DK3628334T5 - Genterapi til behandling af retinitis pigmentosa - Google Patents

Genterapi til behandling af retinitis pigmentosa Download PDF

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Publication number
DK3628334T5
DK3628334T5 DK19186929.6T DK19186929T DK3628334T5 DK 3628334 T5 DK3628334 T5 DK 3628334T5 DK 19186929 T DK19186929 T DK 19186929T DK 3628334 T5 DK3628334 T5 DK 3628334T5
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Prior art keywords
treatment
gene therapy
retinitis pigmentosa
pigmentosa
retinitis
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DK19186929.6T
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Danish (da)
English (en)
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DK3628334T3 (da
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Catherine O'riordan
Matthew Adamowicz
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Genzyme Corp
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    • 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
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    • 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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    • 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
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • 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
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    • C12N2330/00—Production
    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
    • C12N2330/51—Specially adapted vectors
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    • 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
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    • 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

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  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
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  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biomedical Technology (AREA)
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  • Zoology (AREA)
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  • Epidemiology (AREA)
  • Wood Science & Technology (AREA)
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  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Ophthalmology & Optometry (AREA)
  • Virology (AREA)
  • Immunology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Marine Sciences & Fisheries (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)
DK19186929.6T 2014-03-21 2015-03-20 Genterapi til behandling af retinitis pigmentosa DK3628334T5 (da)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201461969027P 2014-03-21 2014-03-21
EP15716232.2A EP3119437B1 (en) 2014-03-21 2015-03-20 Gene therapy for retinitis pigmentosa

Publications (2)

Publication Number Publication Date
DK3628334T3 DK3628334T3 (da) 2023-10-02
DK3628334T5 true DK3628334T5 (da) 2024-09-02

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DK19186929.6T DK3628334T5 (da) 2014-03-21 2015-03-20 Genterapi til behandling af retinitis pigmentosa
DK15716232T DK3119437T3 (da) 2014-03-21 2015-03-20 Genterapi mod retinitis pigmentosa

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DK15716232T DK3119437T3 (da) 2014-03-21 2015-03-20 Genterapi mod retinitis pigmentosa

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US (3) US10383953B2 (cg-RX-API-DMAC7.html)
EP (3) EP3119437B1 (cg-RX-API-DMAC7.html)
JP (3) JP6669664B2 (cg-RX-API-DMAC7.html)
KR (3) KR20160127832A (cg-RX-API-DMAC7.html)
CN (2) CN106456660B (cg-RX-API-DMAC7.html)
AR (1) AR099837A1 (cg-RX-API-DMAC7.html)
AU (3) AU2015230942B2 (cg-RX-API-DMAC7.html)
CA (2) CA2943185C (cg-RX-API-DMAC7.html)
CL (1) CL2016002333A1 (cg-RX-API-DMAC7.html)
CR (1) CR20160480A (cg-RX-API-DMAC7.html)
DK (2) DK3628334T5 (cg-RX-API-DMAC7.html)
DO (1) DOP2016000237A (cg-RX-API-DMAC7.html)
EA (1) EA201691891A1 (cg-RX-API-DMAC7.html)
EC (1) ECSP16083000A (cg-RX-API-DMAC7.html)
ES (2) ES2957840T3 (cg-RX-API-DMAC7.html)
FI (1) FI3628334T3 (cg-RX-API-DMAC7.html)
HR (2) HRP20192141T1 (cg-RX-API-DMAC7.html)
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LT (2) LT3119437T (cg-RX-API-DMAC7.html)
MA (1) MA39390B2 (cg-RX-API-DMAC7.html)
MX (2) MX376190B (cg-RX-API-DMAC7.html)
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PL (2) PL3119437T3 (cg-RX-API-DMAC7.html)
PT (2) PT3628334T (cg-RX-API-DMAC7.html)
RS (2) RS59634B1 (cg-RX-API-DMAC7.html)
SG (3) SG10201808218YA (cg-RX-API-DMAC7.html)
SI (2) SI3628334T1 (cg-RX-API-DMAC7.html)
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WO (1) WO2015143418A2 (cg-RX-API-DMAC7.html)
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Families Citing this family (28)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20160127832A (ko) 2014-03-21 2016-11-04 젠자임 코포레이션 색소성 망막염의 유전자 치료
JP2018506304A (ja) 2015-02-10 2018-03-08 ジェンザイム・コーポレーション バリアントＲＮＡｉ
WO2017093934A1 (en) * 2015-12-03 2017-06-08 Friedrich Miescher Institute For Biomedical Research Synp160, a promoter for the specific expression of genes in rod photoreceptors
US11059871B2 (en) 2015-12-03 2021-07-13 Friedrich Miescher Institute For Biomedical Research SYNP162, a promoter for the specific expression of genes in rod photoreceptors
CA3004807C (en) * 2015-12-04 2022-02-22 Universite Pierre Et Marie Curie (Paris 6) 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
CN109890424A (zh) * 2016-07-05 2019-06-14 约翰霍普金斯大学 用于治疗视网膜变性的基于crispr/cas9的组合物和方法
EP3600446A1 (en) * 2017-03-21 2020-02-05 Michalakis, Stylianos Gene therapy for the treatment of cngb1-linked retinitis pigmentosa
MX2020003095A (es) * 2017-09-22 2020-10-15 Genzyme Corp Arni variante.
CA3077380A1 (en) * 2017-09-27 2019-04-04 Sigilon Therapeutics, Inc. Methods, compositions, and implantable elements comprising active cells
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
EP4186921A1 (en) * 2018-03-23 2023-05-31 The Trustees of Columbia University in the City of New York Gene editing for autosomal dominant diseases
IL277713B2 (en) 2018-04-04 2024-09-01 Sigilon Therapeutics Inc Implantable particles and related methods
US12203074B2 (en) 2018-06-01 2025-01-21 University Of Florida Research Foundation, Incorporated Compositions and methods for 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
EP3934699A4 (en) * 2019-03-04 2022-12-21 The Trustees of The University of Pennsylvania NEUROPROTECTIVE GENE THERAPY TARGETING THE AKT PATHWAY
EA202192543A1 (ru) * 2019-03-21 2021-12-27 Птс Терапьютикс, Инк. Вектор и способ для лечения синдрома ангельмана
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
WO2021142447A1 (en) * 2020-01-10 2021-07-15 Solid Biosciences Inc. Viral vector for combination therapy
US20210261625A1 (en) * 2020-01-29 2021-08-26 Genzyme Corporation Modified adeno-associated viral capsid proteins for ocular gene therapy and methods of use thereof
KR102396200B1 (ko) * 2020-07-24 2022-05-12 알지노믹스 주식회사 로돕신 전사체에 특이적인 트랜스-스플라이싱 라이보자임 및 이의 용도
CA3190214A1 (en) * 2020-07-29 2022-02-03 University Of Florida Research Foundation, Incorporated Improved aav-mediated x-linked retinoschisis therapies
EP4323390A1 (en) * 2021-04-16 2024-02-21 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

Family Cites Families (31)

* 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
US6995006B2 (en) 1997-09-05 2006-02-07 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
CA2379166C (en) 1999-08-09 2013-03-26 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 instrastrand base pairs
ES2505700T3 (es) 2000-06-01 2014-10-10 University Of North Carolina At Chapel Hill Vectores de parvovirus duplicados
ES2258601T3 (es) 2001-11-13 2006-09-01 The Trustees Of The University Of Pennsylvania Un metodo para la identificacion de las secuencias desconocidas del virus adeno-asociado (vaa) y un kit para el metodo.
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
WO2006119432A2 (en) 2005-04-29 2006-11-09 The Government Of The U.S.A., As Rep. By The Sec., Dept. Of Health & Human Services Isolation, cloning and characterization of new adeno-associated virus (aav) serotypes
EP2007795B1 (en) 2006-03-30 2016-11-16 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid proteins
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
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
US9078914B2 (en) 2009-09-10 2015-07-14 Velin-Pharma A/S Method for the preparation of micro-RNA and its therapeutic application
ES2680915T3 (es) * 2010-01-28 2018-09-11 The Children's Hospital Of Philadelphia Plataforma de fabricación escalable para la purificación de vectores virales y vectores virales purificados de este modo para su utilización en terapia génica
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
EP2634253B1 (en) 2010-10-27 2016-05-11 Jichi Medical University Adeno-associated virus virions for transferring genes into neural cells
US9884071B2 (en) 2011-02-17 2018-02-06 The Trustees Of The University Of 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
AU2013221212B2 (en) * 2012-02-17 2018-08-09 The Children's Hospital Of Philadelphia AAV vector compositions and methods for gene transfer to cells, organs and tissues
EP2834260A4 (en) * 2012-04-02 2016-08-10 Moderna Therapeutics Inc MODIFIED POLYNUCLEOTIDES FOR THE PREPARATION 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
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
EP2912177A1 (en) * 2012-10-23 2015-09-02 Cornell University Treatment of metastatic breast cancer
CN105792852B (zh) 2013-10-04 2019-12-10 默沙东公司 葡萄糖响应性的胰岛素缀合物
KR20160127832A (ko) 2014-03-21 2016-11-04 젠자임 코포레이션 색소성 망막염의 유전자 치료

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