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 (https=) | 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 (https=) |
| EP (1) | EP3377042A4 (https=) |
| JP (3) | JP7418957B2 (https=) |
| KR (1) | KR102877920B1 (https=) |
| CN (1) | CN108463211B (https=) |
| AU (1) | AU2016358172B2 (https=) |
| CA (1) | CA3005633C (https=) |
| CO (1) | CO2018006133A2 (https=) |
| EA (1) | EA201891192A1 (https=) |
| HK (1) | HK1257955A1 (https=) |
| IL (1) | IL259441B2 (https=) |
| MX (1) | MX2018006116A (https=) |
| SG (1) | SG11201804166YA (https=) |
| WO (1) | WO2017087395A1 (https=) |
Families Citing this family (40)
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|---|---|---|---|---|
| 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 |
| US9340799B2 (en) | 2013-09-06 | 2016-05-17 | President And Fellows Of Harvard College | MRNA-sensing switchable gRNAs |
| US9388430B2 (en) | 2013-09-06 | 2016-07-12 | President And Fellows Of Harvard College | Cas9-recombinase fusion proteins and uses thereof |
| US9526784B2 (en) | 2013-09-06 | 2016-12-27 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
| US20150165054A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting caspase-9 point mutations |
| WO2016022363A2 (en) | 2014-07-30 | 2016-02-11 | President And Fellows Of Harvard College | Cas9 proteins including ligand-dependent inteins |
| JP7109784B2 (ja) | 2015-10-23 | 2022-08-01 | 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 | 遺伝子編集のための進化したＣａｓ９蛋白質 |
| KR102547316B1 (ko) | 2016-08-03 | 2023-06-23 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 아데노신 핵염기 편집제 및 그의 용도 |
| US11661590B2 (en) | 2016-08-09 | 2023-05-30 | President And Fellows Of Harvard College | Programmable CAS9-recombinase fusion proteins and uses thereof |
| US11542509B2 (en) | 2016-08-24 | 2023-01-03 | 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 |
| WO2018119359A1 (en) | 2016-12-23 | 2018-06-28 | President And Fellows Of Harvard College | Editing of ccr5 receptor gene to protect against hiv infection |
| EP3592853A1 (en) | 2017-03-09 | 2020-01-15 | President and Fellows of Harvard College | Suppression of pain by gene editing |
| JP2020510038A (ja) | 2017-03-09 | 2020-04-02 | 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 | がんワクチン |
| JP2020510439A (ja) | 2017-03-10 | 2020-04-09 | 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 | シトシンからグアニンへの塩基編集因子 |
| CA3057192A1 (en) | 2017-03-23 | 2018-09-27 | President And Fellows Of Harvard College | Nucleobase editors comprising nucleic acid programmable dna binding proteins |
| WO2018209320A1 (en) | 2017-05-12 | 2018-11-15 | 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 |
| KR20250107288A (ko) | 2017-10-16 | 2025-07-11 | 더 브로드 인스티튜트, 인코퍼레이티드 | 아데노신 염기 편집제의 용도 |
| WO2019118949A1 (en) | 2017-12-15 | 2019-06-20 | The Broad Institute, Inc. | Systems and methods for predicting repair outcomes in genetic engineering |
| MX2020007876A (es) | 2018-01-31 | 2020-12-03 | Res Inst Nationwide Childrens Hospital | Terapia genica para la distrofia muscular de cinturas tipo 2c. |
| EP3797160A1 (en) | 2018-05-23 | 2021-03-31 | The Broad Institute Inc. | Base editors and uses thereof |
| MX2020014119A (es) * | 2018-06-29 | 2021-06-18 | Res Inst Nationwide Childrens Hospital | Productos y métodos de virus adenoasociado recombinante para el tratamiento de la distrofia muscular de cinturas 2a. |
| US12522807B2 (en) | 2018-07-09 | 2026-01-13 | The Broad Institute, Inc. | RNA programmable epigenetic RNA modifiers and uses thereof |
| WO2020092453A1 (en) | 2018-10-29 | 2020-05-07 | The Broad Institute, Inc. | Nucleobase editors comprising geocas9 and uses thereof |
| US12351837B2 (en) | 2019-01-23 | 2025-07-08 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| MX2021010356A (es) | 2019-02-26 | 2022-03-04 | Res Inst Nationwide Childrens Hospital | SUMINISTRO DE ß-SARCOGLICANO MEDIANTE VECTORES DE VIRUS ADENOASOCIADO Y EL TRATAMIENTO DE LA DISTROFIA MUSCULAR. |
| JP7657726B2 (ja) | 2019-03-19 | 2025-04-07 | ザ ブロード インスティテュート，インコーポレーテッド | 編集ヌクレオチド配列を編集するための方法および組成物 |
| US12473543B2 (en) | 2019-04-17 | 2025-11-18 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
| PT4017871T (pt) | 2019-08-21 | 2024-04-22 | Res Inst Nationwide Childrens Hospital | Distribuição de vetor de vírus adeno-associado de alfasarcoglicano e o tratamento da distrofia muscular |
| US12435330B2 (en) | 2019-10-10 | 2025-10-07 | The Broad Institute, Inc. | Methods and compositions for prime editing RNA |
| DE112021002672T5 (de) | 2020-05-08 | 2023-04-13 | President And Fellows Of Harvard College | Vefahren und zusammensetzungen zum gleichzeitigen editieren beider stränge einer doppelsträngigen nukleotid-zielsequenz |
| 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 |
| US20250268992A1 (en) * | 2022-04-25 | 2025-08-28 | The Jackson Laboratory | Methods for treating dilated cardiomyopathy and pharmaceutical compositions therefor |
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