JP2019524090A5 - - Google Patents

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Publication number
JP2019524090A5
JP2019524090A5 JP2019500531A JP2019500531A JP2019524090A5 JP 2019524090 A5 JP2019524090 A5 JP 2019524090A5 JP 2019500531 A JP2019500531 A JP 2019500531A JP 2019500531 A JP2019500531 A JP 2019500531A JP 2019524090 A5 JP2019524090 A5 JP 2019524090A5
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JP
Japan
Prior art keywords
rdh12
composition
functional
nucleic acid
acid sequence
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JP2019500531A
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English (en)
Japanese (ja)
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JP2019524090A (ja
JP7101656B2 (ja
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Priority claimed from PCT/US2017/041122 external-priority patent/WO2018009814A1/en
Publication of JP2019524090A publication Critical patent/JP2019524090A/ja
Publication of JP2019524090A5 publication Critical patent/JP2019524090A5/ja
Priority to JP2022076693A priority Critical patent/JP7572990B2/ja
Application granted granted Critical
Publication of JP7101656B2 publication Critical patent/JP7101656B2/ja
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JP2019500531A 2016-07-08 2017-07-07 Rdh12が関与する疾患及び疾病を治療する方法及び組成物 Active JP7101656B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2022076693A JP7572990B2 (ja) 2016-07-08 2022-05-06 Rdh12が関与する疾患及び疾病を治療する方法及び組成物

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662359777P 2016-07-08 2016-07-08
US62/359,777 2016-07-08
PCT/US2017/041122 WO2018009814A1 (en) 2016-07-08 2017-07-07 Methods and compositions for treatment of disorders and diseases involving rdh12

Related Child Applications (1)

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JP2022076693A Division JP7572990B2 (ja) 2016-07-08 2022-05-06 Rdh12が関与する疾患及び疾病を治療する方法及び組成物

Publications (3)

Publication Number Publication Date
JP2019524090A JP2019524090A (ja) 2019-09-05
JP2019524090A5 true JP2019524090A5 (enExample) 2020-07-16
JP7101656B2 JP7101656B2 (ja) 2022-07-15

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ID=60901399

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JP2019500531A Active JP7101656B2 (ja) 2016-07-08 2017-07-07 Rdh12が関与する疾患及び疾病を治療する方法及び組成物
JP2022076693A Active JP7572990B2 (ja) 2016-07-08 2022-05-06 Rdh12が関与する疾患及び疾病を治療する方法及び組成物

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JP2022076693A Active JP7572990B2 (ja) 2016-07-08 2022-05-06 Rdh12が関与する疾患及び疾病を治療する方法及び組成物

Country Status (12)

Country Link
US (4) US11197936B2 (enExample)
EP (1) EP3481958B1 (enExample)
JP (2) JP7101656B2 (enExample)
KR (2) KR102799528B1 (enExample)
CN (1) CN110191954B (enExample)
AU (2) AU2017292010B2 (enExample)
BR (1) BR112019000171A2 (enExample)
CA (1) CA3029646A1 (enExample)
ES (1) ES3069917T3 (enExample)
IL (3) IL264070B2 (enExample)
RU (1) RU2764920C2 (enExample)
WO (1) WO2018009814A1 (enExample)

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3359663A4 (en) * 2015-09-24 2019-08-14 The Trustees of The University of Pennsylvania COMPOSITION AND METHOD FOR THE TREATMENT OF A COMPLEMENT-MEDIATED DISEASE
US11197936B2 (en) * 2016-07-08 2021-12-14 The Trustees Of The University Of Pennsylvania Methods and compositions for treatment of disorders and diseases involving RDH12
CA3099004C (en) 2017-05-04 2024-11-12 Lehigh University ADDITIVE MANUFACTURING SYSTEM WITH ADJUSTABLE MATERIAL PROPERTIES
SMT202400321T1 (it) * 2017-11-15 2024-11-15 Univ Michigan Regents Vettori virali comprendenti regioni codificanti rdh12 e metodi di trattamento di distrofie retiniche
MX2023008826A (es) 2021-02-01 2023-09-15 Regenxbio Inc Terapia génica para lipofuscinosis neuronal ceroidea.
EP4457347A4 (en) * 2021-12-31 2026-07-01 T C Uskudar Univ CRISPR-PE SYSTEM FOR RETINOL DEHYDROGENASE 12 (RDH12) GENE MUTATIONS FOR THE TREATMENT OF RETINITIS PIGMENTARIAN (RP)
TR2021022284A2 (tr) * 2021-12-31 2022-01-21 T C Ueskuedar Ueniversitesi Retinitis pigmentosa (rp) hastalığının tedavisinde kullanılmak üzere retinol dehidrojenaz 12 (rdh12) gen mutasyonlarına yönelik crıspr-pe sistemi.
CN115725659A (zh) * 2022-04-01 2023-03-03 首都医科大学附属北京同仁医院 一种应用视网膜原位基因编辑制作非人灵长类动物先天性黑矇模型的方法
JP7168814B1 (ja) 2022-07-01 2022-11-09 東洋インキScホールディングス株式会社 積層体の分離回収方法
CN118453915A (zh) * 2024-04-29 2024-08-09 中国医学科学院北京协和医院 一种针对rdh12突变的基因治疗药物

Family Cites Families (45)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US5436146A (en) 1989-09-07 1995-07-25 The Trustees Of Princeton University Helper-free stocks of recombinant adeno-associated virus vectors
US6268213B1 (en) 1992-06-03 2001-07-31 Richard Jude Samulski Adeno-associated virus vector and cis-acting regulatory and promoter elements capable of expressing at least one gene and method of using same for gene therapy
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
US5869305A (en) 1992-12-04 1999-02-09 The University Of Pittsburgh Recombinant viral vector system
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
US6093570A (en) 1995-06-07 2000-07-25 The University Of North Carolina At Chapel Hill Helper virus-free AAV production
US5741683A (en) 1995-06-07 1998-04-21 The Research Foundation Of State University Of New York In vitro packaging of adeno-associated virus DNA
EP1007637B1 (en) 1997-04-14 2004-06-30 Cell Genesys, Inc. Methods for increasing the efficiency of recombinant aav product
JP2001517454A (ja) 1997-09-19 2001-10-09 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 組換えアデノ随伴ウイルスの産生に有用な方法および細胞株
US6146874A (en) 1998-05-27 2000-11-14 University Of Florida Method of preparing recombinant adeno-associated virus compositions
EP1135468B1 (en) 1998-11-10 2010-01-06 University Of North Carolina At Chapel Hill Virus vectors and methods of making and administering the same
US6596535B1 (en) 1999-08-09 2003-07-22 Targeted Genetics Corporation Metabolically activated recombinant viral vectors and methods for the preparation and use
AU6814901A (en) 2000-06-01 2001-12-11 Univ North Carolina Methods and compounds for controlled release of recombinant parvovirus vectors
CA2442670A1 (en) 2001-04-13 2002-10-24 The Trustees Of The University Of Pennsylvania Method of treating or retarding the development of blindness
CN103589692B (zh) 2001-11-13 2018-07-24 宾夕法尼亚大学托管会 检测和/或鉴定腺伴随病毒(aav)序列以及分离所鉴定的新型序列的方法
DK2359869T3 (en) 2001-12-17 2019-04-15 Univ Pennsylvania Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof
AU2003274397A1 (en) 2002-06-05 2003-12-22 University Of Florida Production of pseudotyped recombinant aav virions
WO2004058166A2 (en) 2002-12-23 2004-07-15 Vical Incorporated Codon-optimized polynucleotide-based vaccines against human cytomegalovirus infection
EP1486567A1 (en) 2003-06-11 2004-12-15 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Improved adeno-associated virus (AAV) vector for gene therapy
EP1668143B1 (en) 2003-09-30 2013-03-20 The Trustees of The University of Pennsylvania Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
US7422840B2 (en) 2004-11-12 2008-09-09 E.I. Du Pont De Nemours And Company Apparatus and process for forming a printing form having a cylindrical support
DE23193606T1 (de) 2005-04-07 2024-02-15 The Trustees Of The University Of Pennsylvania Verfahren zur erhöhung der funktion eines aav-vektors
JP4495210B2 (ja) 2005-06-09 2010-06-30 パナソニック株式会社 振幅誤差補償装置及び直交度誤差補償装置
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
US20100081707A1 (en) 2008-02-21 2010-04-01 Ali Robin R Devices and methods for delivering polynucleotides into retinal cells of the macula and fovea
US7561973B1 (en) 2008-07-31 2009-07-14 Dna Twopointo, Inc. Methods for determining properties that affect an expression property value of polynucleotides in an expression system
US7561972B1 (en) 2008-06-06 2009-07-14 Dna Twopointo, Inc. Synthetic nucleic acids for expression of encoded proteins
US20120172419A1 (en) * 2009-09-15 2012-07-05 Medical College Of Wisconsin Research Foundation Inc. Reagents and methods for modulating cone photoreceptor activity
US9315825B2 (en) 2010-03-29 2016-04-19 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
WO2011126808A2 (en) 2010-03-29 2011-10-13 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
HUE041571T2 (hu) * 2010-04-23 2019-05-28 Univ Florida RAAV-guanilát-cikláz-készítmények és ejárások 1-es típusú Leber-féle veleszületett vakság (LCA1) kezelésére
US9249425B2 (en) 2011-05-16 2016-02-02 The Trustees Of The University Of Pennslyvania Proviral plasmids and production of recombinant adeno-associated virus
WO2013151664A1 (en) 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of proteins
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
US9719106B2 (en) 2013-04-29 2017-08-01 The Trustees Of The University Of Pennsylvania Tissue preferential codon modified expression cassettes, vectors containing same, and uses thereof
US8962449B1 (en) 2013-07-30 2015-02-24 Micron Technology, Inc. Methods for processing semiconductor devices
CA2930282C (en) 2013-11-20 2024-09-10 Universita Degli Studi Di Napoli Federico Ii Artificial DNA-Binding Proteins and Their Uses
ES2822551T3 (es) * 2014-03-17 2021-05-04 Adverum Biotechnologies Inc Composiciones y métodos para la expresión génica mejorada en células de los conos
LT3137497T (lt) 2014-05-02 2021-07-26 Genzyme Corporation Aav vektoriai, skirti tinklainės ir cns genų terapijai
WO2016019364A1 (en) * 2014-08-01 2016-02-04 The Trustees Of The University Of Pennsylvania Compositions and methods for self-regulated inducible gene expression
WO2017191274A2 (en) * 2016-05-04 2017-11-09 Curevac Ag Rna encoding a therapeutic protein
US11197936B2 (en) 2016-07-08 2021-12-14 The Trustees Of The University Of Pennsylvania Methods and compositions for treatment of disorders and diseases involving RDH12
SMT202400321T1 (it) 2017-11-15 2024-11-15 Univ Michigan Regents Vettori virali comprendenti regioni codificanti rdh12 e metodi di trattamento di distrofie retiniche
TR2021022284A2 (tr) 2021-12-31 2022-01-21 T C Ueskuedar Ueniversitesi Retinitis pigmentosa (rp) hastalığının tedavisinde kullanılmak üzere retinol dehidrojenaz 12 (rdh12) gen mutasyonlarına yönelik crıspr-pe sistemi.

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