CY1123793T1 - Γονιδιακη θεραπεια rpgr για μελαγχρωστικη αμφιβληστροειδοπαθεια - Google Patents
Γονιδιακη θεραπεια rpgr για μελαγχρωστικη αμφιβληστροειδοπαθειαInfo
- Publication number
- CY1123793T1 CY1123793T1 CY20211100002T CY211100002T CY1123793T1 CY 1123793 T1 CY1123793 T1 CY 1123793T1 CY 20211100002 T CY20211100002 T CY 20211100002T CY 211100002 T CY211100002 T CY 211100002T CY 1123793 T1 CY1123793 T1 CY 1123793T1
- Authority
- CY
- Cyprus
- Prior art keywords
- rpgr
- melangrosis
- amphiblistroidopathy
- gene therapy
- rpgr gene
- Prior art date
Links
- 101001104102 Homo sapiens X-linked retinitis pigmentosa GTPase regulator Proteins 0.000 title abstract 2
- 102100040092 X-linked retinitis pigmentosa GTPase regulator Human genes 0.000 title 1
- 238000001415 gene therapy Methods 0.000 title 1
- 208000017442 Retinal disease Diseases 0.000 abstract 2
- 206010038923 Retinopathy Diseases 0.000 abstract 2
- 102000013446 GTP Phosphohydrolases Human genes 0.000 abstract 1
- 108091006109 GTPases Proteins 0.000 abstract 1
- 239000002299 complementary DNA Substances 0.000 abstract 1
- 102000034356 gene-regulatory proteins Human genes 0.000 abstract 1
- 108091006104 gene-regulatory proteins Proteins 0.000 abstract 1
- 102000050188 human RPGR Human genes 0.000 abstract 1
- 230000004777 loss-of-function mutation Effects 0.000 abstract 1
- 102000039446 nucleic acids Human genes 0.000 abstract 1
- 108020004707 nucleic acids Proteins 0.000 abstract 1
- 150000007523 nucleic acids Chemical class 0.000 abstract 1
- 108090000623 proteins and genes Proteins 0.000 abstract 1
- 239000013603 viral vector Substances 0.000 abstract 1
Classifications
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0048—Eye, e.g. artificial tears
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/02—Ophthalmic agents
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
- C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
- C07K14/4702—Regulators; Modulating activity
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Organic Chemistry (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Genetics & Genomics (AREA)
- Molecular Biology (AREA)
- Epidemiology (AREA)
- Engineering & Computer Science (AREA)
- Gastroenterology & Hepatology (AREA)
- Ophthalmology & Optometry (AREA)
- Biotechnology (AREA)
- Toxicology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Zoology (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicinal Preparation (AREA)
- Peptides Or Proteins (AREA)
- Saccharide Compounds (AREA)
- Compounds Of Unknown Constitution (AREA)
Abstract
Μέθοδοι για τη θεραπευτική αγωγή για υποκείμενο που είναι άνθρωπος που έχει φυλοσύνδετη (X-linked) μελαγχρωστική αμφιβληστροειδοπάθεια (XLRP) ή άλλη κλινικά καθορισμένη οφθαλμολογική πάθηση λόγω μετάλλαξης που αφορά απώλεια της λειτουργίας στο γονίδιο που κωδικοποιεί την πρωτεΐνη ρυθμιστή της GTΡάσης της μελαγχρωστικής αμφιβληστροειδοπάθειας (RPGR), με τη μέθοδο να περιλαμβάνει χορήγηση στο υποκείμενο ενός νουκλεϊκού οξέος που περιλαμβάνει έναν αδενο-σχετιζόμενο ιικό φορέα που περιλαμβάνει cDNA συντετμημένου ανθρώπινου RPGR.
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201462028638P | 2014-07-24 | 2014-07-24 | |
| PCT/US2015/040866 WO2016014353A1 (en) | 2014-07-24 | 2015-07-17 | Rpgr gene therapy for retinitis pigmentosa |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CY1123793T1 true CY1123793T1 (el) | 2022-05-27 |
Family
ID=55163577
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CY20211100002T CY1123793T1 (el) | 2014-07-24 | 2021-01-05 | Γονιδιακη θεραπεια rpgr για μελαγχρωστικη αμφιβληστροειδοπαθεια |
Country Status (18)
| Country | Link |
|---|---|
| US (2) | US10314924B2 (el) |
| EP (2) | EP3191139B1 (el) |
| JP (3) | JP6654760B2 (el) |
| CN (1) | CN107206105A (el) |
| CA (1) | CA2991750C (el) |
| CY (1) | CY1123793T1 (el) |
| DK (2) | DK3821912T3 (el) |
| ES (2) | ES2834402T3 (el) |
| FI (1) | FI3821912T3 (el) |
| HR (2) | HRP20241154T1 (el) |
| HU (2) | HUE068183T2 (el) |
| LT (2) | LT3191139T (el) |
| PL (2) | PL3191139T3 (el) |
| PT (2) | PT3191139T (el) |
| RS (2) | RS65909B1 (el) |
| SI (2) | SI3821912T1 (el) |
| SM (2) | SMT202400309T1 (el) |
| WO (1) | WO2016014353A1 (el) |
Families Citing this family (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| SI3821912T1 (sl) | 2014-07-24 | 2024-10-30 | Massachusetts Eye & Ear Infirmary | Genska terapija RPGR za pigmentozni retinitis |
| US20190054117A1 (en) * | 2014-12-19 | 2019-02-21 | Novartis Ag | Dimerization switches and uses thereof |
| US10646588B2 (en) | 2015-03-11 | 2020-05-12 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | RP2 and RPGR vectors for treating X-linked retinitis pigmentosa |
| GB201516066D0 (en) | 2015-09-10 | 2015-10-28 | Young & Co Llp D | Treatment of retinitis pigmentosa |
| GB201704192D0 (en) | 2017-03-16 | 2017-05-03 | Nightstarx Ltd | Treatment of Retinitis Pigmentosa |
| EP3697448A1 (en) * | 2017-10-20 | 2020-08-26 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Methods of expressing a polynucleotide of interest in the cone photoreceptors of a subject comprising the subretinal delivery of a therapeutically effective amount of a recombinant aav9-derived vector |
| JP2021502978A (ja) * | 2017-11-15 | 2021-02-04 | ザ リージェンツ オブ ザ ユニバーシティ オブ ミシガン | 網膜ジストロフィーを治療するｒｄｈ１２コード領域を含むウイルスベクターおよび方法 |
| MX2022011177A (es) | 2020-03-11 | 2022-12-13 | Massachusetts Eye & Ear Infirmary | Terapia genica para la degeneracion de la retina asociada a nmnat1. |
Family Cites Families (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4980286A (en) | 1985-07-05 | 1990-12-25 | Whitehead Institute For Biomedical Research | In vivo introduction and expression of foreign genetic material in epithelial cells |
| JP2703893B2 (ja) | 1985-07-05 | 1998-01-26 | ホワイトヘッド・インスティテュ−ト・フォ−・バイオメディカル・リサ−チ | 外来遺伝子物質を発現する上皮細胞 |
| JPS6259797A (ja) * | 1985-09-10 | 1987-03-16 | 日本電信電話株式会社 | 地中管体の推進方法 |
| WO1989002468A1 (en) | 1987-09-11 | 1989-03-23 | Whitehead Institute For Biomedical Research | Transduced fibroblasts and uses therefor |
| JP2914692B2 (ja) | 1987-12-11 | 1999-07-05 | ホワイトヘツド・インスチチユート・フオー・バイオメデイカル・リサーチ | 内皮細胞の遺伝子修飾 |
| EP0400047B1 (en) | 1988-02-05 | 1997-04-23 | Whitehead Institute For Biomedical Research | Modified hepatocytes and uses therefor |
| JP3249516B2 (ja) | 1990-10-31 | 2002-01-21 | ソマティクス セラピー コーポレイション | 遺伝子治療のためのレトロウイルスのベクター |
| US5767079A (en) * | 1992-07-08 | 1998-06-16 | Celtrix Pharmaceuticals, Inc. | Method of treating ophthalmic disorders using TGF -β |
| AU2044901A (en) | 1999-11-24 | 2001-06-04 | Cornell Research Foundation Inc. | Inherited retinal diseases at the canine rp3 locus: linkage, marker- and mutation-based tests |
| GB0008801D0 (en) | 2000-04-10 | 2000-05-31 | Medical Res Council | Sequences |
| CN102925445A (zh) * | 2011-08-08 | 2013-02-13 | 刘军 | 一种获得rpgr 基因新的转录剪切形式的方法 |
| EP2872183B1 (en) * | 2012-07-11 | 2018-09-26 | The Trustees Of The University Of Pennsylvania | Aav-mediated gene therapy for rpgr x-linked retinal degeneration |
| GB201412011D0 (en) * | 2014-07-04 | 2014-08-20 | Ucl Business Plc | Treatments |
| SI3821912T1 (sl) * | 2014-07-24 | 2024-10-30 | Massachusetts Eye & Ear Infirmary | Genska terapija RPGR za pigmentozni retinitis |
-
2015
- 2015-07-17 SI SI201532022T patent/SI3821912T1/sl unknown
- 2015-07-17 ES ES15825383T patent/ES2834402T3/es active Active
- 2015-07-17 SI SI201531413T patent/SI3191139T1/sl unknown
- 2015-07-17 DK DK20199208.8T patent/DK3821912T3/da active
- 2015-07-17 US US15/328,617 patent/US10314924B2/en active Active
- 2015-07-17 JP JP2017525514A patent/JP6654760B2/ja active Active
- 2015-07-17 PL PL15825383T patent/PL3191139T3/pl unknown
- 2015-07-17 FI FIEP20199208.8T patent/FI3821912T3/fi active
- 2015-07-17 SM SM20240309T patent/SMT202400309T1/it unknown
- 2015-07-17 WO PCT/US2015/040866 patent/WO2016014353A1/en active Application Filing
- 2015-07-17 HU HUE20199208A patent/HUE068183T2/hu unknown
- 2015-07-17 LT LTEP15825383.1T patent/LT3191139T/lt unknown
- 2015-07-17 HU HUE15825383A patent/HUE052781T2/hu unknown
- 2015-07-17 SM SM20200715T patent/SMT202000715T1/it unknown
- 2015-07-17 CA CA2991750A patent/CA2991750C/en active Active
- 2015-07-17 EP EP15825383.1A patent/EP3191139B1/en active Active
- 2015-07-17 RS RS20240901A patent/RS65909B1/sr unknown
- 2015-07-17 RS RS20201416A patent/RS61307B1/sr unknown
- 2015-07-17 PT PT158253831T patent/PT3191139T/pt unknown
- 2015-07-17 EP EP20199208.8A patent/EP3821912B1/en active Active
- 2015-07-17 HR HRP20241154TT patent/HRP20241154T1/hr unknown
- 2015-07-17 PL PL20199208.8T patent/PL3821912T3/pl unknown
- 2015-07-17 DK DK15825383.1T patent/DK3191139T3/da active
- 2015-07-17 ES ES20199208T patent/ES2987090T3/es active Active
- 2015-07-17 CN CN201580051512.3A patent/CN107206105A/zh active Pending
- 2015-07-17 PT PT201992088T patent/PT3821912T/pt unknown
- 2015-07-17 LT LTEP20199208.8T patent/LT3821912T/lt unknown
-
2019
- 2019-06-10 US US16/436,521 patent/US11045558B2/en active Active
- 2019-12-26 JP JP2019235539A patent/JP6966532B2/ja active Active
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2020
- 2020-12-17 HR HRP20202023TT patent/HRP20202023T1/hr unknown
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2021
- 2021-01-05 CY CY20211100002T patent/CY1123793T1/el unknown
- 2021-10-21 JP JP2021172128A patent/JP7198329B2/ja active Active
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