CY1123158T1 - Θεραπειες γονιδιακης ενισχυσης για την κληρονομικη εκφυλιση του αμφιβληστροειδους που προκαλειται απο μεταλλαξεις στο γονιδιο prpf31 - Google Patents
Θεραπειες γονιδιακης ενισχυσης για την κληρονομικη εκφυλιση του αμφιβληστροειδους που προκαλειται απο μεταλλαξεις στο γονιδιο prpf31Info
- Publication number
- CY1123158T1 CY1123158T1 CY20201100718T CY201100718T CY1123158T1 CY 1123158 T1 CY1123158 T1 CY 1123158T1 CY 20201100718 T CY20201100718 T CY 20201100718T CY 201100718 T CY201100718 T CY 201100718T CY 1123158 T1 CY1123158 T1 CY 1123158T1
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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
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- 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
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- 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
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- 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
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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- 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
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
- C12N15/90—Stable introduction of foreign DNA into chromosome
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- 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
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- 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
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- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]
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- 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
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- 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
- C12N2800/00—Nucleic acids vectors
- C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
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- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Biotechnology (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Medicinal Chemistry (AREA)
- Epidemiology (AREA)
- Physics & Mathematics (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Ophthalmology & Optometry (AREA)
- Mycology (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Peptides Or Proteins (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicinal Preparation (AREA)
Abstract
Η παρούσα εφεύρεση αφορά μεθόδους και συνθέσεις για τη γονιδιακή θεραπεία της μελαγχρωστικής αμφιβληστροειδοπάθειας που σχετίζεται με μεταλλάξεις στον παράγοντα επεξεργασίας προ-mRNA 31 (PRPF31).
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201562129638P | 2015-03-06 | 2015-03-06 | |
US201562147307P | 2015-04-14 | 2015-04-14 | |
PCT/US2016/021226 WO2016144892A1 (en) | 2015-03-06 | 2016-03-07 | Gene augmentation therapies for inherited retinal degeneration caused by mutations in the prpf31 gene |
Publications (1)
Publication Number | Publication Date |
---|---|
CY1123158T1 true CY1123158T1 (el) | 2021-10-29 |
Family
ID=56879585
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CY20201100718T CY1123158T1 (el) | 2015-03-06 | 2020-08-04 | Θεραπειες γονιδιακης ενισχυσης για την κληρονομικη εκφυλιση του αμφιβληστροειδους που προκαλειται απο μεταλλαξεις στο γονιδιο prpf31 |
Country Status (15)
Country | Link |
---|---|
US (2) | US20180043034A1 (el) |
EP (2) | EP3265568B1 (el) |
JP (3) | JP6812368B2 (el) |
CY (1) | CY1123158T1 (el) |
DK (1) | DK3265568T3 (el) |
ES (1) | ES2806054T3 (el) |
HK (1) | HK1247637A1 (el) |
HR (1) | HRP20201225T1 (el) |
HU (1) | HUE051491T2 (el) |
LT (1) | LT3265568T (el) |
PL (1) | PL3265568T3 (el) |
PT (1) | PT3265568T (el) |
RS (1) | RS60668B1 (el) |
SI (1) | SI3265568T1 (el) |
WO (1) | WO2016144892A1 (el) |
Families Citing this family (17)
Publication number | Priority date | Publication date | Assignee | Title |
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US8663624B2 (en) | 2010-10-06 | 2014-03-04 | The Regents Of The University Of California | Adeno-associated virus virions with variant capsid and methods of use thereof |
EP2699270B1 (en) | 2011-04-22 | 2017-06-21 | The Regents of The University of California | Adeno-associated virus virions with variant capsid and methods of use thereof |
US11136557B2 (en) | 2013-05-31 | 2021-10-05 | The Regents Of The University Of California | Adeno-associated virus variants and methods of use thereof |
AU2015231439B2 (en) | 2014-03-17 | 2019-11-14 | Adverum Biotechnologies, Inc. | Compositions and methods for enhanced gene expression in cone cells |
KR20170137730A (ko) | 2015-03-02 | 2017-12-13 | 애드베룸 바이오테크놀로지스, 인코포레이티드 | 망막 추상체에 폴리뉴클레오타이드의 유리체 내 전달을 위한 조성물 및 방법 |
PT3265568T (pt) * | 2015-03-06 | 2020-08-20 | Massachusetts Eye & Ear Infirmary | Terapias de aumento de genes para a degeneração hereditária da retina causada por mutações no gene prpf31 |
WO2016154344A1 (en) | 2015-03-24 | 2016-09-29 | The Regents Of The University Of California | Adeno-associated virus variants and methods of use thereof |
KR102508820B1 (ko) | 2016-07-29 | 2023-03-13 | 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 | 변이체 캡시드를 갖는 아데노-관련된 바이러스 비리온 및 이의 사용 방법 |
CN110225975A (zh) | 2016-08-23 | 2019-09-10 | 阿库斯股份有限公司 | 用于治疗人受试者中非年龄相关的听力损害的组合物和方法 |
CA3040179A1 (en) | 2016-10-19 | 2018-04-26 | Adverum Biotechnologies, Inc. | Modified aav capsids and uses thereof |
CA3061968A1 (en) * | 2017-05-10 | 2018-11-15 | Massachusetts Eye And Ear Infirmary | Methods and compositions for modifying assembly-activating protein (aap)-dependence of viruses |
CA3059995A1 (en) | 2017-08-28 | 2019-03-07 | The Regents Of The University Of California | Adeno-associated virus capsid variants and methods of use thereof |
WO2019232517A1 (en) * | 2018-06-01 | 2019-12-05 | University Of Florida Research Foundation, Incorporated | Compositions and methods for treatment of dominant retinitis pigmentosa |
WO2020102740A2 (en) * | 2018-11-16 | 2020-05-22 | Spark Therapeutics, Inc. | Compositions and methods for increasing or enhancing transduction of gene therapy vectors and for removing or reducing immunoglobulins |
JP2023515072A (ja) | 2020-02-21 | 2023-04-12 | アコーオス インコーポレイテッド | ヒト対象において非加齢関連聴力障害を処置するための組成物および方法 |
WO2024086352A2 (en) * | 2022-10-21 | 2024-04-25 | Biogen Ma Inc. | Compositions and methods for treating retinitis pigmentosa |
WO2024123461A1 (en) * | 2022-12-05 | 2024-06-13 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Pigment epithelium-derived factor peptides and use for treating retinal degeneration |
Family Cites Families (23)
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DE3023787A1 (de) | 1980-06-25 | 1982-01-21 | Studiengesellschaft Kohle mbH, 4330 Mülheim | Verfahren zur erhoehung der inkorporation und der expression von genetischem material in die kerne von intakten zellen mit hilfe von liposomen |
IL79289A (en) | 1985-07-05 | 1992-01-15 | Whitehead Biomedical Inst | Introduction and expression of foreign genetic material into keratinocytes using a recombinant retrovirus |
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 |
WO1989002468A1 (en) | 1987-09-11 | 1989-03-23 | Whitehead Institute For Biomedical Research | Transduced fibroblasts and uses therefor |
ATE110108T1 (de) | 1987-12-11 | 1994-09-15 | Whitehead Biomedical Inst | Genetische modifizierung von endothelialen zellen. |
JP2917998B2 (ja) | 1988-02-05 | 1999-07-12 | ホワイトヘッド・インスティチュート・フォー・バイオメディカル・リサーチ | 修飾された肝細胞およびその用途 |
ATE157012T1 (de) | 1989-11-03 | 1997-09-15 | Univ Vanderbilt | Verfahren zur in vivo-verabreichung von funktionsfähigen fremden genen |
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ES2150832B1 (es) | 1996-06-12 | 2001-06-16 | Fichtel & Sachs Ag | Dispositivo de maniobra para la maniobra, en particular maniobra neumatica, de un embrague de friccion. |
US6569662B1 (en) * | 2000-01-21 | 2003-05-27 | Hyseq, Inc. | Nucleic acids and polypeptides |
EP1381276A4 (en) * | 2001-04-13 | 2005-02-02 | Univ Pennsylvania | METHOD FOR TREATMENT OR DEVELOPMENT SLUDGE DEGRADATION |
US20040208847A1 (en) * | 2003-03-28 | 2004-10-21 | Fabienne Rolling | Method and vectors for selectively transducing retinal pigment epithelium cells |
CA2642342A1 (en) * | 2006-02-14 | 2007-08-23 | Dana-Farber Cancer Institute, Inc. | Compositions, kits, and methods for identification, assessment, prevention, and therapy of cancer |
US8257969B2 (en) * | 2007-04-12 | 2012-09-04 | The Provost Fellows And Scholars Of The College Of The Holy And Undivided Trinity Of Queen Elizabeth | Genetic suppression and replacement |
US20090011040A1 (en) * | 2007-05-02 | 2009-01-08 | Naash Muna I | Use of compacted nucleic acid nanoparticles in non-viral treatments of ocular diseases |
WO2009134681A2 (en) * | 2008-04-30 | 2009-11-05 | The Trustees Of The University Of Pennsylvania | Aav7 viral vectors for targeted delivery of rpe cells |
EP2320937A4 (en) * | 2008-06-30 | 2013-01-16 | Univ Johns Hopkins | COMPOSITIONS AND METHODS FOR THE TREATMENT OF OCULAR OXIDATION STRESS AND PIGMENT RETINITY |
GB201103062D0 (en) * | 2011-02-22 | 2011-04-06 | Isis Innovation | Method |
TWI702955B (zh) * | 2012-05-15 | 2020-09-01 | 澳大利亞商艾佛蘭屈澳洲私營有限公司 | 使用腺相關病毒(aav)sflt-1治療老年性黃斑部退化(amd) |
CN105120901A (zh) * | 2012-07-11 | 2015-12-02 | 宾夕法尼亚大学托管会 | Rpgr x染色体连锁视网膜退化的aav介导的基因治疗 |
PT3459965T (pt) | 2013-10-11 | 2021-02-25 | Massachusetts Eye & Ear Infirmary | Métodos de previsão de sequências de vírus ancestrais e suas utilizações |
WO2015189429A1 (en) * | 2014-06-13 | 2015-12-17 | Universidade De Santiago De Compostela | Nanoparticulate systems for use in gene transfer or gene delivery |
PT3265568T (pt) * | 2015-03-06 | 2020-08-20 | Massachusetts Eye & Ear Infirmary | Terapias de aumento de genes para a degeneração hereditária da retina causada por mutações no gene prpf31 |
-
2016
- 2016-03-07 PT PT167623073T patent/PT3265568T/pt unknown
- 2016-03-07 EP EP16762307.3A patent/EP3265568B1/en active Active
- 2016-03-07 WO PCT/US2016/021226 patent/WO2016144892A1/en active Application Filing
- 2016-03-07 DK DK16762307.3T patent/DK3265568T3/da active
- 2016-03-07 EP EP20171646.1A patent/EP3748007B1/en active Active
- 2016-03-07 LT LTEP16762307.3T patent/LT3265568T/lt unknown
- 2016-03-07 ES ES16762307T patent/ES2806054T3/es active Active
- 2016-03-07 RS RS20200945A patent/RS60668B1/sr unknown
- 2016-03-07 PL PL16762307T patent/PL3265568T3/pl unknown
- 2016-03-07 US US15/555,915 patent/US20180043034A1/en not_active Abandoned
- 2016-03-07 JP JP2017564778A patent/JP6812368B2/ja active Active
- 2016-03-07 SI SI201630873T patent/SI3265568T1/sl unknown
- 2016-03-07 HU HUE16762307A patent/HUE051491T2/hu unknown
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2018
- 2018-05-25 HK HK18106900.6A patent/HK1247637A1/zh unknown
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2020
- 2020-08-04 CY CY20201100718T patent/CY1123158T1/el unknown
- 2020-08-04 HR HRP20201225TT patent/HRP20201225T1/hr unknown
- 2020-08-13 US US16/992,689 patent/US20210069347A1/en active Pending
- 2020-12-16 JP JP2020208068A patent/JP7239550B2/ja active Active
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2023
- 2023-03-02 JP JP2023031931A patent/JP2023071867A/ja active Pending
Also Published As
Publication number | Publication date |
---|---|
EP3265568A1 (en) | 2018-01-10 |
HUE051491T2 (hu) | 2021-03-01 |
LT3265568T (lt) | 2020-08-25 |
RS60668B1 (sr) | 2020-09-30 |
US20180043034A1 (en) | 2018-02-15 |
ES2806054T3 (es) | 2021-02-16 |
EP3748007A1 (en) | 2020-12-09 |
JP2018511652A (ja) | 2018-04-26 |
EP3748007B1 (en) | 2024-06-26 |
EP3265568B1 (en) | 2020-05-06 |
HK1247637A1 (zh) | 2018-09-28 |
DK3265568T3 (da) | 2020-08-10 |
SI3265568T1 (sl) | 2020-10-30 |
US20210069347A1 (en) | 2021-03-11 |
EP3265568A4 (en) | 2018-08-08 |
JP6812368B2 (ja) | 2021-01-13 |
JP2023071867A (ja) | 2023-05-23 |
PL3265568T3 (pl) | 2020-11-30 |
JP7239550B2 (ja) | 2023-03-14 |
JP2021059561A (ja) | 2021-04-15 |
PT3265568T (pt) | 2020-08-20 |
HRP20201225T1 (hr) | 2020-11-13 |
WO2016144892A1 (en) | 2016-09-15 |
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