK (2) | DK2561067T3 (pt) |
EA (2) | EA035893B1 (pt) |
ES (2) | ES2711256T3 (pt) |
HR (1) | HRP20190144T1 (pt) |
HU (1) | HUE041571T2 (pt) |
IL (2) | IL222651B (pt) |
LT (1) | LT2561067T (pt) |
MX (1) | MX351865B (pt) |
NZ (1) | NZ602897A (pt) |
PL (2) | PL2561067T3 (pt) |
PT (2) | PT3486320T (pt) |
RS (1) | RS58434B1 (pt) |
SG (4) | SG184876A1 (pt) |
SI (1) | SI2561067T1 (pt) |
TR (1) | TR201901377T4 (pt) |
WO (1) | WO2011133933A2 (pt) |
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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 |
EP3818991A3 (en) | 2012-06-19 | 2021-07-14 | University of Florida Research Foundation, Inc. | Compositions and methods for treating diseases |
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 |
DK3024498T3 (da) | 2013-07-22 | 2020-03-02 | Childrens Hospital Philadelphia | Aav-variant og sammensætninger, fremgangsmåder og anvendelser til genoverførsel til celler, organer og væv |
CA2919995A1 (en) | 2013-08-05 | 2015-02-12 | 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 |
US10799566B2 (en) | 2015-06-23 | 2020-10-13 | The Children's Hospital Of Philadelphia | Modified factor IX, and compositions, methods and uses for gene transfer to cells, organs, and tissues |
KR102606810B1 (ko) * | 2017-05-05 | 2023-11-27 | 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 | 오토펄린을 발현시키기 위한 조성물 및 방법 |
EP3675913A4 (en) | 2017-08-28 | 2021-06-02 | University of Florida Research Foundation, Incorporated | METHODS AND COMPOSITIONS FOR TREATMENT OF CONE-ROD DYSTROPHY |
WO2019099696A1 (en) * | 2017-11-15 | 2019-05-23 | The Regents Of The University Of Michigan | Viral vectors comprising rdh12 coding regions and methods of treating retinal dystrophies |
EP3751000A4 (en) | 2018-02-07 | 2021-04-21 | Nippon Medical School Foundation | IMPROVED ADENO-ASSOCIATED VIRUS VECTOR |
US11660353B2 (en) | 2018-04-27 | 2023-05-30 | Decibel Therapeutics, Inc. | Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems |
US20220213494A1 (en) | 2019-02-15 | 2022-07-07 | Thomas W. Chalberg | Dual leucine zipper kinase inhibitors for gene therapy |
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 |
AU2022310166A1 (en) * | 2021-07-14 | 2024-02-29 | Meiragtx Uk Ii Limited | Retgc gene therapy |
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