JP2020500541A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2020500541A5 JP2020500541A5 JP2019530778A JP2019530778A JP2020500541A5 JP 2020500541 A5 JP2020500541 A5 JP 2020500541A5 JP 2019530778 A JP2019530778 A JP 2019530778A JP 2019530778 A JP2019530778 A JP 2019530778A JP 2020500541 A5 JP2020500541 A5 JP 2020500541A5
- Authority
- JP
- Japan
- Prior art keywords
- sequence encoding
- promoter
- composition
- grna
- sequence
- Prior art date
- Legal status (The legal status 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 status listed.)
- Pending
Links
- 239000000203 mixture Substances 0.000 claims description 56
- 108091033409 CRISPR Proteins 0.000 claims description 29
- 108020005004 Guide RNA Proteins 0.000 claims description 29
- 239000013598 vector Substances 0.000 claims description 22
- 229920001184 polypeptide Polymers 0.000 claims description 17
- 102000004196 processed proteins & peptides Human genes 0.000 claims description 17
- 108090000765 processed proteins & peptides Proteins 0.000 claims description 17
- 239000013603 viral vector Substances 0.000 claims description 10
- 239000013607 AAV vector Substances 0.000 claims description 7
- 108091026890 Coding region Proteins 0.000 claims description 4
- 101001053945 Mus musculus Dystrophin Proteins 0.000 claims description 4
- 230000002950 deficient Effects 0.000 claims description 3
- 230000010076 replication Effects 0.000 claims description 3
- 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
- 241000191967 Staphylococcus aureus Species 0.000 claims description 2
- 239000003937 drug carrier Substances 0.000 claims description 2
- 239000002502 liposome Substances 0.000 claims description 2
- 239000002105 nanoparticle Substances 0.000 claims description 2
- 239000013612 plasmid Substances 0.000 claims description 2
- 108010069091 Dystrophin Proteins 0.000 description 20
- 241000699666 Mus <mouse, genus> Species 0.000 description 19
- 210000004027 cell Anatomy 0.000 description 18
- 238000000034 method Methods 0.000 description 17
- 241000699670 Mus sp. Species 0.000 description 13
- 108091005804 Peptidases Proteins 0.000 description 12
- 239000004365 Protease Substances 0.000 description 12
- 102100037486 Reverse transcriptase/ribonuclease H Human genes 0.000 description 12
- 108700008625 Reporter Genes Proteins 0.000 description 11
- 238000012217 deletion Methods 0.000 description 11
- 230000037430 deletion Effects 0.000 description 11
- 238000010354 CRISPR gene editing Methods 0.000 description 8
- 102100024108 Dystrophin Human genes 0.000 description 6
- 210000000287 oocyte Anatomy 0.000 description 6
- 210000000130 stem cell Anatomy 0.000 description 6
- 238000011144 upstream manufacturing Methods 0.000 description 6
- 108091028043 Nucleic acid sequence Proteins 0.000 description 4
- 238000004519 manufacturing process Methods 0.000 description 4
- 108020005345 3' Untranslated Regions Proteins 0.000 description 3
- 108020004705 Codon Proteins 0.000 description 3
- 108060001084 Luciferase Proteins 0.000 description 3
- 239000005089 Luciferase Substances 0.000 description 3
- 239000000126 substance Substances 0.000 description 3
- 102000004420 Creatine Kinase Human genes 0.000 description 2
- 108010042126 Creatine kinase Proteins 0.000 description 2
- 108700024394 Exon Proteins 0.000 description 2
- 108020004485 Nonsense Codon Proteins 0.000 description 2
- 230000000694 effects Effects 0.000 description 2
- 230000001939 inductive effect Effects 0.000 description 2
- 239000011159 matrix material Substances 0.000 description 2
- 210000004165 myocardium Anatomy 0.000 description 2
- 210000002027 skeletal muscle Anatomy 0.000 description 2
- 238000013518 transcription Methods 0.000 description 2
- 230000035897 transcription Effects 0.000 description 2
- 238000013519 translation Methods 0.000 description 2
- 108091032973 (ribonucleotides)n+m Proteins 0.000 description 1
- 102000040650 (ribonucleotides)n+m Human genes 0.000 description 1
- 238000011156 evaluation Methods 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
- 125000003729 nucleotide group Chemical group 0.000 description 1
- 238000012216 screening Methods 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662431699P | 2016-12-08 | 2016-12-08 | |
| US62/431,699 | 2016-12-08 | ||
| PCT/US2017/065268 WO2018107003A1 (en) | 2016-12-08 | 2017-12-08 | Dmd reporter models containing humanized duschene muscular dystrophy mutations |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2020500541A JP2020500541A (ja) | 2020-01-16 |
| JP2020500541A5 true JP2020500541A5 (enExample) | 2021-01-21 |
Family
ID=60888655
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019530778A Pending JP2020500541A (ja) | 2016-12-08 | 2017-12-08 | ヒト化デュシェンヌ型筋ジストロフィー変異を有するｄｍｄレポーターモデル |
Country Status (6)
| Country | Link |
|---|---|
| US (1) | US20190364862A1 (enExample) |
| EP (1) | EP3551752A1 (enExample) |
| JP (1) | JP2020500541A (enExample) |
| AU (1) | AU2017370730A1 (enExample) |
| CA (1) | CA3046220A1 (enExample) |
| WO (1) | WO2018107003A1 (enExample) |
Families Citing this family (20)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US9828582B2 (en) | 2013-03-19 | 2017-11-28 | Duke University | Compositions and methods for the induction and tuning of gene expression |
| JP6929791B2 (ja) | 2015-02-09 | 2021-09-01 | デューク ユニバーシティ | エピゲノム編集のための組成物および方法 |
| 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 |
| EP4275747A3 (en) | 2016-07-19 | 2024-01-24 | Duke University | Therapeutic applications of cpf1-based genome editing |
| KR102758434B1 (ko) * | 2017-03-30 | 2025-01-21 | 고쿠리츠 다이가쿠 호진 교토 다이가쿠 | 게놈 편집에 의한 엑손 스키핑 유도 방법 |
| US20200260698A1 (en) * | 2017-08-18 | 2020-08-20 | The Board Of Regents Of The University Of Texas System | Exon deletion correction of duchenne muscular dystrophy mutations in the dystrophin actin binding domain 1 using crispr genome editing |
| WO2019136216A1 (en) * | 2018-01-05 | 2019-07-11 | The Board Of Regents Of The University Of Texas System | Therapeutic crispr/cas9 compositions and methods of use |
| US12509492B2 (en) | 2018-01-19 | 2025-12-30 | Duke University | Genome engineering with CRISPR-Cas systems in eukaryotes |
| WO2019246480A1 (en) * | 2018-06-21 | 2019-12-26 | The Board Of Regents Of The University Of Texas System | Correction of dystrophin exon 43, exon 45, or exon 52 deletions in duchenne muscular dystrophy |
| US11168141B2 (en) | 2018-08-02 | 2021-11-09 | 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 |
| WO2020028832A1 (en) | 2018-08-02 | 2020-02-06 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| AU2019316103B2 (en) | 2018-08-02 | 2026-02-19 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| EP3966327A1 (en) | 2019-05-08 | 2022-03-16 | Vertex Pharmaceuticals Incorporated | Crispr/cas all-in-two vector systems for treatment of dmd |
| 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 |
| US11969475B2 (en) | 2021-07-09 | 2024-04-30 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| US11771776B2 (en) | 2021-07-09 | 2023-10-03 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| US11638761B2 (en) | 2021-07-09 | 2023-05-02 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy |
| EP4493703A2 (en) * | 2022-03-18 | 2025-01-22 | University Of Florida Research Foundation, Incorporated | Methods and compositions for treating tmem43 related cardiomyopathy with a viral vector |
Family Cites Families (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4873191A (en) | 1981-06-12 | 1989-10-10 | Ohio University | Genetic transformation of zygotes |
| EP0273085A1 (en) | 1986-12-29 | 1988-07-06 | IntraCel Corporation | A method for internalizing nucleic acids into eukaryotic cells |
| CN108611349A (zh) * | 2011-12-28 | 2018-10-02 | 日本新药株式会社 | 反义核酸 |
| PL4289948T3 (pl) | 2012-05-25 | 2025-06-02 | The Regents Of The University Of California | Sposoby i kompozycje do modyfikacji kierowanego na rna docelowego dna i do nakierowanej na rna modulacji transkrypcji |
-
2017
- 2017-12-08 WO PCT/US2017/065268 patent/WO2018107003A1/en not_active Ceased
- 2017-12-08 CA CA3046220A patent/CA3046220A1/en not_active Abandoned
- 2017-12-08 AU AU2017370730A patent/AU2017370730A1/en not_active Abandoned
- 2017-12-08 US US16/467,445 patent/US20190364862A1/en not_active Abandoned
- 2017-12-08 JP JP2019530778A patent/JP2020500541A/ja active Pending
- 2017-12-08 EP EP17822886.2A patent/EP3551752A1/en not_active Withdrawn
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2020500541A5 (enExample) | ||
| US9896665B2 (en) | Proviral plasmids and production of recombinant adeno-associated virus | |
| JP2020519284A5 (enExample) | ||
| JP2018516978A5 (enExample) | ||
| JP2017509632A5 (enExample) | ||
| HRP20192141T1 (hr) | Genska terapija za retinitis pigmentosa | |
| IL259595B2 (en) | Scalable methods for producing recombinant adeno-associated viral (aav) vector in serum-free suspension cell culture system suitable for clinical use | |
| JP2024099770A5 (enExample) | ||
| JP2020519294A5 (enExample) | ||
| JPWO2020214613A5 (enExample) | ||
| CN118291541B (zh) | 一种诱导型启动子 | |
| CN120359235A (zh) | 用于治疗心脏病和与心脏功能障碍相关的疾病的具有降低的肝脏嗜性和增强的心脏细胞转导的重组腺相关病毒基因治疗载体 | |
| IL303882A (en) | A producer cell has low levels of VA-RNA | |
| KR102874560B1 (ko) | 아데노연관바이러스 변이체 | |
| JP7834758B2 (ja) | 低レベルのｖａ－ｒｎａを有する産生細胞 | |
| US12480098B1 (en) | Adeno-associated virus variant | |
| JP6473438B2 (ja) | プロモーター組成物 | |
| US20260022401A1 (en) | Novel AAV rep ORFs and Rep polypeptides | |
| WO2024223931A1 (en) | Manufacturing and use of recombinant self-complementary aav vectors | |
| AU2023446284A1 (en) | Manufacturing and use of recombinant self-complementary aav vectors | |
| JPWO2021195218A5 (enExample) | ||
| EP4701634A1 (en) | Riboswitches for regulating gene expression and therapeutic methods of using the same | |
| JP2016512028A5 (enExample) | ||
| JPWO2022060841A5 (enExample) | ||
| NZ738841A (en) | Modified factor ix, and compositions, methods and uses for gene transfer to cells, organs and tissues |