JP2018533959A5 - - Google Patents
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- JP2018533959A5 JP2018533959A5 JP2018525652A JP2018525652A JP2018533959A5 JP 2018533959 A5 JP2018533959 A5 JP 2018533959A5 JP 2018525652 A JP2018525652 A JP 2018525652A JP 2018525652 A JP2018525652 A JP 2018525652A JP 2018533959 A5 JP2018533959 A5 JP 2018533959A5
- Authority
- JP
- Japan
- Prior art keywords
- csx1
- dna
- use according
- grna
- titin
- 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.)
- Granted
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- 108010002947 Connectin Proteins 0.000 claims description 53
- 230000005782 double-strand break Effects 0.000 claims description 53
- 108010008532 Deoxyribonuclease I Proteins 0.000 claims description 39
- 102000007260 Deoxyribonuclease I Human genes 0.000 claims description 39
- 238000000034 method Methods 0.000 claims description 38
- 210000004027 cell Anatomy 0.000 claims description 19
- 102000040430 polynucleotide Human genes 0.000 claims description 19
- 108091033319 polynucleotide Proteins 0.000 claims description 19
- 239000002157 polynucleotide Substances 0.000 claims description 19
- 108020005004 Guide RNA Proteins 0.000 claims description 18
- 102000004726 Connectin Human genes 0.000 claims description 15
- 108020004414 DNA Proteins 0.000 claims description 15
- 102000053602 DNA Human genes 0.000 claims description 15
- 229920002477 rna polymer Polymers 0.000 claims description 11
- 108091033409 CRISPR Proteins 0.000 claims description 10
- 230000035772 mutation Effects 0.000 claims description 10
- 102000004533 Endonucleases Human genes 0.000 claims description 9
- 108010042407 Endonucleases Proteins 0.000 claims description 9
- 239000002299 complementary DNA Substances 0.000 claims description 7
- 150000002632 lipids Chemical class 0.000 claims description 7
- 238000012986 modification Methods 0.000 claims description 7
- 230000004048 modification Effects 0.000 claims description 7
- 239000002105 nanoparticle Substances 0.000 claims description 7
- 230000004952 protein activity Effects 0.000 claims description 7
- 125000006850 spacer group Chemical group 0.000 claims description 7
- 230000000295 complement effect Effects 0.000 claims description 6
- 108020004999 messenger RNA Proteins 0.000 claims description 6
- 230000000747 cardiac effect Effects 0.000 claims description 5
- 239000013611 chromosomal DNA Substances 0.000 claims description 5
- 241000702421 Dependoparvovirus Species 0.000 claims description 4
- 101710163270 Nuclease Proteins 0.000 claims description 4
- 102000001708 Protein Isoforms Human genes 0.000 claims description 4
- 108010029485 Protein Isoforms Proteins 0.000 claims description 4
- 210000005260 human cell Anatomy 0.000 claims description 4
- 238000003780 insertion Methods 0.000 claims description 4
- 230000037431 insertion Effects 0.000 claims description 4
- 101150069031 CSN2 gene Proteins 0.000 claims description 3
- 208000031229 Cardiomyopathies Diseases 0.000 claims description 3
- -1 Csm2 Proteins 0.000 claims description 3
- 101150106478 GPS1 gene Proteins 0.000 claims description 3
- 101100219625 Mus musculus Casd1 gene Proteins 0.000 claims description 3
- 101100385413 Neurospora crassa (strain ATCC 24698 / 74-OR23-1A / CBS 708.71 / DSM 1257 / FGSC 987) csm-3 gene Proteins 0.000 claims description 3
- 101100047461 Rattus norvegicus Trpm8 gene Proteins 0.000 claims description 3
- 101150055766 cat gene Proteins 0.000 claims description 3
- 210000000349 chromosome Anatomy 0.000 claims description 3
- 101150055601 cops2 gene Proteins 0.000 claims description 3
- 239000002773 nucleotide Substances 0.000 claims description 3
- 125000003729 nucleotide group Chemical group 0.000 claims description 3
- 230000005783 single-strand break Effects 0.000 claims description 3
- 101150018129 CSF2 gene Proteins 0.000 claims description 2
- 101150074775 Csf1 gene Proteins 0.000 claims description 2
- 108700024394 Exon Proteins 0.000 claims description 2
- 208000021642 Muscular disease Diseases 0.000 claims description 2
- 201000009623 Myopathy Diseases 0.000 claims description 2
- 239000012634 fragment Substances 0.000 claims description 2
- 238000010362 genome editing Methods 0.000 claims description 2
- 230000001575 pathological effect Effects 0.000 claims description 2
- 238000011144 upstream manufacturing Methods 0.000 claims description 2
- 102000039446 nucleic acids Human genes 0.000 claims 13
- 108020004707 nucleic acids Proteins 0.000 claims 13
- 150000007523 nucleic acids Chemical class 0.000 claims 13
- 108091027544 Subgenomic mRNA Proteins 0.000 claims 8
- 238000003776 cleavage reaction Methods 0.000 claims 3
- 230000007017 scission Effects 0.000 claims 3
- 210000004602 germ cell Anatomy 0.000 claims 2
- 230000006780 non-homologous end joining Effects 0.000 claims 2
- 238000011084 recovery Methods 0.000 claims 2
- 108091026890 Coding region Proteins 0.000 claims 1
- 238000007385 chemical modification Methods 0.000 claims 1
- 230000000694 effects Effects 0.000 claims 1
- 238000010363 gene targeting Methods 0.000 claims 1
- 238000000338 in vitro Methods 0.000 claims 1
- 230000001404 mediated effect Effects 0.000 claims 1
- 108090000623 proteins and genes Proteins 0.000 claims 1
- 210000004413 cardiac myocyte Anatomy 0.000 description 5
- 238000001574 biopsy Methods 0.000 description 2
- 230000034431 double-strand break repair via homologous recombination Effects 0.000 description 2
- 108091032973 (ribonucleotides)n+m Proteins 0.000 description 1
- 102000040650 (ribonucleotides)n+m Human genes 0.000 description 1
- 238000004113 cell culture Methods 0.000 description 1
- 238000005119 centrifugation Methods 0.000 description 1
- 102000038379 digestive enzymes Human genes 0.000 description 1
- 108091007734 digestive enzymes Proteins 0.000 description 1
- 238000005194 fractionation Methods 0.000 description 1
- 238000001727 in vivo Methods 0.000 description 1
- 238000002347 injection Methods 0.000 description 1
- 239000007924 injection Substances 0.000 description 1
- 230000010412 perfusion Effects 0.000 description 1
- 238000005067 remediation Methods 0.000 description 1
- 210000000130 stem cell Anatomy 0.000 description 1
- 230000009885 systemic effect Effects 0.000 description 1
- 210000001519 tissue Anatomy 0.000 description 1
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2021177441A JP2022009734A (ja) | 2015-11-16 | 2021-10-29 | タイチン系ミオパチー及び他のタイチノパチーの治療のための材料及び方法 |
| JP2023190737A JP2023184679A (ja) | 2015-11-16 | 2023-11-08 | タイチン系ミオパチー及び他のタイチノパチーの治療のための材料及び方法 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562255887P | 2015-11-16 | 2015-11-16 | |
| US62/255,887 | 2015-11-16 | ||
| PCT/US2016/062052 WO2017087395A1 (en) | 2015-11-16 | 2016-11-15 | Materials and methods for treatment of titin-based myopathies and other titinopaties |
Related Child Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021177441A Division JP2022009734A (ja) | 2015-11-16 | 2021-10-29 | タイチン系ミオパチー及び他のタイチノパチーの治療のための材料及び方法 |
| JP2023190737A Division JP2023184679A (ja) | 2015-11-16 | 2023-11-08 | タイチン系ミオパチー及び他のタイチノパチーの治療のための材料及び方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018533959A JP2018533959A (ja) | 2018-11-22 |
| JP2018533959A5 true JP2018533959A5 (enExample) | 2019-12-26 |
| JP7418957B2 JP7418957B2 (ja) | 2024-01-22 |
Family
ID=58717720
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018525652A Active JP7418957B2 (ja) | 2015-11-16 | 2016-11-15 | タイチン系ミオパチー及び他のタイチノパチーの治療のための材料及び方法 |
| JP2021177441A Pending JP2022009734A (ja) | 2015-11-16 | 2021-10-29 | タイチン系ミオパチー及び他のタイチノパチーの治療のための材料及び方法 |
| JP2023190737A Withdrawn JP2023184679A (ja) | 2015-11-16 | 2023-11-08 | タイチン系ミオパチー及び他のタイチノパチーの治療のための材料及び方法 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021177441A Pending JP2022009734A (ja) | 2015-11-16 | 2021-10-29 | タイチン系ミオパチー及び他のタイチノパチーの治療のための材料及び方法 |
| JP2023190737A Withdrawn JP2023184679A (ja) | 2015-11-16 | 2023-11-08 | タイチン系ミオパチー及び他のタイチノパチーの治療のための材料及び方法 |
Country Status (14)
| Country | Link |
|---|---|
| US (2) | US11617783B2 (enExample) |
| EP (1) | EP3377042A4 (enExample) |
| JP (3) | JP7418957B2 (enExample) |
| KR (1) | KR102877920B1 (enExample) |
| CN (1) | CN108463211B (enExample) |
| AU (1) | AU2016358172B2 (enExample) |
| CA (1) | CA3005633C (enExample) |
| CO (1) | CO2018006133A2 (enExample) |
| EA (1) | EA201891192A1 (enExample) |
| HK (1) | HK1257955A1 (enExample) |
| IL (1) | IL259441B2 (enExample) |
| MX (1) | MX2018006116A (enExample) |
| SG (1) | SG11201804166YA (enExample) |
| WO (1) | WO2017087395A1 (enExample) |
Families Citing this family (39)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP6261500B2 (ja) | 2011-07-22 | 2018-01-17 | プレジデント アンド フェローズ オブ ハーバード カレッジ | ヌクレアーゼ切断特異性の評価および改善 |
| US20150044192A1 (en) | 2013-08-09 | 2015-02-12 | 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 |
| US9322037B2 (en) | 2013-09-06 | 2016-04-26 | President And Fellows Of Harvard College | Cas9-FokI fusion proteins and uses thereof |
| US9737604B2 (en) | 2013-09-06 | 2017-08-22 | President And Fellows Of Harvard College | Use of cationic lipids to deliver CAS9 |
| US9228207B2 (en) | 2013-09-06 | 2016-01-05 | President And Fellows Of Harvard College | Switchable gRNAs comprising aptamers |
| US9068179B1 (en) | 2013-12-12 | 2015-06-30 | President And Fellows Of Harvard College | Methods for correcting presenilin point mutations |
| US10077453B2 (en) | 2014-07-30 | 2018-09-18 | President And Fellows Of Harvard College | CAS9 proteins including ligand-dependent inteins |
| EP3365356B1 (en) | 2015-10-23 | 2023-06-28 | President and Fellows of Harvard College | Nucleobase editors and uses thereof |
| JP7231935B2 (ja) | 2016-08-03 | 2023-03-08 | プレジデント アンド フェローズ オブ ハーバード カレッジ | アデノシン核酸塩基編集因子およびそれらの使用 |
| EP3497214B1 (en) | 2016-08-09 | 2023-06-28 | President and Fellows of Harvard College | Programmable cas9-recombinase fusion proteins and uses thereof |
| WO2018039438A1 (en) | 2016-08-24 | 2018-03-01 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
| EP3526320A1 (en) | 2016-10-14 | 2019-08-21 | President and Fellows of Harvard College | Aav delivery of nucleobase editors |
| US10745677B2 (en) | 2016-12-23 | 2020-08-18 | President And Fellows Of Harvard College | Editing of CCR5 receptor gene to protect against HIV infection |
| WO2018165631A1 (en) | 2017-03-09 | 2018-09-13 | President And Fellows Of Harvard College | Cancer vaccine |
| WO2018165504A1 (en) | 2017-03-09 | 2018-09-13 | President And Fellows Of Harvard College | Suppression of pain by gene editing |
| KR20190127797A (ko) | 2017-03-10 | 2019-11-13 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 시토신에서 구아닌으로의 염기 편집제 |
| KR20240116572A (ko) | 2017-03-23 | 2024-07-29 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제 |
| 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 |
| WO2019023680A1 (en) | 2017-07-28 | 2019-01-31 | President And Fellows Of Harvard College | METHODS AND COMPOSITIONS FOR EVOLUTION OF BASIC EDITORS USING PHAGE-ASSISTED CONTINUOUS EVOLUTION (PACE) |
| EP3676376B1 (en) | 2017-08-30 | 2025-01-15 | President and Fellows of Harvard College | High efficiency base editors comprising gam |
| CN111757937A (zh) | 2017-10-16 | 2020-10-09 | 布罗德研究所股份有限公司 | 腺苷碱基编辑器的用途 |
| WO2019118949A1 (en) | 2017-12-15 | 2019-06-20 | The Broad Institute, Inc. | Systems and methods for predicting repair outcomes in genetic engineering |
| JP7361701B2 (ja) | 2018-01-31 | 2023-10-16 | リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル | 肢帯型筋ジストロフィー２ｃ型のための遺伝子治療 |
| US12157760B2 (en) | 2018-05-23 | 2024-12-03 | The Broad Institute, Inc. | Base editors and uses thereof |
| WO2020006458A1 (en) * | 2018-06-29 | 2020-01-02 | Research Institute At Nationwide Children's Hospital | Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2a |
| US12281338B2 (en) | 2018-10-29 | 2025-04-22 | The Broad Institute, Inc. | Nucleobase editors comprising GeoCas9 and uses thereof |
| WO2020154500A1 (en) | 2019-01-23 | 2020-07-30 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| EP3930765A1 (en) | 2019-02-26 | 2022-01-05 | Research Institute at Nationwide Children's Hospital | Adeno-associated virus vector delivery of b-sarcoglycan and the treatment of muscular dystrophy |
| MX2021011426A (es) | 2019-03-19 | 2022-03-11 | Broad Inst Inc | Metodos y composiciones para editar secuencias de nucleótidos. |
| EP3956349A1 (en) | 2019-04-17 | 2022-02-23 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
| SI4017871T1 (sl) | 2019-08-21 | 2024-06-28 | Research Institute At Nationwide Children's Hospital | Dostava alfa-sarkoglikana z adeno povezanim virusnim vektorjem in zdravljenje mišične distrofije |
| US12435330B2 (en) | 2019-10-10 | 2025-10-07 | The Broad Institute, Inc. | Methods and compositions for prime editing RNA |
| MX2022014008A (es) | 2020-05-08 | 2023-02-09 | Broad Inst Inc | Métodos y composiciones para la edición simultánea de ambas cadenas de una secuencia de nucleótidos de doble cadena objetivo. |
| EP4288541A1 (en) | 2021-02-05 | 2023-12-13 | Christiana Care Gene Editing Institute, Inc. | Methods of and compositions for reducing gene expression and/or activity |
| CN113512536A (zh) * | 2021-05-07 | 2021-10-19 | 山东大学深圳研究院 | 一种用于制备人体多巴胺神经元的试剂盒及其应用 |
| JP2023059858A (ja) | 2021-10-15 | 2023-04-27 | リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル | 自己相補的アデノ随伴ウイルスベクター及び筋ジストロフィーの治療におけるその使用 |
| WO2023190935A1 (en) * | 2022-03-31 | 2023-10-05 | Modalis Therapeutics Corporation | Method for treating myopathies by targeting titin gene |
| EP4514967A2 (en) * | 2022-04-25 | 2025-03-05 | The Jackson Laboratory | Methods for treating dilated cardiomyopathy and pharmaceutical compositions therefor |
Family Cites Families (107)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP0127839B1 (en) | 1983-05-27 | 1992-07-15 | THE TEXAS A&M UNIVERSITY SYSTEM | Method for producing a recombinant baculovirus expression vector |
| DK518384A (da) | 1984-01-31 | 1985-07-01 | Idaho Res Found | Vektor til fremstilling af et gen-produkt i insektceller, fremgangsmaade til dens fremstilling samt dens anvendelse |
| US5449616A (en) | 1990-05-23 | 1995-09-12 | University Of Iowa Research Foundation | Nucleic acid encoding dystrophin-associated protein |
| US5173414A (en) | 1990-10-30 | 1992-12-22 | Applied Immune Sciences, Inc. | Production of recombinant adeno-associated virus vectors |
| BR9407135A (pt) | 1993-07-23 | 1996-09-17 | Unichema Chemie Bv | Processo para produzir material de sabão transparente e barras macarrões ou pelotas de sabão transparentes |
| DE69433922T2 (de) | 1993-11-09 | 2005-07-28 | Medical College Of Ohio, Toledo | Stabile zellinie, die in der lage ist, das replikationsgen des adenoassoziertenvirus zu exprimieren |
| DK0733103T3 (da) | 1993-11-09 | 2004-07-12 | Univ Johns Hopkins | Fremstilling af höje titre af rekombinante AAV vektorer |
| US5658785A (en) | 1994-06-06 | 1997-08-19 | Children's Hospital, Inc. | Adeno-associated virus materials and methods |
| US6204059B1 (en) | 1994-06-30 | 2001-03-20 | University Of Pittsburgh | AAV capsid vehicles for molecular transfer |
| US5856152A (en) | 1994-10-28 | 1999-01-05 | The Trustees Of The University Of Pennsylvania | Hybrid adenovirus-AAV vector and methods of use therefor |
| CA2207927A1 (en) | 1994-12-06 | 1996-06-13 | 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 |
| WO1997008298A1 (en) | 1995-08-30 | 1997-03-06 | Genzyme Corporation | Chromatographic purification of adenovirus and aav |
| CA2230758A1 (en) | 1995-09-08 | 1997-03-13 | Genzyme Corporation | Improved aav vectors for gene therapy |
| US5672694A (en) | 1995-10-24 | 1997-09-30 | University Of Iowa Research Foundation | β-sarcoglycan nucleic acid sequence, and nucleic acid probes |
| US5910434A (en) | 1995-12-15 | 1999-06-08 | Systemix, Inc. | Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant |
| CA2264483C (en) | 1996-09-06 | 2011-03-22 | James M. Wilson | Method for recombinant adeno-associated virus-directed gene therapy |
| EP1944362B1 (en) | 1997-09-05 | 2015-10-21 | Genzyme Corporation | Methods for generating high titer helper-free preparations of recombinant AAV vectors |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| AU2882899A (en) | 1998-02-26 | 1999-09-15 | Trustees Of The University Of Pennsylvania, The | Stable protection from dystrophic sarcolemmal degeneration and restoration of the sarcoglycan complex |
| US6262035B1 (en) | 1998-10-01 | 2001-07-17 | University Of Iowa Research Foundation | Gene replacement therapy for muscular dystrophy |
| 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 |
| US6632800B1 (en) | 1999-08-17 | 2003-10-14 | Mayo Foundation For Medical Education And Research | System for monitoring the expression of transgenes |
| JP4520569B2 (ja) | 2000-02-18 | 2010-08-04 | 照彦 豊岡 | 拡張型心筋症の遺伝子治療剤 |
| JP2004514407A (ja) | 2000-04-28 | 2004-05-20 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | 異種キャプシド中にシュードタイピングされたａａｖ５キャプシドおよびａａｖ５ベクターを含む組換えａａｖベクター |
| US6962815B2 (en) | 2001-01-05 | 2005-11-08 | Children's Hopital Inc. | AAV2 vectors and methods |
| CA2455884C (en) | 2001-08-15 | 2015-09-22 | Justin R. Fallon | Treatment of muscular dystrophies and related disorders |
| US20040126762A1 (en) | 2002-12-17 | 2004-07-01 | Morris David W. | Novel compositions and methods in cancer |
| EP1453547B1 (en) | 2001-12-17 | 2016-09-21 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (aav) serotype 8 sequences, vectors containing same, and uses therefor |
| WO2003074714A1 (en) | 2002-03-05 | 2003-09-12 | Stichting Voor De Technische Wetenschappen | Baculovirus expression system |
| WO2003092594A2 (en) | 2002-04-30 | 2003-11-13 | Duke University | Adeno-associated viral vectors and methods for their production from hybrid adenovirus and for their use |
| US20030225260A1 (en) | 2002-04-30 | 2003-12-04 | Snyder Richard O. | Production of recombinant AAV virions |
| EP1751275B1 (en) | 2004-06-01 | 2017-08-16 | Avigen, Inc. | Compositions and methods to prevent aav vector aggregation |
| US7972593B2 (en) | 2004-06-10 | 2011-07-05 | Saint Louis University | Delivery of therapeutic agents to the bone |
| US20090054823A1 (en) | 2004-09-30 | 2009-02-26 | The Trustees Of The University Of Pennsylvania | Perfusion circuit and use therein in targeted delivery of macromolecules |
| JP2006121961A (ja) | 2004-10-28 | 2006-05-18 | Univ Of Tokushima | 顎骨骨幹異形成症ｇｄｄの原因遺伝子ｇｄｄ１とその用途 |
| US7883858B2 (en) | 2005-01-27 | 2011-02-08 | Institute For Systems Biology | Methods for identifying and monitoring drug side effects |
| US7788045B2 (en) | 2005-09-01 | 2010-08-31 | Meditasks, Llc | Systems and method for homeostatic blood states |
| US20070099251A1 (en) | 2005-10-17 | 2007-05-03 | Institute For Systems Biology | Tissue-and serum-derived glycoproteins and methods of their use |
| WO2007049095A1 (en) | 2005-10-25 | 2007-05-03 | Cellectis | Laglidadg homing endonuclease variants having mutations in two functional subdomains and use thereof |
| EP2018421B1 (en) | 2006-04-28 | 2012-12-19 | The Trustees of the University of Pennsylvania | Scalable production method for aav |
| WO2008016356A2 (en) | 2006-08-02 | 2008-02-07 | Genizon Biosciences | Genemap of the human genes associated with psoriasis |
| AU2007284651B2 (en) | 2006-08-09 | 2014-03-20 | Institute For Systems Biology | Organ-specific proteins and methods of their use |
| WO2008058273A2 (en) * | 2006-11-09 | 2008-05-15 | The Johns Hopkins University | Dedifferentiation of adult mammalian cardiomyocytes into cardiac stem cells |
| CN101711164B (zh) | 2007-01-18 | 2014-06-04 | 密苏里-哥伦比亚大学 | 可修复肌纤维膜中的神经元型一氧化氮合酶的合成小/微-抗肌萎缩蛋白基因 |
| FR2919305B1 (fr) | 2007-07-26 | 2009-09-18 | Genethon Ass Loi De 1901 | Vecteurs viraux adeno-associes pour l'expression de la dysferline. |
| GB0715087D0 (en) | 2007-08-03 | 2007-09-12 | Summit Corp Plc | Drug combinations for the treatment of duchenne muscular dystrophy |
| HUE028662T2 (en) | 2007-10-26 | 2016-12-28 | Academisch Ziekenhuis Leiden | Preparations and methods for controlling muscle disorders |
| CN102027123B (zh) | 2008-03-14 | 2016-03-16 | 人体酶有限公司 | 使用人细胞表达系统重组产生可靠的人蛋白质 |
| US20090280103A1 (en) | 2008-04-04 | 2009-11-12 | Martin Flueck | Regulation of muscle repair |
| US8632764B2 (en) | 2008-04-30 | 2014-01-21 | University Of North Carolina At Chapel Hill | Directed evolution and in vivo panning of virus vectors |
| US8236557B2 (en) | 2008-05-28 | 2012-08-07 | University Of Missouri-Columbia | Hybrid-AAV vectors to deliver large gene expression cassette |
| US20100026655A1 (en) | 2008-07-31 | 2010-02-04 | Avago Technologies Ecbu Ip (Singapore) Pte. Ltd. | Capacitive Touchscreen or Touchpad for Finger or Stylus |
| WO2010065630A2 (en) | 2008-12-03 | 2010-06-10 | The Johns Hopkins University | Compositions and methods for treating hepatic neoplasia |
| US20110023139A1 (en) * | 2008-12-04 | 2011-01-27 | Sigma-Aldrich Co. | Genomic editing of genes involved in cardiovascular disease |
| US20100247495A1 (en) | 2009-03-30 | 2010-09-30 | Tom Ichim | Treatment of Muscular Dystrophy |
| US8946451B2 (en) | 2009-10-05 | 2015-02-03 | Catabasis Pharmaceuticals, Inc. | Lipoic acid acylated salicylate derivatives and their uses |
| JP2013510434A (ja) | 2009-11-03 | 2013-03-21 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア | 低温水溶液中で多重表面上に酸化亜鉛層が被覆された高輝度発光ダイオード |
| FR2962041B1 (fr) | 2010-07-01 | 2012-07-27 | Genethon | Inhibiteurs de la calpaine 3 pour le traitement de dystrophies musculaires et de cardiomyopathies |
| EP4488370A2 (en) | 2011-07-25 | 2025-01-08 | Nationwide Children's Hospital, Inc. | Recombinant virus products and methods for inhibition of expresssion of dux4 |
| WO2013075008A1 (en) | 2011-11-16 | 2013-05-23 | University Of Florida Research Foundation Inc. | Aav dual vector systems for gene therapy |
| WO2013078316A1 (en) | 2011-11-23 | 2013-05-30 | Nationwide Children's Hospital, Inc. | Recombinant adeno-associated virus delivery of alpha-sarcoglycan polynucleotides |
| JP6373763B2 (ja) | 2012-02-17 | 2018-08-22 | ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィアＴｈｅ Ｃｈｉｌｄｒｅｎ’Ｓ Ｈｏｓｐｉｔａｌ Ｏｆ Ｐｈｉｌａｄｅｌｐｈｉａ | Ａａｖベクター成分、及び、細胞、臓器並びに組織への遺伝子導入のための方法 |
| AU2013243954A1 (en) | 2012-04-02 | 2014-10-30 | Moderna Therapeutics, Inc. | Modified polynucleotides for the production of cosmetic proteins and peptides |
| US9254311B2 (en) | 2012-04-02 | 2016-02-09 | Moderna Therapeutics, Inc. | Modified polynucleotides for the production of proteins |
| MY189533A (en) | 2012-05-25 | 2022-02-16 | Univ California | Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription |
| BR112015005091B1 (pt) | 2012-09-06 | 2021-07-06 | The University Of Chicago | uso de um polinucleotídeo antissenso isolado |
| EP2893038A1 (en) | 2012-09-07 | 2015-07-15 | INSERM - Institut National de la Santé et de la Recherche Médicale | In vitro genetic diagnostic of inherited neuromuscular disorders |
| AU2013323820B2 (en) * | 2012-09-25 | 2019-06-27 | Genzyme Corporation | Peptide-linked morpholino antisense oligonucleotides for treatment of myotonic dystrophy |
| US9624282B2 (en) | 2012-11-26 | 2017-04-18 | The Curators Of The University Of Missouri | Microdystrophin peptides and methods for treating muscular dystrophy using the same |
| EP4299741A3 (en) | 2012-12-12 | 2024-02-28 | The Broad Institute, Inc. | Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications |
| CN119752887A (zh) * | 2012-12-12 | 2025-04-04 | 布罗德研究所有限公司 | 用于序列操纵的系统、方法和优化的指导组合物的工程化 |
| CA2894668A1 (en) | 2012-12-12 | 2014-06-19 | The Broad Institute, Inc. | Crispr-cas systems and methods for altering expression of gene products in eukaryotic cells |
| FR3004463A1 (fr) | 2013-04-11 | 2014-10-17 | Genethon | Systeme d'expression pour une therapie genique selective |
| CN105492611A (zh) | 2013-06-17 | 2016-04-13 | 布罗德研究所有限公司 | 用于序列操纵的优化的crispr-cas双切口酶系统、方法以及组合物 |
| ITTO20130669A1 (it) | 2013-08-05 | 2015-02-06 | Consiglio Nazionale Ricerche | Vettore adeno-associato ricombinante muscolo-specifico e suo impiego nel trattamento di patologie muscolari |
| TW201536329A (zh) | 2013-08-09 | 2015-10-01 | Isis Pharmaceuticals Inc | 用於調節失養性肌強直蛋白質激酶(dmpk)表現之化合物及方法 |
| ES2666322T3 (es) | 2013-10-08 | 2018-05-04 | Ystem S.R.L. | Composiciones farmacéuticas para el tratamiento de trastornos musculares |
| US9850497B2 (en) * | 2013-11-04 | 2017-12-26 | Regents Of The University Of Minnesota | Gene targeting methods and tools |
| WO2015089419A2 (en) * | 2013-12-12 | 2015-06-18 | The Broad Institute Inc. | Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using particle delivery components |
| SG11201605906UA (en) | 2014-01-21 | 2016-08-30 | Univ Bruxelles | Muscle-specific nucleic acid regulatory elements and methods and use thereof |
| DE102014207498A1 (de) | 2014-04-17 | 2015-10-22 | Universitätsklinikum Hamburg-Eppendorf | Viraler Vektor für den zielgerichteten Gentransfer in Gehirn und Rückenmark |
| EP2960336A1 (en) | 2014-06-27 | 2015-12-30 | Genethon | Efficient systemic treatment of dystrophic muscle pathologies |
| EP4599851A3 (en) | 2015-01-16 | 2025-12-31 | University of Washington | NEW MICRO-DYSTROPHINS AND ASSOCIATED METHODS OF USE |
| WO2017165859A1 (en) | 2016-03-24 | 2017-09-28 | Research Institute At Nationwide Children's Hospital | Modified viral capsid proteins |
| MA45477A (fr) | 2016-04-15 | 2019-02-20 | Res Inst Nationwide Childrens Hospital | Administration à vecteurs de virus adéno-associé de microarn-29 et micro-dystrophine pour traiter la dystrophie musculaire |
| JP7079733B2 (ja) | 2016-04-15 | 2022-06-02 | リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル | β－サルコグリカン及びマイクロＲＮＡ－２９のアデノ随伴ウイルスベクター送達ならびに筋ジストロフィーの治療 |
| SG11201808422QA (en) | 2016-04-15 | 2018-10-30 | Univ Pennsylvania | Gene therapy for treating hemophilia a |
| CA2971303A1 (en) | 2016-06-21 | 2017-12-21 | Bamboo Therapeutics, Inc. | Optimized mini-dystrophin genes and expression cassettes and their use |
| JP7162021B2 (ja) | 2017-03-17 | 2022-10-27 | ニューカッスル ユニバーシティ | 筋ジストロフィーを治療するためのマイクロジストロフィン断片のアデノ随伴ウイルスベクター送達 |
| IL311713B2 (en) | 2017-03-17 | 2025-10-01 | Res Inst Nationwide Childrens Hospital | Muscle-specific delivery of microdystrophin using an adeno-associated virus vector for the treatment of muscular dystrophy |
| WO2019078916A1 (en) | 2017-10-18 | 2019-04-25 | Research Institute At Nationwide Children's Hospital | ADENO-ASSOCIATED VIRUS VECTOR ADMINISTRATION OF MUSCLE-SPECIFIC MICRO-DYSTROPHINE TO TREAT MUSCLE DYSTROPHY |
| AU2018351528B2 (en) | 2017-10-20 | 2025-02-27 | Research Institute At Nationwide Children's Hospital | Methods and materials for NT-3 gene therapy |
| WO2019118806A1 (en) | 2017-12-14 | 2019-06-20 | Solid Biosciences Inc. | Non-viral production and delivery of genes |
| JP7361701B2 (ja) | 2018-01-31 | 2023-10-16 | リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル | 肢帯型筋ジストロフィー２ｃ型のための遺伝子治療 |
| JP2021520196A (ja) | 2018-04-03 | 2021-08-19 | ザ・キュレイターズ・オブ・ザ・ユニバーシティー・オブ・ミズーリＴｈｅ Ｃｕｒａｔｏｒｓ Ｏｆ Ｔｈｅ Ｕｎｉｖｅｒｓｉｔｙ Ｏｆ Ｍｉｓｓｏｕｒｉ | ジストロフィン異常症（ｄｙｓｔｒｏｐｈｉｎｏｐａｔｈｙ）治療のためのヒンジ１および／または４の改変ジストロフィン |
| WO2019209777A1 (en) | 2018-04-23 | 2019-10-31 | The Curators Of The University Of Missouri | Improved crispr therapy |
| JP7646362B2 (ja) | 2018-06-18 | 2025-03-17 | リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル | 筋ジストロフィーを治療するための筋肉特異的マイクロジストロフィンのアデノ随伴ウイルスベクターデリバリー |
| WO2020006458A1 (en) | 2018-06-29 | 2020-01-02 | Research Institute At Nationwide Children's Hospital | Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2a |
| JP7788855B2 (ja) | 2018-12-12 | 2025-12-19 | ソリッド・バイオサイエンシーズ・インコーポレーテッド | 筋ジストロフィーの治療のための併用療法 |
| EP3930765A1 (en) | 2019-02-26 | 2022-01-05 | Research Institute at Nationwide Children's Hospital | Adeno-associated virus vector delivery of b-sarcoglycan and the treatment of muscular dystrophy |
| SI4017871T1 (sl) | 2019-08-21 | 2024-06-28 | Research Institute At Nationwide Children's Hospital | Dostava alfa-sarkoglikana z adeno povezanim virusnim vektorjem in zdravljenje mišične distrofije |
| EP4117783A4 (en) | 2019-12-16 | 2024-08-21 | Research Institute at Nationwide Children's Hospital | COMPOSITIONS AND METHODS FOR RESTORING AND MAINTAINING DYSTROPHIN-ASSOCIATED PROTEIN COMPLEX (DAPC) |
| WO2021257595A1 (en) | 2020-06-15 | 2021-12-23 | Research Institute At Nationwide Children's Hospital | Adeno-associated virus vector delivery for muscular dystrophies |
| CA3182523A1 (en) | 2020-06-17 | 2021-12-23 | Rajasekhar Naga Venkata Sai Suragani | Actrii-alk4 antagonists and methods of treating heart failure |
| TWI887479B (zh) | 2020-09-08 | 2025-06-21 | 美商薩羅塔治療公司 | 表現γ-肌聚醣之腺相關病毒載體之全身性遞送及肌肉失養症之治療 |
| US20230390417A1 (en) | 2022-06-02 | 2023-12-07 | Research Institute At Nationwide Children's Hospital | Recombinant Adeno-Associated Virus Products and Methods for Treating Limb Girdle Muscular Dystrophy 2A |
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