JP7561624B2 - トランススプライシング分子 - Google Patents

トランススプライシング分子 Download PDF

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JP7561624B2
JP7561624B2 JP2020557945A JP2020557945A JP7561624B2 JP 7561624 B2 JP7561624 B2 JP 7561624B2 JP 2020557945 A JP2020557945 A JP 2020557945A JP 2020557945 A JP2020557945 A JP 2020557945A JP 7561624 B2 JP7561624 B2 JP 7561624B2
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abca4
nucleotides
trans
splicing
intron
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JP2021520837A (ja
JPWO2019204514A5 (enExample
Inventor
フィリップ アール． ジョンソン
ブルース シー． シュネップ
ジャン ベネット
スコット ジェイ． ドゥーリー
クリシュナ ジャワハルラール フィッシャー
ジュンウェイ スン
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ザ トラスティーズ オブ ザ ユニバーシティ オブ ペンシルバニア
アスィディアン セラピューティクス インコーポレイテッド
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Publication of JP2021520837A publication Critical patent/JP2021520837A/ja
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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
    • 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
    • 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
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • 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
    • C12N15/1138—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 against receptors or cell surface proteins
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/70—Fusion polypeptide containing domain for protein-protein interaction
    • C07K2319/71—Fusion polypeptide containing domain for protein-protein interaction containing domain for transcriptional activaation, e.g. VP16
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/85—Fusion polypeptide containing an RNA binding domain
    • 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 [CRISPR]
    • 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
    • C12N2320/33—Alteration of splicing
    • 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
    • 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
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Cell Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Immunology (AREA)
  • Epidemiology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Virology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
JP2020557945A 2018-04-17 2019-04-17 トランススプライシング分子 Active JP7561624B2 (ja)

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JP2024073380A JP2024099770A (ja) 2018-04-17 2024-04-30 トランススプライシング分子

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US201862658658P 2018-04-17 2018-04-17
US201862658667P 2018-04-17 2018-04-17
US62/658,658 2018-04-17
US62/658,667 2018-04-17
PCT/US2019/027981 WO2019204514A1 (en) 2018-04-17 2019-04-17 Trans-splicing molecules

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JP2021520837A JP2021520837A (ja) 2021-08-26
JPWO2019204514A5 JPWO2019204514A5 (enExample) 2022-04-25
JP7561624B2 true JP7561624B2 (ja) 2024-10-04

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EP (1) EP3781213A4 (enExample)
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KR (2) KR102866133B1 (enExample)
CN (1) CN112449605A (enExample)
AU (1) AU2019255708A1 (enExample)
BR (1) BR112020021228A2 (enExample)
CA (1) CA3097004A1 (enExample)
IL (1) IL278019A (enExample)
MX (2) MX2020010959A (enExample)
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Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102866133B1 (ko) 2018-04-17 2025-09-30 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 트랜스-스플라이싱 분자
KR20220002910A (ko) * 2019-04-17 2022-01-07 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 효율적인 rna 트랜스-스플라이싱을 위한 삼중 나선 종결인자
EP4284335A1 (en) 2021-02-01 2023-12-06 RegenxBio Inc. Gene therapy for neuronal ceroid lipofuscinoses
JP2024533163A (ja) 2021-09-03 2024-09-12 タシト・セラピューティクス・インコーポレーテッド スプライソソーム構成要素のリクルートを介したｒｎａ編集
WO2023220742A2 (en) * 2022-05-13 2023-11-16 Ascidian Therapeutics, Inc. Abca4 trans-splicing molecules
WO2024011203A2 (en) * 2022-07-07 2024-01-11 Intergalactic Therapeutics, Inc. Ocular vectors and uses thereof
EP4594505A1 (en) * 2022-09-30 2025-08-06 Centre National de la Recherche Scientifique Therapy by trans-splicing of opa1 pre-messenger rnas for the treatment of diseases associated with opa1 gene mutations
AU2024223513A1 (en) * 2023-02-15 2025-09-18 Ascidian Therapeutics, Inc. Htt trans-splicing molecules
WO2025166360A1 (en) * 2024-02-03 2025-08-07 Duke University Compositions for and methods of engineering the transcriptome

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2016516424A (ja) 2013-04-18 2016-06-09 フォンダッツィオーネ・テレソン デュアルａａｖベクターによる大型遺伝子の効果的送達
WO2017087900A1 (en) 2015-11-19 2017-05-26 The Trustees Of The University Of Pennsylvania Compositions and methods for correction of heritable ocular disease

Family Cites Families (40)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0883344B2 (en) 1995-12-15 2010-06-09 VIRxSYS Corporation Therapeutic molecules generated by trans-splicing
US6280978B1 (en) 1995-12-15 2001-08-28 Intronn Holdings, Llc Methods and compositions for use in spliceosome mediated RNA trans-splicing
AU735272B2 (en) 1996-10-04 2001-07-05 Intronn Llc Sample collection devices and methods using markers and the use of such markers as controls in sample validation, laboratory evaluation and/or accreditation
US6929907B2 (en) 1999-12-31 2005-08-16 North Carolina State University Methods and compositions for determining the purity of chemically synthesized nucleic acids
CA2452690C (en) 2001-06-29 2016-08-16 Veri-Q, Inc. Methods and compositions for determining the purity of and purifying chemically synthesized nucleic acids
JP2006505242A (ja) 2002-02-12 2006-02-16 イントロン，インコーポレーテッド スプライセオソームにより媒介されるｒｎａトランス−スプライシングにおいて使用するための方法および組成物
US7399753B2 (en) 2002-02-25 2008-07-15 Virxsys Corporation Trans-splicing mediated photodynamic therapy
EP1485397A4 (en) 2002-02-25 2005-05-25 Intronn Inc IMAGING OF GENE EXPRESSION MEDIATED BY TRANS-SPISSAGE
CA2488249A1 (en) 2002-06-05 2003-12-18 Intronn, Inc. Spliceosome mediated rna trans-splicing in stem cells
EP1521766B1 (en) 2002-06-05 2012-11-07 VIRxSYS Corporation SPLICEOSOME MEDIATED RNA i TRANS /i -SPLICING AND CORRECTION OF FACTOR VIII GENETIC DEFECTS USING SPLICEOSOME MEDIATED RNA TRANS SPLING
US20040018622A1 (en) 2002-07-17 2004-01-29 Mitchell Lloyd G. Spliceosome mediated RNA trans-splicing for correction of skin disorders
DE60333032D1 (de) 2002-10-23 2010-07-29 Virxsys Corp Screening-verfahren zur identifizierung wirksamer prä-trans-spleissmoleküle
WO2005070948A1 (en) 2004-01-23 2005-08-04 Intronn, Inc. Correction of alpha-1-antitrypsin genetic defects using spliceosome mediated rna trans splicing
US20060177933A1 (en) 2004-01-23 2006-08-10 Madaiah Puttaraju Expression of apoA-1 and variants thereof using spliceosome mediated RNA trans-splicing
WO2006026611A2 (en) 2004-08-31 2006-03-09 Mitchell Lloyd G Reverse transcriptase mediated rna gene expression
US7943374B2 (en) 2005-08-21 2011-05-17 Markus Hildinger Super-size adeno-associated viral vector harboring a recombinant genome larger than 5.7 kb
US20090214478A1 (en) 2008-02-21 2009-08-27 Alberto Auricchio Method of treating ocular diseases by gene therapy
US8236557B2 (en) 2008-05-28 2012-08-07 University Of Missouri-Columbia Hybrid-AAV vectors to deliver large gene expression cassette
EP2151248A1 (en) 2008-07-30 2010-02-10 Johann Bauer Improved pre-mRNA trans-splicing molecule (RTM) molecules and their uses
AU2013234376B2 (en) * 2008-10-22 2016-07-28 Acucela, Inc. Compounds for treating ophthalmic diseases and disorders
US8648053B2 (en) 2010-10-20 2014-02-11 Rosalind Franklin University Of Medicine And Science Antisense oligonucleotides that target a cryptic splice site in Ush1c as a therapeutic for Usher syndrome
WO2012158757A1 (en) 2011-05-16 2012-11-22 The Trustees Of The University Of Pennsylvania Proviral plasmids for production of recombinant adeno-associated virus
PL2753694T3 (pl) * 2011-09-05 2018-01-31 Stichting Katholieke Univ Oligonukleotydy antysensowne do leczenia wrodzonej ślepoty Lebera
CA2878171C (en) 2012-07-11 2021-04-27 The Trustees Of The University Of Pennsylvania Aav-mediated gene therapy for rpgr x-linked retinal degeneration
WO2014062801A1 (en) 2012-10-16 2014-04-24 Massachusetts Institute Of Technology Production of stable non-polyadenylated rnas
GB201219762D0 (en) 2012-11-02 2012-12-19 Bauer Johann A RNA trans-splicing molecule (RTM) for use in the treatment of cancer
US10266845B2 (en) 2013-02-08 2019-04-23 The Trustees Of The University Of Pennsylvania Enhanced AAV-mediated gene transfer for retinal therapies
US9567376B2 (en) 2013-02-08 2017-02-14 The Trustees Of The University Of Pennsylvania Enhanced AAV-mediated gene transfer for retinal therapies
WO2015009575A1 (en) 2013-07-16 2015-01-22 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of disorders related to cep290
EP3553176A1 (en) 2014-03-10 2019-10-16 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating leber's congenital amaurosis 10 (lca10)
JP6930910B2 (ja) 2014-07-09 2021-09-01 レクソジェン・ゲゼルシャフト・ミット・ベシュレンクテル・ハフツングＬＥＸＯＧＥＮ ＧｍｂＨ Ｒｎａ転写産物バリアントを定量するための方法及び製品
DK3265571T3 (da) * 2015-03-03 2022-06-27 Fond Telethon Fler-vektorsystem og anvendelse heraf
EP3390634A4 (en) * 2015-12-14 2019-08-14 Cold Spring Harbor Laboratory COMPOSITIONS AND METHODS FOR THE TREATMENT OF EYE DISEASES
TWI820034B (zh) 2017-07-31 2023-11-01 香港商映像生物有限公司 眼部疾病之細胞模式及用於眼部疾病的療法
US11795455B2 (en) 2017-07-31 2023-10-24 Massachusetts Institute Of Technology RNA cleavage-induced transcript stabilizer and uses thereof
KR102866133B1 (ko) 2018-04-17 2025-09-30 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 트랜스-스플라이싱 분자
KR20220002910A (ko) 2019-04-17 2022-01-07 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 효율적인 rna 트랜스-스플라이싱을 위한 삼중 나선 종결인자
WO2020214990A1 (en) 2019-04-17 2020-10-22 Ascidian Therapeutics, Inc. Abca4 cell line and methods of use
EP3962295A1 (en) 2019-05-01 2022-03-09 The Procter & Gamble Company Probiotic bacterial strains that produce short chain fatty acids and compositions comprising same
WO2023220742A2 (en) 2022-05-13 2023-11-16 Ascidian Therapeutics, Inc. Abca4 trans-splicing molecules

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2016516424A (ja) 2013-04-18 2016-06-09 フォンダッツィオーネ・テレソン デュアルａａｖベクターによる大型遺伝子の効果的送達
WO2017087900A1 (en) 2015-11-19 2017-05-26 The Trustees Of The University Of Pennsylvania Compositions and methods for correction of heritable ocular disease

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
Colella, P. et al.，Efficient gene delivery to the coneenriched pig retina by dual AAV vectors，Investigative Ophthalmology and Visual Science，2014年，Vol. 55(13)，URL: https://iovs.arvojournals.org/article.aspx?articleid=2268747，[retrieved on 2023.5.17]

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KR20250150157A (ko) 2025-10-17
EP3781213A1 (en) 2021-02-24
KR20210018222A (ko) 2021-02-17
JP2021520837A (ja) 2021-08-26
BR112020021228A2 (pt) 2021-03-02
AU2019255708A1 (en) 2020-11-26
MX2020010959A (es) 2021-01-15
US20240318186A1 (en) 2024-09-26
IL278019A (en) 2020-11-30
JP2024099770A (ja) 2024-07-25
CA3097004A1 (en) 2019-10-24
CN112449605A (zh) 2021-03-05
KR102866133B1 (ko) 2025-09-30
WO2019204514A1 (en) 2019-10-24
US20210155938A1 (en) 2021-05-27
MX2025007209A (es) 2025-07-01
US12442003B2 (en) 2025-10-14
EP3781213A4 (en) 2022-10-19
US11993776B2 (en) 2024-05-28

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