MX2021010149A - Composiciones y metodos para tratar la distrofia cristalina de bietti. - Google Patents

Composiciones y metodos para tratar la distrofia cristalina de bietti.

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Publication number
MX2021010149A
MX2021010149A MX2021010149A MX2021010149A MX2021010149A MX 2021010149 A MX2021010149 A MX 2021010149A MX 2021010149 A MX2021010149 A MX 2021010149A MX 2021010149 A MX2021010149 A MX 2021010149A MX 2021010149 A MX2021010149 A MX 2021010149A
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Mexico
Prior art keywords
treat
compositions
methods
crystalline dystrophy
bietti crystalline
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MX2021010149A
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English (en)
Inventor
Botond Roska
Josephine Juettner
Jacek Krol
Christie L Bell
Terri Mcgee
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Novartis Ag
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Publication date
Application filed by Novartis Ag filed Critical Novartis Ag
Publication of MX2021010149A publication Critical patent/MX2021010149A/es

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    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

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Abstract

En la presente se proveen vectores virales para insertar un gen del CYP4V2 heterólogo en la retina, por ej., las células del EPR de la retina para tratar sujetos con distrofia cristalina de Bietti.
MX2021010149A 2019-02-25 2020-02-24 Composiciones y metodos para tratar la distrofia cristalina de bietti. MX2021010149A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962810250P 2019-02-25 2019-02-25
PCT/IB2020/051557 WO2020174368A1 (en) 2019-02-25 2020-02-24 Compositions and methods to treat bietti crystalline dystrophy

Publications (1)

Publication Number Publication Date
MX2021010149A true MX2021010149A (es) 2021-09-14

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Application Number Title Priority Date Filing Date
MX2021010149A MX2021010149A (es) 2019-02-25 2020-02-24 Composiciones y metodos para tratar la distrofia cristalina de bietti.

Country Status (14)

Country Link
US (1) US20220154210A1 (es)
EP (1) EP3931334A1 (es)
JP (1) JP2022521025A (es)
KR (1) KR20210132684A (es)
CN (1) CN113677801A (es)
AU (1) AU2020229085A1 (es)
BR (1) BR112021016501A2 (es)
CA (1) CA3130731A1 (es)
CR (1) CR20210444A (es)
IL (1) IL285447A (es)
MX (1) MX2021010149A (es)
SG (1) SG11202108044YA (es)
TW (1) TW202045729A (es)
WO (1) WO2020174368A1 (es)

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ES2881782T3 (es) 2015-10-14 2021-11-30 Friedrich Miescher Institute For Biomedical Res Promotor para la expresión específica de genes en células endoteliales retinianas.
ES2761328T3 (es) 2015-12-03 2020-05-19 Friedrich Miescher Institute For Biomedical Res SynP161, un promotor para la expresión específica de genes en fotorreceptores de bastón
US10995344B2 (en) 2015-12-03 2021-05-04 Friedrich Miescher Institute For Biomedical Research SYNP159, a promoter for the specific expression of genes in rod photoreceptors
CN108472390B (zh) 2015-12-03 2022-04-15 弗里德里克·米谢尔生物医学研究所 SynP162,用于基因在视杆光感受器中特异性表达的启动子
KR20190077471A (ko) 2016-11-02 2019-07-03 프리드리히 미셔 인스티튜트 포 바이오메디칼 리서치 방향 선택적인 망막 신경절 세포에서의 유전자의 특이적 발현을 위한 프로모터, synp198
US11371060B2 (en) 2017-02-08 2022-06-28 Friedrich Miescher Institute For Biomedical Research SYNP88, a promoter for the specific expression of genes in retinal ganglion cells
CR20200210A (es) 2017-11-15 2020-09-23 Friedrich Miescher Institute For Biomedical Res Promotor especìfico de las cèlulas del epitelio pigmentario retinal en primates
JP2021503934A (ja) 2017-11-30 2021-02-15 フリードリッヒ ミーシェー インスティトゥート フォー バイオメディカル リサーチ 網膜色素上皮中の遺伝子の特異的発現のためのプロモーターＳｙｎＰＩＩＩ
CN117916365A (zh) * 2021-07-15 2024-04-19 上海天泽云泰生物医药有限公司 用于治疗结晶样视网膜变性的重组腺相关病毒载体
CN118028318A (zh) * 2021-12-22 2024-05-14 苏州诺洁贝生物技术有限公司 优化的cyp4v2基因及其用途

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