JP2019536782A5 - - Google Patents
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- JP2019536782A5 JP2019536782A5 JP2019528478A JP2019528478A JP2019536782A5 JP 2019536782 A5 JP2019536782 A5 JP 2019536782A5 JP 2019528478 A JP2019528478 A JP 2019528478A JP 2019528478 A JP2019528478 A JP 2019528478A JP 2019536782 A5 JP2019536782 A5 JP 2019536782A5
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- 239000000203 mixture Substances 0.000 claims description 102
- 238000000034 method Methods 0.000 claims description 42
- 239000013598 vector Substances 0.000 claims description 37
- 108090000765 processed proteins & peptides Proteins 0.000 claims description 27
- 229920001184 polypeptide Polymers 0.000 claims description 24
- 102000004196 processed proteins & peptides Human genes 0.000 claims description 24
- 102100024108 Dystrophin Human genes 0.000 claims description 17
- 201000006938 muscular dystrophy Diseases 0.000 claims description 17
- 108010069091 Dystrophin Proteins 0.000 claims description 16
- 108020005004 Guide RNA Proteins 0.000 claims description 16
- 210000004027 cell Anatomy 0.000 claims description 16
- 102000001039 Dystrophin Human genes 0.000 claims description 15
- 210000000663 muscle cell Anatomy 0.000 claims description 11
- 239000013603 viral vector Substances 0.000 claims description 10
- 239000013607 AAV vector Substances 0.000 claims description 6
- 239000011734 sodium Substances 0.000 claims description 6
- 108091028043 Nucleic acid sequence Proteins 0.000 claims description 5
- 108010051583 Ventricular Myosins Proteins 0.000 claims description 5
- 230000007547 defect Effects 0.000 claims description 4
- 230000010076 replication Effects 0.000 claims description 4
- 102000007469 Actins Human genes 0.000 claims description 3
- 108010085238 Actins Proteins 0.000 claims description 3
- 101710158332 Diuretic hormone Proteins 0.000 claims description 3
- 101710204261 Diuretic hormone class 2 Proteins 0.000 claims description 3
- DGAQECJNVWCQMB-PUAWFVPOSA-M Ilexoside XXIX Chemical compound C[C@@H]1CC[C@@]2(CC[C@@]3(C(=CC[C@H]4[C@]3(CC[C@@H]5[C@@]4(CC[C@@H](C5(C)C)OS(=O)(=O)[O-])C)C)[C@@H]2[C@]1(C)O)C)C(=O)O[C@H]6[C@@H]([C@H]([C@@H]([C@H](O6)CO)O)O)O.[Na+] DGAQECJNVWCQMB-PUAWFVPOSA-M 0.000 claims description 3
- 102100026925 Myosin regulatory light chain 2, ventricular/cardiac muscle isoform Human genes 0.000 claims description 3
- 101000987816 Triticum aestivum 16.9 kDa class I heat shock protein 1 Proteins 0.000 claims description 3
- 102000013640 alpha-Crystallin B Chain Human genes 0.000 claims description 3
- 108010051585 alpha-Crystallin B Chain Proteins 0.000 claims description 3
- 230000002490 cerebral effect Effects 0.000 claims description 3
- 210000005260 human cell Anatomy 0.000 claims description 3
- 230000001939 inductive effect Effects 0.000 claims description 3
- 102000006495 integrins Human genes 0.000 claims description 3
- 108010044426 integrins Proteins 0.000 claims description 3
- 239000002502 liposome Substances 0.000 claims description 3
- 108010065781 myosin light chain 2 Proteins 0.000 claims description 3
- 239000002105 nanoparticle Substances 0.000 claims description 3
- 239000013612 plasmid Substances 0.000 claims description 3
- 229910052708 sodium Inorganic materials 0.000 claims description 3
- 241000604451 Acidaminococcus Species 0.000 claims description 2
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims description 2
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims description 2
- 241000202702 Adeno-associated virus - 3 Species 0.000 claims description 2
- 241000580270 Adeno-associated virus - 4 Species 0.000 claims description 2
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims description 2
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims description 2
- 241001164823 Adeno-associated virus - 7 Species 0.000 claims description 2
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims description 2
- 241000649045 Adeno-associated virus 10 Species 0.000 claims description 2
- 241000649046 Adeno-associated virus 11 Species 0.000 claims description 2
- 241000702421 Dependoparvovirus Species 0.000 claims description 2
- 108700024394 Exon Proteins 0.000 claims description 2
- 241001112693 Lachnospiraceae Species 0.000 claims description 2
- 239000003937 drug carrier Substances 0.000 claims description 2
- 125000003729 nucleotide group Chemical group 0.000 claims description 2
- 238000003776 cleavage reaction Methods 0.000 claims 2
- 238000012224 gene deletion Methods 0.000 claims 1
- 208000024891 symptom Diseases 0.000 description 10
- 210000003205 muscle Anatomy 0.000 description 9
- 239000003550 marker Substances 0.000 description 6
- 210000001087 myotubule Anatomy 0.000 description 6
- 210000001519 tissue Anatomy 0.000 description 4
- 230000000926 neurological effect Effects 0.000 description 3
- 230000000750 progressive effect Effects 0.000 description 3
- 208000010428 Muscle Weakness Diseases 0.000 description 2
- 206010028372 Muscular weakness Diseases 0.000 description 2
- 206010033799 Paralysis Diseases 0.000 description 2
- 230000000735 allogeneic effect Effects 0.000 description 2
- 101150015424 dmd gene Proteins 0.000 description 2
- 238000002347 injection Methods 0.000 description 2
- 239000007924 injection Substances 0.000 description 2
- 210000002966 serum Anatomy 0.000 description 2
- 201000006347 Intellectual Disability Diseases 0.000 description 1
- 208000034819 Mobility Limitation Diseases 0.000 description 1
- 108091034117 Oligonucleotide Proteins 0.000 description 1
- 108020005067 RNA Splice Sites Proteins 0.000 description 1
- 208000000875 Spinal Curvatures Diseases 0.000 description 1
- 230000002159 abnormal effect Effects 0.000 description 1
- 230000014461 bone development Effects 0.000 description 1
- 230000009194 climbing Effects 0.000 description 1
- 230000007812 deficiency Effects 0.000 description 1
- 238000003745 diagnosis Methods 0.000 description 1
- 230000001747 exhibiting effect Effects 0.000 description 1
- 230000002068 genetic effect Effects 0.000 description 1
- 210000005003 heart tissue Anatomy 0.000 description 1
- 238000000338 in vitro Methods 0.000 description 1
- 238000001727 in vivo Methods 0.000 description 1
- 238000011065 in-situ storage Methods 0.000 description 1
- 238000001802 infusion Methods 0.000 description 1
- 238000010255 intramuscular injection Methods 0.000 description 1
- 239000007927 intramuscular injection Substances 0.000 description 1
- 238000001990 intravenous administration Methods 0.000 description 1
- 230000007775 late Effects 0.000 description 1
- 210000004962 mammalian cell Anatomy 0.000 description 1
- 108020004999 messenger RNA Proteins 0.000 description 1
- 238000012986 modification Methods 0.000 description 1
- 230000004048 modification Effects 0.000 description 1
- 230000035772 mutation Effects 0.000 description 1
- 210000004197 pelvis Anatomy 0.000 description 1
- 239000002243 precursor Substances 0.000 description 1
- 210000003314 quadriceps muscle Anatomy 0.000 description 1
- 210000002027 skeletal muscle Anatomy 0.000 description 1
- 210000001057 smooth muscle myoblast Anatomy 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662426853P | 2016-11-28 | 2016-11-28 | |
| US62/426,853 | 2016-11-28 | ||
| PCT/US2017/063468 WO2018098480A1 (en) | 2016-11-28 | 2017-11-28 | Prevention of muscular dystrophy by crispr/cpf1-mediated gene editing |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2019536782A JP2019536782A (ja) | 2019-12-19 |
| JP2019536782A5 true JP2019536782A5 (https=) | 2021-01-14 |
Family
ID=60703123
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019528478A Pending JP2019536782A (ja) | 2016-11-28 | 2017-11-28 | ＣＲＩＳＰＲ／Ｃｐｆ１媒介性遺伝子編集による筋ジストロフィーの予防 |
Country Status (8)
| Country | Link |
|---|---|
| US (1) | US20200046854A1 (https=) |
| EP (1) | EP3545090A1 (https=) |
| JP (1) | JP2019536782A (https=) |
| CN (1) | CN110382695A (https=) |
| AU (1) | AU2017364106A1 (https=) |
| CA (1) | CA3044531A1 (https=) |
| MX (1) | MX2019006157A (https=) |
| WO (1) | WO2018098480A1 (https=) |
Families Citing this family (67)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US10323236B2 (en) | 2011-07-22 | 2019-06-18 | President And Fellows Of Harvard College | Evaluation and improvement of nuclease cleavage specificity |
| WO2013163628A2 (en) | 2012-04-27 | 2013-10-31 | Duke University | Genetic correction of mutated genes |
| US9828582B2 (en) | 2013-03-19 | 2017-11-28 | Duke University | Compositions and methods for the induction and tuning of gene expression |
| US9163284B2 (en) | 2013-08-09 | 2015-10-20 | President And Fellows Of Harvard College | Methods for identifying a target site of a Cas9 nuclease |
| US9359599B2 (en) | 2013-08-22 | 2016-06-07 | President And Fellows Of Harvard College | Engineered transcription activator-like effector (TALE) domains and uses thereof |
| US9526784B2 (en) | 2013-09-06 | 2016-12-27 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
| US9228207B2 (en) | 2013-09-06 | 2016-01-05 | President And Fellows Of Harvard College | Switchable gRNAs comprising aptamers |
| US9322037B2 (en) | 2013-09-06 | 2016-04-26 | President And Fellows Of Harvard College | Cas9-FokI fusion proteins and uses thereof |
| US20150165054A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting caspase-9 point mutations |
| US10077453B2 (en) | 2014-07-30 | 2018-09-18 | President And Fellows Of Harvard College | CAS9 proteins including ligand-dependent inteins |
| WO2016130600A2 (en) | 2015-02-09 | 2016-08-18 | Duke University | Compositions and methods for epigenome editing |
| EP4089175A1 (en) | 2015-10-13 | 2022-11-16 | Duke University | Genome engineering with type i crispr systems in eukaryotic cells |
| IL310721B2 (en) | 2015-10-23 | 2025-11-01 | Harvard College | Nucleobase editors and uses thereof |
| EP4036228A1 (en) | 2015-11-13 | 2022-08-03 | Avellino Lab USA, Inc. | Methods for the treatment of corneal dystrophies |
| KR102787119B1 (ko) | 2015-11-30 | 2025-03-27 | 듀크 유니버시티 | 유전자 편집에 의한 인간 디스트로핀 유전자의 교정을 위한 치료용 표적 및 사용 방법 |
| US20190127713A1 (en) | 2016-04-13 | 2019-05-02 | Duke University | Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use |
| JP7490211B2 (ja) * | 2016-07-19 | 2024-05-27 | デューク ユニバーシティ | Ｃｐｆ１に基づくゲノム編集の治療適用 |
| 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 |
| WO2018031683A1 (en) | 2016-08-09 | 2018-02-15 | President And Fellows Of Harvard College | Programmable cas9-recombinase fusion proteins and uses thereof |
| WO2020225754A1 (en) * | 2019-05-06 | 2020-11-12 | Mcmullen Tara | Crispr gene editing for autosomal dominant diseases |
| US11542509B2 (en) | 2016-08-24 | 2023-01-03 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
| KR102622411B1 (ko) | 2016-10-14 | 2024-01-10 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 핵염기 에디터의 aav 전달 |
| WO2018119359A1 (en) | 2016-12-23 | 2018-06-28 | President And Fellows Of Harvard College | Editing of ccr5 receptor gene to protect against hiv infection |
| US12390514B2 (en) | 2017-03-09 | 2025-08-19 | President And Fellows Of Harvard College | Cancer vaccine |
| EP3592853A1 (en) | 2017-03-09 | 2020-01-15 | President and Fellows of Harvard College | Suppression of pain by gene editing |
| US11542496B2 (en) | 2017-03-10 | 2023-01-03 | President And Fellows Of Harvard College | Cytosine to guanine base editor |
| KR20240116572A (ko) | 2017-03-23 | 2024-07-29 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제 |
| EP3617311B1 (en) * | 2017-03-30 | 2024-11-20 | Kyoto University | Method for inducing exon skipping by genome editing |
| US11560566B2 (en) | 2017-05-12 | 2023-01-24 | President And Fellows Of Harvard College | Aptazyme-embedded guide RNAs for use with CRISPR-Cas9 in genome editing and transcriptional activation |
| 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 |
| EP3676376B1 (en) | 2017-08-30 | 2025-01-15 | President and Fellows of Harvard College | High efficiency base editors comprising gam |
| KR20250107288A (ko) | 2017-10-16 | 2025-07-11 | 더 브로드 인스티튜트, 인코퍼레이티드 | 아데노신 염기 편집제의 용도 |
| US12406749B2 (en) | 2017-12-15 | 2025-09-02 | The Broad Institute, Inc. | Systems and methods for predicting repair outcomes in genetic engineering |
| EP3740580A4 (en) | 2018-01-19 | 2021-10-20 | Duke University | GENOME ENGINEERING WITH CRISPR-CAS SYSTEMS IN EUKARYONTS |
| US12157760B2 (en) | 2018-05-23 | 2024-12-03 | The Broad Institute, Inc. | Base editors and uses thereof |
| US12522807B2 (en) | 2018-07-09 | 2026-01-13 | The Broad Institute, Inc. | RNA programmable epigenetic RNA modifiers and uses thereof |
| CA3108282A1 (en) | 2018-08-02 | 2020-02-06 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| US12018087B2 (en) | 2018-08-02 | 2024-06-25 | Dyne Therapeutics, Inc. | Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject |
| SG11202100928QA (en) | 2018-08-02 | 2021-02-25 | Dyne Therapeutics Inc | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| US11168141B2 (en) | 2018-08-02 | 2021-11-09 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| WO2020092453A1 (en) | 2018-10-29 | 2020-05-07 | The Broad Institute, Inc. | Nucleobase editors comprising geocas9 and uses thereof |
| WO2020122104A1 (ja) | 2018-12-11 | 2020-06-18 | 国立大学法人京都大学 | ゲノムｄｎａに欠失を誘導する方法 |
| US12351837B2 (en) | 2019-01-23 | 2025-07-08 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| MX2021009750A (es) * | 2019-02-12 | 2021-09-08 | Univ Degli Studi Di Trento | Moleculas del acido ribonucleico guia de cas12a y usos de las mismas. |
| WO2020191233A1 (en) | 2019-03-19 | 2020-09-24 | The Broad Institute, Inc. | Methods and compositions for editing nucleotide sequences |
| JP2022526669A (ja) * | 2019-04-12 | 2022-05-25 | デューク ユニバーシティ | ジストロフィン機能を修復するためのＣＲＩＳＰＲ／Ｃａｓをベースにした塩基編集組成物 |
| US12473543B2 (en) | 2019-04-17 | 2025-11-18 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
| CN110499333A (zh) * | 2019-08-01 | 2019-11-26 | 广州德赫生物科技有限公司 | 用于修复dmd基因突变的核酸序列及系统 |
| US12435330B2 (en) | 2019-10-10 | 2025-10-07 | The Broad Institute, Inc. | Methods and compositions for prime editing RNA |
| CN115279184A (zh) * | 2020-03-04 | 2022-11-01 | 雷杰纳荣制药公司 | B4galt1介导的功能的啮齿动物模型 |
| WO2021216674A1 (en) * | 2020-04-24 | 2021-10-28 | University Of Massachusetts | Improved cas 12a/nls mediated therapeutic gene editing platforms |
| IL297761A (en) | 2020-05-08 | 2022-12-01 | Broad Inst Inc | Methods and compositions for simultaneously editing two helices of a designated double-helix nucleotide sequence |
| EP4149622A4 (en) * | 2020-05-12 | 2024-12-18 | Myogene Bio LLC | IMMUNOSUPPRESSIVE AGENTS AND METHODS OF RE-DOSING VIRAL ADMINISTRATION FOR GENE THERAPY |
| US20220096606A1 (en) * | 2020-09-09 | 2022-03-31 | Vertex Pharmaceuticals Incorporated | Compositions and Methods for Treatment of Duchenne Muscular Dystrophy |
| WO2022140340A1 (en) * | 2020-12-22 | 2022-06-30 | Vertex Pharmaceuticals Incorporated | Compositions comprising an rna guide targeting dmd and uses thereof |
| EP4347832A1 (en) * | 2021-05-25 | 2024-04-10 | The Board Of Regents Of The University Of Texas System | Correction of duchenne muscular dystrophy mutations with all-in-one adeno-associated virus-delivered single-cut crispr |
| US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
| US11969475B2 (en) | 2021-07-09 | 2024-04-30 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| JP2024527362A (ja) * | 2021-07-09 | 2024-07-24 | ダイン セラピューティクス，インコーポレーテッド | 筋標的化複合体およびジストロフィノパチーを処置するためのその使用 |
| JP2024525608A (ja) | 2021-07-09 | 2024-07-12 | ダイン セラピューティクス，インコーポレーテッド | ジストロフィン異常症を処置するための筋標的化複合体および製剤 |
| US11771776B2 (en) | 2021-07-09 | 2023-10-03 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| CN114214360A (zh) * | 2021-12-27 | 2022-03-22 | 西安英创生物技术有限公司 | 先天性肌无力小鼠模型、其构建方法及应用 |
| EP4215614A1 (en) | 2022-01-24 | 2023-07-26 | Dynacure | Combination therapy for dystrophin-related diseases |
| WO2023172927A1 (en) * | 2022-03-08 | 2023-09-14 | Vertex Pharmaceuticals Incorporated | Precise excisions of portions of exon 44, 50, and 53 for treatment of duchenne muscular dystrophy |
| IL317874A (en) | 2022-06-24 | 2025-02-01 | Tune Therapeutics Inc | Compounds, systems and methods for reducing low density lipoproteins through targeted gene suppression |
| CN120648680A (zh) * | 2024-03-15 | 2025-09-16 | 迦进生物医药(上海)有限公司 | 治疗杜兴型肌营养不良症和贝克型肌营养不良症的药物 |
| CN118086408A (zh) * | 2024-04-25 | 2024-05-28 | 华南理工大学 | 一种可实现靶向肌肉细胞基因编辑的仿病毒颗粒及其制备方法和应用 |
Family Cites Families (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| DK99653C (da) | 1959-06-24 | 1964-08-31 | Skaanska Aettikfabriken Ab | Pynteliste til brug som sammenfojningsliste, hjørneliste eller endeliste ved montering af beklædningsplader. |
| EP0273085A1 (en) | 1986-12-29 | 1988-07-06 | IntraCel Corporation | A method for internalizing nucleic acids into eukaryotic cells |
| BR122020021379B1 (pt) * | 2008-10-24 | 2021-05-11 | Sarepta Therapeutics, Inc. | oligômero morfolino fosforodiamidato, composição que compreende o mesmo e uso do dito oligômero para tratar distrofia muscular |
| AU2013266968B2 (en) | 2012-05-25 | 2017-06-29 | Emmanuelle CHARPENTIER | Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription |
| US20140329762A1 (en) * | 2013-03-15 | 2014-11-06 | Sarepta Therapeutics, Inc. | Compositions for treating muscular dystrophy |
| WO2014197748A2 (en) * | 2013-06-05 | 2014-12-11 | Duke University | Rna-guided gene editing and gene regulation |
| EP4659765A3 (en) * | 2014-03-12 | 2026-02-18 | Precision Biosciences, Inc. | Dystrophin gene exon deletion using engineered nucleases |
| CN106714845A (zh) * | 2014-08-11 | 2017-05-24 | 得克萨斯州大学系统董事会 | 通过crispr/cas9介导的基因编辑预防肌营养不良 |
| JP6744019B2 (ja) * | 2014-08-11 | 2020-08-19 | ザ ボード オブ トラスティーズ オブ ザ ユニヴァーシティー オブ イリノイ | 生物流体の表皮特性評価のためのデバイス及び関連する方法 |
| US9790490B2 (en) * | 2015-06-18 | 2017-10-17 | The Broad Institute Inc. | CRISPR enzymes and systems |
| WO2017035416A2 (en) * | 2015-08-25 | 2017-03-02 | Duke University | Compositions and methods of improving specificity in genomic engineering using rna-guided endonucleases |
| WO2017049407A1 (en) * | 2015-09-23 | 2017-03-30 | UNIVERSITé LAVAL | Modification of the dystrophin gene and uses thereof |
| AU2016344609B2 (en) * | 2015-10-28 | 2022-05-12 | Vertex Pharmaceuticals Incorporated | Materials and methods for treatment of duchenne muscular dystrophy |
| JP7490211B2 (ja) * | 2016-07-19 | 2024-05-27 | デューク ユニバーシティ | Ｃｐｆ１に基づくゲノム編集の治療適用 |
-
2017
- 2017-11-28 AU AU2017364106A patent/AU2017364106A1/en not_active Abandoned
- 2017-11-28 US US16/464,124 patent/US20200046854A1/en not_active Abandoned
- 2017-11-28 CA CA3044531A patent/CA3044531A1/en not_active Abandoned
- 2017-11-28 MX MX2019006157A patent/MX2019006157A/es unknown
- 2017-11-28 CN CN201780084906.8A patent/CN110382695A/zh active Pending
- 2017-11-28 EP EP17817498.3A patent/EP3545090A1/en not_active Withdrawn
- 2017-11-28 WO PCT/US2017/063468 patent/WO2018098480A1/en not_active Ceased
- 2017-11-28 JP JP2019528478A patent/JP2019536782A/ja active Pending
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