JPWO2020142479A5 - - Google Patents
Download PDFInfo
- Publication number
- JPWO2020142479A5 JPWO2020142479A5 JP2021538230A JP2021538230A JPWO2020142479A5 JP WO2020142479 A5 JPWO2020142479 A5 JP WO2020142479A5 JP 2021538230 A JP2021538230 A JP 2021538230A JP 2021538230 A JP2021538230 A JP 2021538230A JP WO2020142479 A5 JPWO2020142479 A5 JP WO2020142479A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- seq
- set forth
- nucleotide sequence
- composition
- 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
- 150000007523 nucleic acids Chemical class 0.000 claims description 87
- 108020004707 nucleic acids Proteins 0.000 claims description 83
- 102000039446 nucleic acids Human genes 0.000 claims description 83
- 239000002773 nucleotide Substances 0.000 claims description 68
- 125000003729 nucleotide group Chemical group 0.000 claims description 68
- 241000702421 Dependoparvovirus Species 0.000 claims description 54
- 102100021158 Double homeobox protein 4 Human genes 0.000 claims description 44
- 101000968549 Homo sapiens Double homeobox protein 4 Proteins 0.000 claims description 44
- 108020005004 Guide RNA Proteins 0.000 claims description 40
- 108090000623 proteins and genes Proteins 0.000 claims description 36
- 238000000034 method Methods 0.000 claims description 32
- 102000004169 proteins and genes Human genes 0.000 claims description 32
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims description 14
- 108010059343 MM Form Creatine Kinase Proteins 0.000 claims description 14
- 230000002401 inhibitory effect Effects 0.000 claims description 14
- 210000004027 cell Anatomy 0.000 claims description 11
- 238000010362 genome editing Methods 0.000 claims description 11
- 230000014509 gene expression Effects 0.000 claims description 10
- 241000700605 Viruses Species 0.000 claims description 9
- 201000006938 muscular dystrophy Diseases 0.000 claims description 8
- 102100036912 Desmin Human genes 0.000 claims description 4
- 108010044052 Desmin Proteins 0.000 claims description 4
- 208000037149 Facioscapulohumeral dystrophy Diseases 0.000 claims description 4
- 102000005604 Myosin Heavy Chains Human genes 0.000 claims description 4
- 108010084498 Myosin Heavy Chains Proteins 0.000 claims description 4
- 206010028980 Neoplasm Diseases 0.000 claims description 4
- 108091028043 Nucleic acid sequence Proteins 0.000 claims description 4
- 201000011510 cancer Diseases 0.000 claims description 4
- 210000005045 desmin Anatomy 0.000 claims description 4
- 208000008570 facioscapulohumeral muscular dystrophy Diseases 0.000 claims description 4
- 230000002452 interceptive effect Effects 0.000 claims description 4
- 230000001404 mediated effect Effects 0.000 claims description 4
- 201000006402 rhabdoid cancer Diseases 0.000 claims description 4
- 241000580270 Adeno-associated virus - 4 Species 0.000 claims description 2
- 206010005003 Bladder cancer Diseases 0.000 claims description 2
- 206010006187 Breast cancer Diseases 0.000 claims description 2
- 208000026310 Breast neoplasm Diseases 0.000 claims description 2
- 206010008342 Cervix carcinoma Diseases 0.000 claims description 2
- 101150054841 DUX4 gene Proteins 0.000 claims description 2
- 206010014733 Endometrial cancer Diseases 0.000 claims description 2
- 206010014759 Endometrial neoplasm Diseases 0.000 claims description 2
- 208000000461 Esophageal Neoplasms Diseases 0.000 claims description 2
- 208000008839 Kidney Neoplasms Diseases 0.000 claims description 2
- 206010058467 Lung neoplasm malignant Diseases 0.000 claims description 2
- -1 MHCK7 Proteins 0.000 claims description 2
- 206010027480 Metastatic malignant melanoma Diseases 0.000 claims description 2
- 206010030155 Oesophageal carcinoma Diseases 0.000 claims description 2
- 206010033128 Ovarian cancer Diseases 0.000 claims description 2
- 206010061535 Ovarian neoplasm Diseases 0.000 claims description 2
- 206010038389 Renal cancer Diseases 0.000 claims description 2
- 208000008938 Rhabdoid tumor Diseases 0.000 claims description 2
- 206010039491 Sarcoma Diseases 0.000 claims description 2
- 208000005718 Stomach Neoplasms Diseases 0.000 claims description 2
- 208000024313 Testicular Neoplasms Diseases 0.000 claims description 2
- 206010057644 Testis cancer Diseases 0.000 claims description 2
- 101150023087 UNC45B gene Proteins 0.000 claims description 2
- 208000007097 Urinary Bladder Neoplasms Diseases 0.000 claims description 2
- 208000006105 Uterine Cervical Neoplasms Diseases 0.000 claims description 2
- 201000010881 cervical cancer Diseases 0.000 claims description 2
- 230000002950 deficient Effects 0.000 claims description 2
- 239000003937 drug carrier Substances 0.000 claims description 2
- 201000004101 esophageal cancer Diseases 0.000 claims description 2
- 206010017758 gastric cancer Diseases 0.000 claims description 2
- 201000010982 kidney cancer Diseases 0.000 claims description 2
- 201000005202 lung cancer Diseases 0.000 claims description 2
- 208000020816 lung neoplasm Diseases 0.000 claims description 2
- 201000001441 melanoma Diseases 0.000 claims description 2
- 208000021039 metastatic melanoma Diseases 0.000 claims description 2
- 210000003205 muscle Anatomy 0.000 claims description 2
- 210000002027 skeletal muscle Anatomy 0.000 claims description 2
- 201000011549 stomach cancer Diseases 0.000 claims description 2
- 201000003120 testicular cancer Diseases 0.000 claims description 2
- 208000008732 thymoma Diseases 0.000 claims description 2
- 201000005112 urinary bladder cancer Diseases 0.000 claims description 2
- 238000012986 modification Methods 0.000 description 1
- 230000004048 modification Effects 0.000 description 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2024219757A JP2025032342A (ja) | 2018-12-31 | 2024-12-16 | Ｒｎａ標的化ｃｒｉｓｐｒ－ｃａｓ１３ｂを使用するｄｕｘ４ ｒｎａ発現停止 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862786670P | 2018-12-31 | 2018-12-31 | |
| US62/786,670 | 2018-12-31 | ||
| PCT/US2019/069048 WO2020142479A1 (en) | 2018-12-31 | 2019-12-31 | Dux4 rna silencing using rna targeting crispr-cas13b |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024219757A Division JP2025032342A (ja) | 2018-12-31 | 2024-12-16 | Ｒｎａ標的化ｃｒｉｓｐｒ－ｃａｓ１３ｂを使用するｄｕｘ４ ｒｎａ発現停止 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2022516515A JP2022516515A (ja) | 2022-02-28 |
| JP2022516515A5 JP2022516515A5 (https=) | 2023-01-05 |
| JPWO2020142479A5 true JPWO2020142479A5 (https=) | 2023-01-05 |
Family
ID=69376003
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021538230A Pending JP2022516515A (ja) | 2018-12-31 | 2019-12-31 | Ｒｎａ標的化ｃｒｉｓｐｒ－ｃａｓ１３ｂを使用するｄｕｘ４ ｒｎａ発現停止 |
| JP2024219757A Pending JP2025032342A (ja) | 2018-12-31 | 2024-12-16 | Ｒｎａ標的化ｃｒｉｓｐｒ－ｃａｓ１３ｂを使用するｄｕｘ４ ｒｎａ発現停止 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024219757A Pending JP2025032342A (ja) | 2018-12-31 | 2024-12-16 | Ｒｎａ標的化ｃｒｉｓｐｒ－ｃａｓ１３ｂを使用するｄｕｘ４ ｒｎａ発現停止 |
Country Status (8)
| Country | Link |
|---|---|
| US (1) | US12545909B2 (https=) |
| EP (1) | EP3906308A1 (https=) |
| JP (2) | JP2022516515A (https=) |
| KR (1) | KR20210110345A (https=) |
| AU (1) | AU2019419494B2 (https=) |
| CA (1) | CA3124963A1 (https=) |
| IL (1) | IL284447B2 (https=) |
| WO (1) | WO2020142479A1 (https=) |
Families Citing this family (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3829595A4 (en) | 2018-08-02 | 2022-08-24 | Dyne Therapeutics, Inc. | MUSCLE-DIRECTED COMPLEXES AND USES THEREOF IN THE TREATMENT OF DYSTROPHINOPATHIES |
| US20220193250A1 (en) | 2018-08-02 | 2022-06-23 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| 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 |
| WO2021113270A1 (en) | 2019-12-02 | 2021-06-10 | Shape Therapeutics Inc. | Therapeutic editing |
| WO2021142267A1 (en) | 2020-01-08 | 2021-07-15 | Scarpmap Llc | Methods and computing system for processing ultrasound image to determine health of subdermal tissue |
| US11555190B2 (en) | 2020-03-19 | 2023-01-17 | Avidity Biosciences, Inc. | Compositions and methods of treating Facioscapulohumeral muscular dystrophy |
| IL301187A (en) | 2020-09-11 | 2023-05-01 | Arrowhead Pharmaceuticals Inc | RNAi agents for suppressing DUX4 expression, preparations containing them and uses thereof |
| IL303230A (en) * | 2020-11-30 | 2023-07-01 | Res Inst Nationwide Childrens Hospital | Compositions and methods for treating facioscapulohumeral muscular dystrophy (fshd) |
| US20250011775A1 (en) * | 2021-05-25 | 2025-01-09 | Adrian Briggs | Engineered Guide RNAs and Polynucleotides |
| GB2624791A (en) * | 2021-06-17 | 2024-05-29 | Epicrispr Biotechnologies Inc | Systems and methods for regulating aberrant gene expressions |
| AU2022307365A1 (en) * | 2021-07-08 | 2024-02-01 | Tenaya Therapeutics, Inc. | Optimized expression cassettes for gene therapy |
| MX2024003266A (es) | 2021-09-16 | 2024-04-03 | Avidity Biosciences Inc | Composiciones y metodos de tratamiento de la distrofia muscular facioescapulohumeral. |
| WO2025137547A1 (en) * | 2023-12-22 | 2025-06-26 | Dyne Therapeutics, Inc. | Muscle targeting complexes and formulations for treating facioscapulohumeral muscular dystrophy |
Family Cites Families (39)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5173414A (en) | 1990-10-30 | 1992-12-22 | Applied Immune Sciences, Inc. | Production of recombinant adeno-associated virus vectors |
| PT728214E (pt) | 1993-11-09 | 2004-11-30 | Ohio Med College | Linhas celulares estaveis capazes de expressar o gene de replicacao do virus adeno-associado |
| PT733103E (pt) | 1993-11-09 | 2004-07-30 | Targeted Genetics Corp | Criacao de elevados titulos de vectores de aav recombinantes |
| US5658785A (en) | 1994-06-06 | 1997-08-19 | Children's Hospital, Inc. | Adeno-associated virus materials and methods |
| US5856152A (en) | 1994-10-28 | 1999-01-05 | The Trustees Of The University Of Pennsylvania | Hybrid adenovirus-AAV vector and methods of use therefor |
| AU707866B2 (en) | 1994-12-06 | 1999-07-22 | Targeted Genetics Corporation | Packaging cell lines for generation of high titers of recombinant AAV vectors |
| FR2737730B1 (fr) | 1995-08-10 | 1997-09-05 | Pasteur Merieux Serums Vacc | Procede de purification de virus par chromatographie |
| AU722196B2 (en) | 1995-08-30 | 2000-07-27 | Genzyme Corporation | Chromatographic purification of adenovirus and AAV |
| EP1983057A3 (en) | 1995-09-08 | 2009-01-07 | Genzyme Corporation | Improved AAV vectors for gene therapy |
| US5910434A (en) | 1995-12-15 | 1999-06-08 | Systemix, Inc. | Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant |
| EP0932418B1 (en) | 1996-09-06 | 2007-12-05 | The Trustees Of The University Of Pennsylvania | Method for recombinant adeno-associated virus-directed gene therapy |
| JP2001514845A (ja) | 1997-09-05 | 2001-09-18 | ターゲティッド ジェネティクス コーポレイション | 組換えａａｖベクターの高力価ヘルパーなし調製物を生成するための方法 |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| US6258595B1 (en) | 1999-03-18 | 2001-07-10 | The Trustees Of The University Of Pennsylvania | Compositions and methods for helper-free production of recombinant adeno-associated viruses |
| CA2406743A1 (en) | 2000-04-28 | 2001-11-08 | The Trustees Of The University Of Pennsylvania | Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids |
| USH2191H1 (en) * | 2000-10-24 | 2007-06-05 | Snp Consortium | Identification and mapping of single nucleotide polymorphisms in the human genome |
| AU2002248297A1 (en) | 2001-01-05 | 2002-07-16 | Children's Hospital, Inc. | Aav2 vectors and methods |
| DK2359869T3 (en) | 2001-12-17 | 2019-04-15 | Univ Pennsylvania | Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof |
| US7250496B2 (en) * | 2002-11-14 | 2007-07-31 | Rosetta Genomics Ltd. | Bioinformatically detectable group of novel regulatory genes and uses thereof |
| WO2012024535A2 (en) * | 2010-08-18 | 2012-02-23 | Fred Hutchinson Cancer Research Center | Methods for determining the presence or risk of developing facioscapulohumeral dystrophy (fshd) |
| WO2012109570A1 (en) * | 2011-02-10 | 2012-08-16 | The University Of North Carolina At Chapel Hill | Viral vectors with modified transduction profiles and methods of making and using the same |
| AU2012286994B2 (en) | 2011-07-25 | 2017-08-10 | Nationwide Children's Hospital, Inc. | Recombinant virus products and methods for inhibition of expression of DUX4 |
| DE102012007232B4 (de) | 2012-04-07 | 2014-03-13 | Susanne Weller | Verfahren zur Herstellung von rotierenden elektrischen Maschinen |
| JP2015092462A (ja) | 2013-09-30 | 2015-05-14 | Tdk株式会社 | 正極及びそれを用いたリチウムイオン二次電池 |
| JP6202701B2 (ja) | 2014-03-21 | 2017-09-27 | 株式会社日立国際電気 | 基板処理装置、半導体装置の製造方法及びプログラム |
| JP6197169B2 (ja) | 2014-09-29 | 2017-09-20 | 東芝メモリ株式会社 | 半導体装置の製造方法 |
| US10538763B2 (en) * | 2015-01-16 | 2020-01-21 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulation of DUX4 |
| CN109153980B (zh) * | 2015-10-22 | 2023-04-14 | 布罗德研究所有限公司 | Vi-b型crispr酶和系统 |
| US10828377B2 (en) * | 2015-10-26 | 2020-11-10 | The University Of Tokyo | Method for determining presence or absence of suffering from malignant lymphoma or leukemia, and agent for treatment and/or prevention of leukemia |
| FI3373980T3 (fi) * | 2015-11-12 | 2025-07-10 | The Res Institute At Nationwide Childrens Hospital | Menetelmiä lihasdystrofian hoitamiseksi |
| AU2017222653B2 (en) * | 2016-02-26 | 2023-06-01 | Research Institute At Nationwide Children's Hospital | Recombinant virus products and methods for inducing DUX4 exon skipping |
| BR112018070249A2 (pt) * | 2016-04-02 | 2019-01-29 | Research Institute At Nationwide Children's Hospital | sistema promotor de u6 modificada para expressão específica de tecido |
| CA3074723A1 (en) * | 2016-09-23 | 2018-03-29 | University Of Massachusetts | Silencing of dux4 by recombinant gene editing complexes |
| JP7231226B2 (ja) * | 2016-11-07 | 2023-03-01 | ユニバーシティ オブ マサチューセッツ | 顔面肩甲上腕型筋ジストロフィーのための治療標的 |
| AU2018234825B2 (en) | 2017-03-15 | 2020-12-17 | Massachusetts Institute Of Technology | Novel CAS13B orthologues CRISPR enzymes and systems |
| AU2018251801B2 (en) * | 2017-04-12 | 2024-11-07 | Massachusetts Institute Of Technology | Novel type VI CRISPR orthologs and systems |
| EP3640329A1 (en) * | 2018-10-18 | 2020-04-22 | Fundación del Sector Público Estatal Centro Nacional de Investigaciones Oncológicas Carlos III (F.S.P. CNIO) | Gene editing based cancer treatment |
| IL303230A (en) * | 2020-11-30 | 2023-07-01 | Res Inst Nationwide Childrens Hospital | Compositions and methods for treating facioscapulohumeral muscular dystrophy (fshd) |
| WO2022169922A1 (en) * | 2021-02-03 | 2022-08-11 | Research Institute At Nationwide Children's Hospital | Compositions and methods for treating disease associated with dux4 overexpression |
-
2019
- 2019-12-31 AU AU2019419494A patent/AU2019419494B2/en active Active
- 2019-12-31 US US17/419,171 patent/US12545909B2/en active Active
- 2019-12-31 IL IL284447A patent/IL284447B2/en unknown
- 2019-12-31 EP EP19845752.5A patent/EP3906308A1/en active Pending
- 2019-12-31 WO PCT/US2019/069048 patent/WO2020142479A1/en not_active Ceased
- 2019-12-31 KR KR1020217023871A patent/KR20210110345A/ko active Pending
- 2019-12-31 JP JP2021538230A patent/JP2022516515A/ja active Pending
- 2019-12-31 CA CA3124963A patent/CA3124963A1/en active Pending
-
2024
- 2024-12-16 JP JP2024219757A patent/JP2025032342A/ja active Pending
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JPWO2020142479A5 (https=) | ||
| JP2021534755A5 (https=) | ||
| JP2022500066A5 (https=) | ||
| JP2020019772A5 (https=) | ||
| FI3851537T3 (fi) | Hyperbilirubinemian hoito | |
| CA2562788A1 (en) | Adeno-associated virus vectors with intravector heterologous terminal palindromic sequences | |
| IL320791A (en) | Muscle-specific delivery of microdystrophin using an adeno-associated virus vector for the treatment of muscular dystrophy | |
| EP2623605A1 (en) | RAAV vector compositions having tyrosine-modified capsid proteins and methods for use | |
| JP2024095717A5 (https=) | ||
| IL320714A (en) | Gene therapy for limb-girdle muscular dystrophy type 2c | |
| JP2022516515A (ja) | Ｒｎａ標的化ｃｒｉｓｐｒ－ｃａｓ１３ｂを使用するｄｕｘ４ ｒｎａ発現停止 | |
| IL314884A (en) | Methods and materials for galgt2 gene therapy | |
| RU2019133002A (ru) | ЭКСПРЕССИОННЫЕ КАССЕТЫ MeCP2 | |
| JPWO2020069461A5 (https=) | ||
| WO2020142714A1 (en) | Aav expression cassette and aav vectors comprising the same | |
| JP2021534794A5 (https=) | ||
| FI3373980T3 (fi) | Menetelmiä lihasdystrofian hoitamiseksi | |
| JPWO2020214609A5 (https=) | ||
| JPWO2020041634A5 (https=) | ||
| JPWO2021226363A5 (https=) | ||
| IL320715A (en) | Gene therapy with dual dysperlin vectors | |
| JPWO2020047268A5 (https=) | ||
| JPWO2022115745A5 (https=) | ||
| JPWO2019143950A5 (https=) | ||
| JPWO2022060841A5 (https=) |