CN115038789A - 治疗性编辑 - Google Patents

治疗性编辑 Download PDF

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Publication number
CN115038789A
CN115038789A CN202080095469.1A CN202080095469A CN115038789A CN 115038789 A CN115038789 A CN 115038789A CN 202080095469 A CN202080095469 A CN 202080095469A CN 115038789 A CN115038789 A CN 115038789A
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target rna
rna
polypeptide
sequence
composition
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CN202080095469.1A
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Chinese (zh)
Inventor
大卫·胡斯
普拉桑特·马里
阿努帕玛·拉克什马南
克里斯托弗·奈耶
扬尼斯·萨瓦
利亚纳·斯泰因
理查德·沙利文
拉斐尔·庞塞
苏珊·伯恩
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Shape Therapy Co
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Shape Therapy Co
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    • C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
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    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/533—Physical structure partially self-complementary or closed having a mismatch or nick in at least one of the strands
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    • 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
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CN202080095469.1A 2019-12-02 2020-12-01 治疗性编辑 Pending CN115038789A (zh)

Applications Claiming Priority (13)

Application Number Priority Date Filing Date Title
US201962942693P 2019-12-02 2019-12-02
US201962942683P 2019-12-02 2019-12-02
US201962942667P 2019-12-02 2019-12-02
US62/942,667 2019-12-02
US62/942,693 2019-12-02
US62/942,683 2019-12-02
US202063022727P 2020-05-11 2020-05-11
US63/022,727 2020-05-11
US202063030165P 2020-05-26 2020-05-26
US63/030,165 2020-05-26
US202063112286P 2020-11-11 2020-11-11
US63/112,286 2020-11-11
PCT/US2020/062756 WO2021113270A1 (en) 2019-12-02 2020-12-01 Therapeutic editing

Publications (1)

Publication Number Publication Date
CN115038789A true CN115038789A (zh) 2022-09-09

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CN202080095469.1A Pending CN115038789A (zh) 2019-12-02 2020-12-01 治疗性编辑

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US (2) US11827880B2 (https=)
EP (1) EP4069842A1 (https=)
JP (1) JP2023504314A (https=)
CN (1) CN115038789A (https=)
AU (1) AU2020395113A1 (https=)
CA (1) CA3159944A1 (https=)
WO (1) WO2021113270A1 (https=)

Families Citing this family (39)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017220751A1 (en) 2016-06-22 2017-12-28 Proqr Therapeutics Ii B.V. Single-stranded rna-editing oligonucleotides
CN110352244B (zh) 2016-09-01 2023-03-21 ProQR治疗上市公司Ⅱ 化学修饰的编辑rna的单链寡核苷酸
GB201808146D0 (en) 2018-05-18 2018-07-11 Proqr Therapeutics Ii Bv Stereospecific Linkages in RNA Editing Oligonucleotides
AU2020395113A1 (en) 2019-12-02 2022-06-09 Shape Therapeutics Inc. Therapeutic editing
US20230059753A1 (en) * 2019-12-09 2023-02-23 Astellas Pharma Inc. Antisense guide rna with added functional region for editing target rna
CN115777020A (zh) * 2020-04-22 2023-03-10 塑造治疗公司 使用snrna组分的组合物和方法
JP2024500279A (ja) * 2020-11-11 2024-01-09 シェイプ セラピューティクス インコーポレイテッド Ｒｎａ編集組成物及び使用方法
WO2022268835A1 (en) * 2021-06-21 2022-12-29 Uniqure Biopharma B.V. Gene constructs for silencing alpha-synuclein and uses thereof
CA3223109A1 (en) * 2021-06-29 2023-01-05 Richard Thomas SULLIVAN Engineered guide rnas and polynucleotides
WO2023278436A1 (en) * 2021-06-29 2023-01-05 Shape Therapeutics Inc. Engineered guide rnas and polynucleotides
EP4388089A4 (en) * 2021-08-18 2025-01-15 Peking University Engineered adar-recruiting rnas and methods of use thereof
TW202328449A (zh) 2021-09-24 2023-07-16 美商艾拉倫製藥公司 微管相關蛋白ＴＡＵ（ＭＡＰＴ）ｉＲＮＡ試劑組合物及其使用方法
AU2022375788A1 (en) * 2021-10-27 2024-05-23 Shape Therapeutics Inc. Engineered rnas
EP4479535A1 (en) 2022-02-14 2024-12-25 ProQR Therapeutics II B.V. Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia
JP2025524566A (ja) 2022-07-15 2025-07-30 プロキューアール セラピューティクス ツー ベスローテン フェンノートシャップ Ａｄａｒ媒介ｒｎａ編集のためのオリゴヌクレオチドおよびその使用
US20260027237A1 (en) 2022-07-15 2026-01-29 Proqr Therapeutics Ii B.V. Chemically modified oligonucleotides for adar-mediated rna editing
GB202215614D0 (en) 2022-10-21 2022-12-07 Proqr Therapeutics Ii Bv Heteroduplex rna editing oligonucleotide complexes
WO2024110565A1 (en) 2022-11-24 2024-05-30 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of hereditary hfe-hemochromatosis
GB202218090D0 (en) 2022-12-01 2023-01-18 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of aldehyde dehydrogenase 2 deficiency
AU2023390632A1 (en) 2022-12-09 2025-07-10 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of cardiovascular disease
WO2024137991A2 (en) * 2022-12-23 2024-06-27 Shape Therapeutics Inc. Engineered guide rnas and polynucleotides
GB202300865D0 (en) 2023-01-20 2023-03-08 Proqr Therapeutics Ii Bv Delivery of oligonucleotides
WO2024166019A1 (en) * 2023-02-08 2024-08-15 Biorchestra Co., Ltd. Sirna targeting alpha-synuclein and uses thereof
WO2024175550A1 (en) 2023-02-20 2024-08-29 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of atherosclerotic cardiovascular disease
EP4689110A1 (en) 2023-03-24 2026-02-11 ProQR Therapeutics II B.V. Antisense oligonucleotides for the treatment of neurological disorders
GB202304363D0 (en) 2023-03-24 2023-05-10 Proqr Therapeutics Ii Bv Chemically modified antisense oligonucleotides for use in RNA editing
EP4688143A1 (en) 2023-03-27 2026-02-11 ProQR Therapeutics II B.V. Antisense oligonucleotides for the treatment of liver disease
TW202516003A (zh) 2023-06-16 2025-04-16 荷蘭商Ｐｒｏｑｒ治療上市公司Ｉｉ 用於治療神經退化性疾病之反義寡核苷酸
WO2025051946A1 (en) 2023-09-07 2025-03-13 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of metabolic disorders
WO2025104239A1 (en) 2023-11-16 2025-05-22 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of classic galactosemia
WO2025132708A1 (en) 2023-12-20 2025-06-26 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of huntington's disease
WO2025137637A2 (en) * 2023-12-21 2025-06-26 Shape Therapeutics Inc. Wobble base pairs for improving specificity of rna editing
GB202404661D0 (en) 2024-04-02 2024-05-15 Proqr Therapeutics Ii Bv Antisense oligoncleotides for the treatment of liver disease
GB202405143D0 (en) 2024-04-11 2024-05-29 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of poly-q disease
WO2025224230A1 (en) 2024-04-25 2025-10-30 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of fatty liver disease
GB202410081D0 (en) 2024-07-11 2024-08-28 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of cardiovascular disease
WO2026022136A1 (en) 2024-07-23 2026-01-29 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of metabolic disorders
WO2026060374A2 (en) 2024-09-16 2026-03-19 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of neurological disorders
WO2026068781A1 (en) 2024-09-30 2026-04-02 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of liver disease

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7456269B2 (en) * 1998-09-24 2008-11-25 Pharmacia & Upjohn Company Alzheimer's disease secretase, APP substrates thereof, and uses thereof
CN108884460A (zh) * 2016-03-19 2018-11-23 F1肿瘤医学公司 淋巴细胞转导及其扩增调节的方法与组合物
US20190040383A1 (en) * 2014-12-17 2019-02-07 Proqr Therapeutics Ii B.V. Targeted rna editing

Family Cites Families (109)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5759833A (en) 1994-05-27 1998-06-02 Cubist Pharmaceuticals, Inc. Human isoleucyl-tRNA synthetase proteins, nucleic acids and tester strains comprising same
JP2002508959A (ja) 1998-01-14 2002-03-26 ヒューマン ジーン セラピー リサーチ インスティテュート ヒトのサプレッサーｔＲＮＡオリゴヌクレオチド、及びそれを用いる方法
ATE318923T1 (de) 2000-06-01 2006-03-15 Univ North Carolina Doppelsträngige parvovirus-vektoren
JP5642916B2 (ja) 2003-04-17 2014-12-17 ザ スクリプス リサーチ インスティテュート 真核遺伝コードの拡張
EP2410331B1 (en) 2003-06-18 2015-09-23 The Scripps Research Institute Aminoacyl-tRNA synthetase for aminoacylation tRNA with unnatural amino acids
WO2005094370A2 (en) 2004-03-29 2005-10-13 The General Hospital Corporation Oligonucleotide complex compositions and methods of use as gene alteration tools
JP5697297B2 (ja) 2004-05-14 2015-04-08 ロゼッタ ジノミクス リミテッド マイクロｎａｓおよびその使用
SI2049664T1 (sl) 2006-08-11 2012-04-30 Prosensa Technologies Bv Enojna veriga oligonukleotidov, komplementarna repetitivnim elementom, za zdravljenje genetskih bolezni, povezanih z nestabilnostjo dnk ponovitev
EP2489732A1 (en) 2007-05-31 2012-08-22 Novozymes Inc. Compositions for degrading cellulosic material
WO2012161144A1 (ja) 2011-05-20 2012-11-29 学校法人 福岡大学 Rna配列上の修飾を識別するリボザイムおよびそれを用いたrna開裂方法
EP3730618A1 (en) 2011-11-18 2020-10-28 Alnylam Pharmaceuticals, Inc. Rnai agents, compositions and methods of use thereof for treating transthyretin (ttr) associated diseases
LT2841578T (lt) 2012-04-23 2017-09-25 Biomarin Technologies B.V. Rnr moduliuojantys oligonukleotidai su pagerintomis savybėmis, skirti neuromuskulinių susirgimų gydymui
RS54944B1 (sr) 2012-07-12 2016-11-30 Proqr Therapeutics Ii Bv Oligonukleotidi za pravljenje promena u sekvencama ciljanih rnk molekula prisutnih u živoj ćeliji
US9650627B1 (en) 2012-07-19 2017-05-16 University Of Puerto Rico Site-directed RNA editing
CN113355357B (zh) 2012-12-12 2024-12-03 布罗德研究所有限公司 对用于序列操纵的改进的系统、方法和酶组合物进行的工程化和优化
MX374532B (es) 2013-06-17 2025-03-06 Broad Inst Inc Suministro, uso y aplicaciones terapéuticas de los sistemas y composiciones crispr-cas, para actuar sobre trastornos y enfermedades utilizando componentes víricos.
HRP20200250T1 (hr) 2013-08-08 2020-05-29 The Scripps Research Institute Metoda site-specifično enzimsko obilježavanje nukleinskih kiselina in vitro ugradnjom neprirodnih nukleotida
EP3041958B1 (en) 2013-09-04 2019-12-04 Cold Spring Harbor Laboratory Reducing nonsense-mediated mrna decay
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
WO2015120197A1 (en) 2014-02-05 2015-08-13 Regents Of The University Of California Rna editing biomarkers for diagnosis, pharmacological screening and prognostication in cancer
TWI638047B (zh) 2014-04-09 2018-10-11 史基普研究協會 藉由核酸三磷酸酯轉運子將非天然或經修飾的核苷三磷酸酯輸入至細胞中
EP3140403A4 (en) * 2014-05-09 2017-12-20 Université Laval Prevention and treatment of alzheimer's disease by genome editing using the crispr/cas system
CA2957661A1 (en) 2014-08-09 2016-02-18 Kevin FLANIGAN Methods and materials for activating an internal ribosome entry site in exon 5 of the dmd gene
US12359197B2 (en) * 2014-12-12 2025-07-15 Etagen Pharma, Inc. Compositions and methods for editing nucleic acids in cells utilizing oligonucleotides
WO2016106236A1 (en) 2014-12-23 2016-06-30 The Broad Institute Inc. Rna-targeting system
GB201503408D0 (en) 2015-02-27 2015-04-15 Proqr Therapeutics N V Oligonucleotides
GB201504124D0 (en) 2015-03-11 2015-04-22 Proqr Therapeutics B V Oligonucleotides
EP3288594B1 (en) 2015-04-27 2022-06-29 The Trustees of The University of Pennsylvania Dual aav vector system for crispr/cas9 mediated correction of human disease
EP3323890A4 (en) 2015-07-14 2019-01-30 Fukuoka University METHOD OF INDUSTRYING SITE-SPECIFIC RNA MUTATIONS, TARGETED EDITING GUIDE RNA USED IN THE METHOD AND TARGET RNA TARGET EDITING GUIDE RNA COMPLEX
JP7044373B2 (ja) 2015-07-15 2022-03-30 ラトガース，ザ ステート ユニバーシティ オブ ニュージャージー ヌクレアーゼ非依存的な標的化遺伝子編集プラットフォームおよびその用途
IL320434A (en) 2015-07-22 2025-06-01 Wave Life Sciences Ltd Oligonucleotide preparations and methods
WO2017040477A1 (en) 2015-08-31 2017-03-09 The University Of Chicago Composition and methods for detecting adenosine modifications
DE102015012522B3 (de) 2015-09-26 2016-06-02 Eberhard Karls Universität Tübingen Verfahren und Substanzen zur gerichteten RNA-Editierung
EP3359667A1 (en) 2015-10-05 2018-08-15 ProQR Therapeutics II B.V. Use of single-stranded antisense oligonucleotide in prevention or treatment of genetic diseases involving a trinucleotide repeat expansion
IL295755A (en) 2015-10-09 2022-10-01 Wave Life Sciences Ltd Oligonucleotide compositions and methods thereof
AU2016337034A1 (en) 2015-10-16 2018-05-10 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for use in treating Alzheimer's disease
US10370667B2 (en) 2015-11-18 2019-08-06 Rosalind Franklin University Of Medicine And Science Antisense compounds targeting leucine-rich repeat kinase 2 (LRRK2) for the treatment of parkinsons disease
WO2017106767A1 (en) 2015-12-18 2017-06-22 The Scripps Research Institute Production of unnatural nucleotides using a crispr/cas9 system
PL3458074T3 (pl) 2016-05-16 2024-11-12 Board Of Regents Of The University Of Texas System KOMPOZYCJA DO DOSTARCZANIA tRNA W POSTACI NANOCZĄSTEK I SPOSOBY ICH STOSOWANIA
CN109642231A (zh) 2016-06-17 2019-04-16 博德研究所 Vi型crispr直向同源物和系统
WO2017220751A1 (en) 2016-06-22 2017-12-28 Proqr Therapeutics Ii B.V. Single-stranded rna-editing oligonucleotides
WO2018017144A1 (en) 2016-07-19 2018-01-25 Brandeis University Compositions and methods for identifying rna binding polypeptide targets
CN110214183A (zh) 2016-08-03 2019-09-06 哈佛大学的校长及成员们 腺苷核碱基编辑器及其用途
CN110352244B (zh) 2016-09-01 2023-03-21 ProQR治疗上市公司Ⅱ 化学修饰的编辑rna的单链寡核苷酸
WO2018045336A1 (en) 2016-09-02 2018-03-08 PFFA Acquisition LLC Database and system architecture for analyzing multiparty interactions
GB201616202D0 (en) 2016-09-23 2016-11-09 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of eye deisease
EP3565608A4 (en) 2017-01-05 2020-12-16 Rutgers, The State University of New Jersey TARGETED GENE EDITING PLATFORM INDEPENDENT OF BICATENARY DNA BREAKAGE AND ITS USES
EP3571300A1 (en) 2017-01-19 2019-11-27 ProQR Therapeutics II B.V. Oligonucleotide complexes for use in rna editing
CN110612353A (zh) 2017-03-03 2019-12-24 加利福尼亚大学董事会 经由抑制性tRNAs和脱氨酶对突变进行RNA靶向
GB201706009D0 (en) 2017-04-13 2017-05-31 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of stargardt disease
WO2018208998A1 (en) 2017-05-10 2018-11-15 The Regents Of The University Of California Directed editing of cellular rna via nuclear delivery of crispr/cas9
US11866697B2 (en) 2017-05-18 2024-01-09 The Broad Institute, Inc. Systems, methods, and compositions for targeted nucleic acid editing
WO2018237372A1 (en) 2017-06-23 2018-12-27 Cornell University RNA MOLECULES, METHODS FOR PRODUCING CIRCULAR RNA, AND METHODS OF TREATMENT
KR102761791B1 (ko) 2017-06-26 2025-02-05 더 브로드 인스티튜트, 인코퍼레이티드 표적화된 핵산 편집을 위한 crispr/cas-아데닌 데아미나아제 기반 조성물, 시스템 및 방법
WO2019014262A1 (en) 2017-07-11 2019-01-17 The Scripps Research Institute INCORPORATION OF NON-NATURAL NUCLEOTIDES AND METHODS OF IN VIVO USE THEREOF
JP7325341B2 (ja) 2017-07-11 2023-08-14 シンソークス，インク． 非天然ヌクレオチドの組み込み及びその方法
CN111801345A (zh) 2017-07-28 2020-10-20 哈佛大学的校长及成员们 使用噬菌体辅助连续进化(pace)的进化碱基编辑器的方法和组合物
US10476825B2 (en) 2017-08-22 2019-11-12 Salk Institue for Biological Studies RNA targeting methods and compositions
GB201714027D0 (en) 2017-09-01 2017-10-18 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of huntington's disease
WO2019071048A1 (en) * 2017-10-04 2019-04-11 The Broad Institute, Inc. SYSTEMS, METHODS AND COMPOSITIONS FOR TARGETED NUCLEIC ACID EDITION
WO2019071274A1 (en) 2017-10-06 2019-04-11 Oregon Health & Science University Compositions and methods for editing rna
WO2019084062A1 (en) 2017-10-23 2019-05-02 The Broad Institute, Inc. SYSTEMS, METHODS AND COMPOSITIONS FOR TARGETED NUCLEIC ACID EDITION
JP7385282B2 (ja) 2017-11-07 2023-11-22 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル 環状ｒｎａ分子のための方法及び組成物
WO2019104094A2 (en) 2017-11-21 2019-05-31 The Regents Of The University Of California Fusion proteins and methods for site-directed genome editing
JP7333508B2 (ja) 2017-12-06 2023-08-25 学校法人福岡大学 オリゴヌクレオチド、その製造方法及び標的ｒｎａの部位特異的編集方法
WO2019158475A1 (en) * 2018-02-14 2019-08-22 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for rna editing
GB201803010D0 (en) 2018-02-26 2018-04-11 Royal Holloway & Bedford New College Neurodegenerative disorders
KR20250133482A (ko) 2018-05-11 2025-09-05 빔 테라퓨틱스, 인크. 프로그래밍가능한 염기 편집기 시스템을 이용하여 단일 뉴클레오티드 다형성을 편집하는 방법
GB201808146D0 (en) 2018-05-18 2018-07-11 Proqr Therapeutics Ii Bv Stereospecific Linkages in RNA Editing Oligonucleotides
JP7347830B2 (ja) 2018-06-29 2023-09-20 エバーハルト カール ウニヴェルジテート テュービンゲン Ｒｎａ編集のための人工核酸
WO2020018918A1 (en) 2018-07-19 2020-01-23 The Board Of Trustees Of The University Of Illinois Methods for exon skipping and gene knockout using base editors
WO2020023655A1 (en) 2018-07-24 2020-01-30 Flagship Pioneering, Inc. Compositions comprising circular polyribonucleotides and uses thereof
US20220010333A1 (en) 2018-09-06 2022-01-13 The Regents Of The University Of California Rna and dna base editing via engineered adar recruitment
EP3864152B1 (en) 2018-10-12 2023-08-16 Peking University Methods and compositions for editing rnas
JP2022513159A (ja) 2018-11-29 2022-02-07 フラッグシップ パイオニアリング イノベーションズ ブイ， インコーポレイテッド Ｒｎａを調節する方法
JP2022513719A (ja) 2018-12-06 2022-02-09 ウェイブ ライフ サイエンシズ リミテッド オリゴヌクレオチド組成物及びその方法
BR102019009665A2 (pt) 2018-12-21 2022-02-08 Jacques P. Tremblay Modificação da proteína precursora beta amiloide (app) através da edição de base usando o sistema crispr/cas9
EP3906308A1 (en) 2018-12-31 2021-11-10 Research Institute at Nationwide Children's Hospital Dux4 rna silencing using rna targeting crispr-cas13b
EP3914260A4 (en) 2019-01-22 2023-05-17 Korro Bio, Inc. RNA-EDITING OLIGONUCLEOTIDES AND THEIR USE
US20200385713A1 (en) 2019-01-22 2020-12-10 Korro Bio, Inc. Rna-editing oligonucleotides and uses thereof
CA3127243A1 (en) 2019-01-22 2020-07-30 Korro Bio, Inc. Rna-editing oligonucleotides and uses thereof
JP2022518808A (ja) 2019-01-28 2022-03-16 プロキューアール セラピューティクス ツー ベスローテン フェンノートシャップ レーバー先天黒内障の処置のためのアンチセンスオリゴヌクレオチド
US12398393B2 (en) 2019-01-28 2025-08-26 Proqr Therapeutics Ii B.V. RNA-editing oligonucleotides for the treatment of usher syndrome
CN113423385A (zh) 2019-02-01 2021-09-21 波涛生命科学有限公司 寡核苷酸组合物及其方法
GB201901873D0 (en) 2019-02-11 2019-04-03 Proqr Therapeutics Ii Bv Antisense oligonucleotides for nucleic acid editing
US20220177894A1 (en) 2019-04-02 2022-06-09 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for immunotherapy
GB201904709D0 (en) 2019-04-03 2019-05-15 Proqr Therapeutics Ii Bv Chemically modified oligonucleotides
EP3956449B1 (en) 2019-04-15 2026-02-25 EdiGene Therapeutics (Beijing) Inc. Methods and compositions for editing rnas
CA3136172A1 (en) 2019-04-18 2020-10-22 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of usher syndrome
GB201905732D0 (en) 2019-04-24 2019-06-05 Proqr Therapeutics Ii Bv RNA editing inhibitors and methods of use
EP3981436A4 (en) 2019-06-05 2024-06-19 Fukuoka University Stable target-editing guide rna having chemically modified nucleic acid introduced thereinto
AU2020290517B2 (en) 2019-06-13 2026-02-26 Proqr Therapeutics Ii B.V. Antisense RNA editing oligonucleotides comprising cytidine analogs
WO2020254249A1 (en) 2019-06-21 2020-12-24 Proqr Therapeutics Ii B.V. Delivery of nucleic acids for the treatment of auditory disorders
EP3997229A4 (en) 2019-07-12 2024-07-03 Peking University TARGETED RNA EDITING BY HARNESSING ENDOGENOUS ADAR USING MODIFIED RNA
WO2021018750A1 (en) 2019-07-26 2021-02-04 Proqr Therapeutics Ii B.V. Ophthalmic compositions comprising viscosifying polymers and nucleic acids
EP4008784A1 (en) 2019-08-01 2022-06-08 Astellas Pharma Inc. Guide rna for targeted-editing with functional base sequence added thereto
JP7738323B2 (ja) 2019-09-27 2025-09-12 学校法人福岡大学 オリゴヌクレオチド及び標的ｒｎａの部位特異的編集方法
AU2020395113A1 (en) 2019-12-02 2022-06-09 Shape Therapeutics Inc. Therapeutic editing
US20230059753A1 (en) 2019-12-09 2023-02-23 Astellas Pharma Inc. Antisense guide rna with added functional region for editing target rna
CA3158528A1 (en) 2019-12-23 2021-07-01 Jim SWILDENS Antisense oligonucleotides for nucleotide deamination in the treatment of stargardt disease
KR20220119129A (ko) 2019-12-30 2022-08-26 에디진 테라퓨틱스 (베이징) 인크. Leaper 기술에 기반한 mps ih 치료 방법 및 조성물
BR112022012921A2 (pt) 2019-12-30 2022-09-06 Edigene Therapeutics Beijing Inc Métodos para editar de forma alvejada ácido ribonucleico e para tratar a síndrome de usher tipo ii, ácido ribonucleico de recrutamento da adenosina desaminase, construto ou vetor de entrega, célula, e, uso do ácido ribonucleico de recrutamento da adenosina desaminase ou do construto ou vetor de entrega
WO2021136520A1 (zh) 2019-12-31 2021-07-08 博雅辑因（北京）生物科技有限公司 一种靶向编辑rna的新方法
CA3166720A1 (en) 2020-03-04 2021-09-10 Jim SWILDENS Antisense oligonucleotides for use in the treatment of usher syndrome
AU2021257508A1 (en) 2020-04-15 2022-11-17 Edigene Therapeutics (Beijing) Inc. Method and drug for treating Hurler syndrome
CN115777020A (zh) 2020-04-22 2023-03-10 塑造治疗公司 使用snrna组分的组合物和方法
JP2023528039A (ja) 2020-05-26 2023-07-03 シェイプ セラピューティクス インコーポレイテッド 改変環状ポリヌクレオチド
KR102606949B1 (ko) 2020-06-26 2023-11-29 애터미주식회사 스마트 칫솔 및 스마트 살균기를 포함하는 구강 관리 시스템
EP4256050A4 (en) 2020-12-01 2025-01-22 Shape Therapeutics Inc. High throughput rna-editing screening methods

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US7456269B2 (en) * 1998-09-24 2008-11-25 Pharmacia & Upjohn Company Alzheimer's disease secretase, APP substrates thereof, and uses thereof
US20190040383A1 (en) * 2014-12-17 2019-02-07 Proqr Therapeutics Ii B.V. Targeted rna editing
CN108884460A (zh) * 2016-03-19 2018-11-23 F1肿瘤医学公司 淋巴细胞转导及其扩增调节的方法与组合物

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
BENCE GYÖRGY等: ""CRISPR/Cas9 Mediated Disruption of the Swedish APP Allele as a Therapeutic Approach for Early-Onset Alzheimer\'s Disease"", 《MOL THER NUCLEIC ACIDS》, 16 March 2018 (2018-03-16), pages 429 - 440 *
KATRINA A.LEHMANN等: ""The importance of internal loops within RNA substrates of ADAR1"", 《J. MOL. BIOL.》, 6 September 1999 (1999-09-06), pages 1 - 13 *
SUDHANSHU P RAIKWAR等: ""Next Generation Precision Medicine: CRISPR-mediated Genome Editing for the Treatment of Neurodegenerative Disorders"", 《J NEUROIMMUNE PHARMACOL》, 23 April 2019 (2019-04-23), pages 608 - 641, XP037057993, DOI: 10.1007/s11481-019-09849-y *

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