TWI780401B - 用於色素性視網膜炎的基因療法 - Google Patents

用於色素性視網膜炎的基因療法 Download PDF

Info

Publication number
TWI780401B
TWI780401B TW109103078A TW109103078A TWI780401B TW I780401 B TWI780401 B TW I780401B TW 109103078 A TW109103078 A TW 109103078A TW 109103078 A TW109103078 A TW 109103078A TW I780401 B TWI780401 B TW I780401B
Authority
TW
Taiwan
Prior art keywords
mir
rhodopsin
nucleic acid
seq
aav
Prior art date
Application number
TW109103078A
Other languages
English (en)
Chinese (zh)
Other versions
TW202021627A (zh
Inventor
凱瑟琳 歐里爾丹
馬修 亞達莫威茲
Original Assignee
美商健臻公司
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=52829343&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=TWI780401(B) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by 美商健臻公司 filed Critical 美商健臻公司
Publication of TW202021627A publication Critical patent/TW202021627A/zh
Application granted granted Critical
Publication of TWI780401B publication Critical patent/TWI780401B/zh

Links

Images

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • 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
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • 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
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141—MicroRNAs, miRNAs
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/34—Allele or polymorphism specific uses
    • 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
    • C12N2330/00—Production
    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
    • C12N2330/51—Specially adapted vectors
    • 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
    • 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
    • C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Virology (AREA)
  • Immunology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Dermatology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
TW109103078A 2014-03-21 2015-03-23 用於色素性視網膜炎的基因療法 TWI780401B (zh)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201461969027P 2014-03-21 2014-03-21
US61/969,027 2014-03-21

Publications (2)

Publication Number Publication Date
TW202021627A TW202021627A (zh) 2020-06-16
TWI780401B true TWI780401B (zh) 2022-10-11

Family

ID=52829343

Family Applications (2)

Application Number Title Priority Date Filing Date
TW109103078A TWI780401B (zh) 2014-03-21 2015-03-23 用於色素性視網膜炎的基因療法
TW104109152A TWI706789B (zh) 2014-03-21 2015-03-23 用於色素性視網膜炎的基因療法

Family Applications After (1)

Application Number Title Priority Date Filing Date
TW104109152A TWI706789B (zh) 2014-03-21 2015-03-23 用於色素性視網膜炎的基因療法

Country Status (36)

Country Link
US (3) US10383953B2 (enExample)
EP (3) EP4345165A3 (enExample)
JP (3) JP6669664B2 (enExample)
KR (2) KR20160127832A (enExample)
CN (2) CN115252823A (enExample)
AR (1) AR099837A1 (enExample)
AU (3) AU2015230942B2 (enExample)
CA (2) CA2943185C (enExample)
CL (1) CL2016002333A1 (enExample)
CR (1) CR20160480A (enExample)
DK (2) DK3628334T5 (enExample)
DO (1) DOP2016000237A (enExample)
EA (1) EA201691891A1 (enExample)
EC (1) ECSP16083000A (enExample)
ES (2) ES2957840T3 (enExample)
FI (1) FI3628334T3 (enExample)
HR (2) HRP20192141T1 (enExample)
HU (2) HUE046454T2 (enExample)
IL (3) IL247543B (enExample)
LT (2) LT3119437T (enExample)
MA (1) MA39390B2 (enExample)
MX (2) MX376190B (enExample)
MY (2) MY203654A (enExample)
NZ (1) NZ724622A (enExample)
PE (1) PE20161252A1 (enExample)
PH (1) PH12016501684B1 (enExample)
PL (2) PL3119437T3 (enExample)
PT (2) PT3119437T (enExample)
RS (2) RS64611B1 (enExample)
SG (3) SG10201808218YA (enExample)
SI (2) SI3119437T1 (enExample)
TW (2) TWI780401B (enExample)
UA (1) UA120050C2 (enExample)
UY (1) UY36044A (enExample)
WO (1) WO2015143418A2 (enExample)
ZA (1) ZA201605924B (enExample)

Families Citing this family (27)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2943185C (en) 2014-03-21 2025-09-16 Genzyme Corp Gene therapy for retinitis pigmentosa
HK1247641A1 (zh) 2015-02-10 2018-09-28 Genzyme Corporation 变体RNAi
ES2886664T3 (es) * 2015-12-03 2021-12-20 Friedrich Miescher Institute For Biomedical Res SynP162, un promotor para la expresión específica de genes en fotorreceptores de bastón
US10994026B2 (en) 2015-12-03 2021-05-04 Friedrich Miescher Institute For Biomedical Research SynP160, a promoter for the specific expression of genes in rod photoreceptors
AU2016363830C1 (en) * 2015-12-04 2022-03-24 Centre National De La Recherche Scientifique Promoters and uses thereof
AU2017227776C1 (en) * 2016-03-01 2024-01-25 The Trustees Of The University Of Pennsylvania AAV vectors for treatment of dominant retinitis pigmentosa
EP3481434A4 (en) * 2016-07-05 2020-06-24 The Johns Hopkins University Crispr/cas9-based compositions and methods for treating retinal degenerations
CA3056211A1 (en) * 2017-03-21 2018-09-27 Stylianos MICHALAKIS Gene therapy for the treatment of cngb1-linked retinitis pigmentosa
EP3684932A1 (en) 2017-09-22 2020-07-29 Genzyme Corporation Variant rnai
BR112020006149A2 (pt) * 2017-09-27 2020-10-20 Sigilon Therapeutics, Inc. célula ativa manipulada, elemento implantável, e, composição farmacêutica.
CN107961380B (zh) * 2017-11-23 2020-05-05 清华大学 试剂在制备药物中的用途、筛选药物的方法以及药物组合物
US12162994B2 (en) 2018-03-02 2024-12-10 Sigilon Therapeutics, Inc. Biocompatible hydrogel capsules and process for preparing same
US20210017509A1 (en) * 2018-03-23 2021-01-21 The Trustees Of Columbia University In The City Of New York Gene Editing for Autosomal Dominant Diseases
UY38160A (es) 2018-04-04 2019-11-29 Sigilon Therapeutics Inc Partículas implantables y métodos relacionados
EP3802836A4 (en) 2018-06-01 2022-10-19 University of Florida Research Foundation, Incorporated COMPOSITIONS AND METHODS FOR THE TREATMENT OF DOMINANT RETINITIS PIGMENTOSA
GB201817469D0 (en) * 2018-10-26 2018-12-12 Univ Oxford Innovation Ltd Gene therapy for retinal disease
CN111518813B (zh) * 2019-02-03 2023-04-28 武汉纽福斯生物科技有限公司 视紫红质的编码序列、其表达载体构建及其应用
WO2020180886A1 (en) * 2019-03-04 2020-09-10 The Trustees Of The University Of Pennsylvania Neuroprotective gene therapy targeting the akt pathway
BR112021018354A2 (pt) * 2019-03-21 2021-11-23 Ptc Therapeutics Inc Vetor e método para tratar síndrome de angelman
CN114555084A (zh) * 2019-08-15 2022-05-27 费城儿童医院 用于治疗SCA1的转基因和内含子衍生miRNA联合疗法
AU2021205965A1 (en) * 2020-01-10 2022-09-01 Solid Biosciences Inc. Viral vector for combination therapy
KR20220133941A (ko) * 2020-01-29 2022-10-05 젠자임 코포레이션 안과 유전자 치료를 위한 변형 아데노-관련 바이러스 캡시드 단백질 및 이의 사용 방법
CN114521214A (zh) * 2020-07-24 2022-05-20 尔知渃米斯股份有限公司 视紫红质转录体特异性反式剪接核酶及其用途
WO2022221405A1 (en) * 2021-04-16 2022-10-20 Regeneron Pharmaceuticals, Inc. Treatment of x-linked juvenile retinoschisis
TWI832531B (zh) * 2022-11-02 2024-02-11 慈濟學校財團法人慈濟大學 Rip1抑制劑或mlkl抑制劑用於治療或預防遺傳性視網膜失養症的用途
CN120098992A (zh) * 2023-12-05 2025-06-06 云舟生物科技(广州)股份有限公司 核酸分子及其作为特异启动子的应用
CN120230798A (zh) * 2023-12-29 2025-07-01 上海朗昇生物科技有限公司 用于视网膜基因递送的重组腺相关病毒载体及其应用

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2008125846A2 (en) * 2007-04-12 2008-10-23 The Provost, Fellows And Scholars Of The College Of The Holy And Undivided Trinity Of Queen Elizabeth Genetic suppression and replacement

Family Cites Families (30)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5262529A (en) * 1990-01-24 1993-11-16 President And Fellows Of Harvard College Diagnosis of hereditary retinal degenerative diseases
US5985583A (en) * 1992-06-23 1999-11-16 Mount Sinai School Of Medicine Of The City University Of New York Cloning and expression of gonadotropin-releasing hormone receptor
US6989264B2 (en) 1997-09-05 2006-01-24 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
EP2369002A1 (en) 1999-08-09 2011-09-28 Targeted Genetics Corporation Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms intrastrand base pairs
ES2256265T3 (es) 2000-06-01 2006-07-16 University Of North Carolina At Chapel Hill Vectores de parvovirus duplicados.
NZ618298A (en) 2001-11-13 2015-04-24 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
EP1486567A1 (en) 2003-06-11 2004-12-15 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Improved adeno-associated virus (AAV) vector for gene therapy
US8741650B2 (en) * 2004-01-22 2014-06-03 Dnavec Research Inc. Methods for producing minus-strand RNA viral vectors using hybrid promoter comprising cytomegalovirus enhancer and chicken β-actin promoter
US7765583B2 (en) 2005-02-28 2010-07-27 France Telecom System and method for managing virtual user domains
US8283151B2 (en) 2005-04-29 2012-10-09 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Isolation, cloning and characterization of new adeno-associated virus (AAV) serotypes
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
US20090214478A1 (en) * 2008-02-21 2009-08-27 Alberto Auricchio Method of treating ocular diseases by gene therapy
US9217155B2 (en) * 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
DK2475372T4 (da) * 2009-09-10 2020-11-30 Velin Pharma As Fremgangsmåde til fremstilling af micro-RNA og dets terapeutiske anvendelse
CN102947453A (zh) * 2010-01-28 2013-02-27 费城儿童医院 用于病毒载体纯化的可扩缩生产平台和用于基因治疗中的如此纯化的病毒载体
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
JP5704361B2 (ja) 2010-10-27 2015-04-22 学校法人自治医科大学 神経系細胞への遺伝子導入のためのアデノ随伴ウイルスビリオン
SG10201601110VA (en) 2011-02-17 2016-03-30 Univ Pennsylvania Compositions and methods for altering tissue specificity and improving aav9-mediated gene transfer
JP5884152B2 (ja) 2011-07-29 2016-03-15 シャープ株式会社 基地局、端末、通信システムおよび通信方法
ITRM20110685A1 (it) * 2011-12-23 2013-06-24 Internat Ct For Genetic En Gineering And Microrna per la rigenerazione cardiaca attraverso l induzione della proliferazione dei miociti cardiaci
RU2653444C2 (ru) * 2012-02-17 2018-05-08 Дзе Чилдрен'З Хоспитал Оф Филадельфия Композиции вектора aav и способы переноса генов в клетки, органы и ткани
WO2013151663A1 (en) * 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of membrane proteins
US9127274B2 (en) * 2012-04-26 2015-09-08 Alnylam Pharmaceuticals, Inc. Serpinc1 iRNA compositions and methods of use thereof
WO2013176772A1 (en) 2012-05-25 2013-11-28 The Regents Of The University Of California Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
AU2013287281B2 (en) * 2012-07-11 2018-04-26 The Trustees Of The University Of Pennsylvania AAV-mediated gene therapy for RPGR x-linked retinal degeneration
EP2912177A1 (en) * 2012-10-23 2015-09-02 Cornell University Treatment of metastatic breast cancer
EP3052134B1 (en) 2013-10-04 2021-05-05 Merck Sharp & Dohme Corp. Glucose-responsive insulin conjugates
CA2943185C (en) 2014-03-21 2025-09-16 Genzyme Corp Gene therapy for retinitis pigmentosa

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2008125846A2 (en) * 2007-04-12 2008-10-23 The Provost, Fellows And Scholars Of The College Of The Holy And Undivided Trinity Of Queen Elizabeth Genetic suppression and replacement

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
期刊 Behrman S et al. "A CHOP-regulated microRNA controls rhodopsin expression" The Journal of Cell Biology vol.192 no.6 The Rockefeller University Press 2011/03/14 p.919-927 *

Also Published As

Publication number Publication date
MY190726A (en) 2022-05-12
JP2022084810A (ja) 2022-06-07
IL284741A (en) 2021-08-31
UY36044A (es) 2015-10-30
US10383953B2 (en) 2019-08-20
US20170173183A1 (en) 2017-06-22
AU2015230942B2 (en) 2020-11-19
US20200046851A1 (en) 2020-02-13
AU2024227776A1 (en) 2024-11-28
ES2760263T3 (es) 2020-05-13
IL275918A (en) 2020-08-31
HUE063460T2 (hu) 2024-01-28
IL247543A0 (en) 2016-11-30
BR112016021017A2 (pt) 2017-10-03
JP7048563B2 (ja) 2022-04-05
CN106456660B (zh) 2022-05-31
IL247543B (en) 2020-07-30
JP7534348B2 (ja) 2024-08-14
EP4345165A2 (en) 2024-04-03
ES2957840T3 (es) 2024-01-26
PH12016501684B1 (en) 2023-03-17
NZ762841A (en) 2023-11-24
PT3628334T (pt) 2023-09-26
EP3628334A1 (en) 2020-04-01
EP3119437B1 (en) 2019-09-04
CA2943185C (en) 2025-09-16
HRP20231077T1 (hr) 2023-12-22
EP3119437A2 (en) 2017-01-25
CL2016002333A1 (es) 2017-05-26
CA3254798A1 (en) 2025-12-01
WO2015143418A2 (en) 2015-09-24
CN106456660A (zh) 2017-02-22
EP4345165A3 (en) 2024-09-25
IL284741B (en) 2022-10-01
FI3628334T3 (fi) 2023-09-15
AR099837A1 (es) 2016-08-24
LT3119437T (lt) 2019-12-27
AU2021200988B2 (en) 2024-08-01
ZA201605924B (en) 2017-09-27
JP2020059737A (ja) 2020-04-16
TWI706789B (zh) 2020-10-11
US12201698B2 (en) 2025-01-21
CN115252823A (zh) 2022-11-01
KR20160127832A (ko) 2016-11-04
AU2015230942A1 (en) 2016-10-13
RS64611B1 (sr) 2023-10-31
DOP2016000237A (es) 2016-10-16
NZ724622A (en) 2022-05-27
MX2020010694A (es) 2020-11-06
SG11201607005UA (en) 2016-09-29
PL3119437T3 (pl) 2020-04-30
MA39390B2 (fr) 2022-04-29
US11103598B2 (en) 2021-08-31
WO2015143418A3 (en) 2015-11-19
EA201691891A1 (ru) 2017-01-30
PT3119437T (pt) 2019-12-12
JP2017509632A (ja) 2017-04-06
EP3628334B1 (en) 2023-06-28
KR20230006039A (ko) 2023-01-10
JP6669664B2 (ja) 2020-03-18
TW202021627A (zh) 2020-06-16
IL275918B (en) 2021-07-29
MY203654A (en) 2024-07-11
HRP20192141T1 (hr) 2020-02-21
MA39390A1 (fr) 2017-12-29
TW201622752A (zh) 2016-07-01
MX376190B (es) 2025-03-07
DK3119437T3 (da) 2019-12-09
AU2021200988A1 (en) 2021-03-11
US20220054657A1 (en) 2022-02-24
UA120050C2 (uk) 2019-09-25
ECSP16083000A (es) 2017-02-24
DK3628334T3 (da) 2023-10-02
DK3628334T5 (da) 2024-09-02
CR20160480A (es) 2016-12-14
PL3628334T3 (pl) 2023-12-18
CA2943185A1 (en) 2015-09-24
IL284741B2 (en) 2023-02-01
PE20161252A1 (es) 2016-11-30
LT3628334T (lt) 2023-09-25
RS59634B1 (sr) 2020-01-31
HUE046454T2 (hu) 2020-03-30
SI3119437T1 (sl) 2020-01-31
SI3628334T1 (sl) 2023-11-30
SG10201808218YA (en) 2018-10-30
MX2016012201A (es) 2017-01-19
PH12016501684A1 (en) 2016-10-03
SG10201912968WA (en) 2020-02-27

Similar Documents

Publication Publication Date Title
JP7534348B2 (ja) 網膜色素変性症のための遺伝子治療
US20240197837A1 (en) Gene editing of deep intronic mutations
TWI781079B (zh) 變體ＲＮＡｉ
AU2019314529A1 (en) Variant RNAi against alpha-synuclein
JP2023534037A (ja) シャルコー・マリー・トゥース病の治療に有用な組成物
HK40109446A (en) Gene therapy for retinitis pigmentosa
HK40080889A (en) Gene therapy for retinitis pigmentosa
HK40026944A (en) Gene therapy for retinitis pigmentosa
HK40026944B (en) Gene therapy for retinitis pigmentosa
HK1233535B (en) Gene therapy for retinitis pigmentosa
HK1233535A1 (en) Gene therapy for retinitis pigmentosa
BR112016021017B1 (pt) Partículas de raav, composições e uso das mesmas no tratamento para retinite pigmentosa ou no tratamento do estresse do retículo endoplasmático
TW202449166A (zh) Ｄｍ－１肌強直性營養不良的靶向基因療法
TW201923079A (zh) 變體ＲＮＡｉ

Legal Events

Date Code Title Description
GD4A Issue of patent certificate for granted invention patent