DK3011031T3 - Fremføring og anvendelse af crispr-cas-systemerne, vektorer og sammensætninger til levermålretning og -terapi - Google Patents
Fremføring og anvendelse af crispr-cas-systemerne, vektorer og sammensætninger til levermålretning og -terapi Download PDFInfo
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- DK3011031T3 DK3011031T3 DK14734710.8T DK14734710T DK3011031T3 DK 3011031 T3 DK3011031 T3 DK 3011031T3 DK 14734710 T DK14734710 T DK 14734710T DK 3011031 T3 DK3011031 T3 DK 3011031T3
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- crispr
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- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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- A01K2217/00—Genetically modified animals
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- A01K2267/00—Animals characterised by purpose
- A01K2267/03—Animal model, e.g. for test or diseases
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- C12N2310/10—Type of nucleic acid
- C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]
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Applications Claiming Priority (8)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201361836123P | 2013-06-17 | 2013-06-17 | |
US201361847537P | 2013-07-17 | 2013-07-17 | |
US201361862355P | 2013-08-05 | 2013-08-05 | |
US201361871301P | 2013-08-28 | 2013-08-28 | |
US201361915325P | 2013-12-12 | 2013-12-12 | |
PCT/US2013/074667 WO2014093622A2 (en) | 2012-12-12 | 2013-12-12 | Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications |
US201461979733P | 2014-04-15 | 2014-04-15 | |
PCT/US2014/041804 WO2014204726A1 (en) | 2013-06-17 | 2014-06-10 | Delivery and use of the crispr-cas systems, vectors and compositions for hepatic targeting and therapy |
Publications (1)
Publication Number | Publication Date |
---|---|
DK3011031T3 true DK3011031T3 (da) | 2020-12-21 |
Family
ID=52105121
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
DK14734710.8T DK3011031T3 (da) | 2013-06-17 | 2014-06-10 | Fremføring og anvendelse af crispr-cas-systemerne, vektorer og sammensætninger til levermålretning og -terapi |
Country Status (14)
Country | Link |
---|---|
US (2) | US10577630B2 (da) |
EP (2) | EP3825406A1 (da) |
JP (4) | JP6738728B2 (da) |
KR (1) | KR20160030187A (da) |
CN (2) | CN113425857A (da) |
AU (3) | AU2014281028B2 (da) |
BR (1) | BR112015031608A2 (da) |
CA (1) | CA2915842C (da) |
DK (1) | DK3011031T3 (da) |
IL (1) | IL243196B (da) |
MX (2) | MX2015017312A (da) |
RU (1) | RU2716420C2 (da) |
SG (2) | SG11201510284XA (da) |
WO (1) | WO2014204726A1 (da) |
Families Citing this family (329)
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US9217155B2 (en) | 2008-05-28 | 2015-12-22 | University Of Massachusetts | Isolation of novel AAV'S and uses thereof |
US8734809B2 (en) | 2009-05-28 | 2014-05-27 | University Of Massachusetts | AAV's and uses thereof |
CA3066596A1 (en) | 2010-04-23 | 2011-10-27 | University Of Massachusetts | Cns targeting aav vectors and methods of use thereof |
CA3050894C (en) | 2010-04-23 | 2022-10-18 | University Of Massachusetts | Multicistronic expression constructs |
EP3318634A1 (en) | 2011-04-21 | 2018-05-09 | University of Massachusetts | Raav-based compositions and methods for treating diseases involving dominant-negative or gain of function mutations |
US10323236B2 (en) | 2011-07-22 | 2019-06-18 | President And Fellows Of Harvard College | Evaluation and improvement of nuclease cleavage specificity |
EP2841572B1 (en) | 2012-04-27 | 2019-06-19 | Duke University | Genetic correction of mutated genes |
LT3241902T (lt) | 2012-05-25 | 2018-06-25 | The Regents Of The University Of California | Būdai ir kompozicijos, skirti rnr nukreiptos tikslinės dnr modifikacijai ir rnr nukreiptos transkripcijos moduliacijai |
US10323279B2 (en) | 2012-08-14 | 2019-06-18 | 10X Genomics, Inc. | Methods and systems for processing polynucleotides |
US10584381B2 (en) | 2012-08-14 | 2020-03-10 | 10X Genomics, Inc. | Methods and systems for processing polynucleotides |
US9388465B2 (en) | 2013-02-08 | 2016-07-12 | 10X Genomics, Inc. | Polynucleotide barcode generation |
CN114891871A (zh) | 2012-08-14 | 2022-08-12 | 10X基因组学有限公司 | 微胶囊组合物及方法 |
US10221442B2 (en) | 2012-08-14 | 2019-03-05 | 10X Genomics, Inc. | Compositions and methods for sample processing |
US10752949B2 (en) | 2012-08-14 | 2020-08-25 | 10X Genomics, Inc. | Methods and systems for processing polynucleotides |
US9951386B2 (en) | 2014-06-26 | 2018-04-24 | 10X Genomics, Inc. | Methods and systems for processing polynucleotides |
US11591637B2 (en) | 2012-08-14 | 2023-02-28 | 10X Genomics, Inc. | Compositions and methods for sample processing |
US10273541B2 (en) | 2012-08-14 | 2019-04-30 | 10X Genomics, Inc. | Methods and systems for processing polynucleotides |
US9701998B2 (en) | 2012-12-14 | 2017-07-11 | 10X Genomics, Inc. | Methods and systems for processing polynucleotides |
CN110643600A (zh) | 2012-10-23 | 2020-01-03 | 基因工具股份有限公司 | 用于切割靶dna的系统及其用途 |
PL3138912T3 (pl) | 2012-12-06 | 2019-04-30 | Sigma Aldrich Co Llc | Modyfikacja i regulacja genomu oparta na CRISPR |
EP3434776A1 (en) | 2012-12-12 | 2019-01-30 | The Broad Institute, Inc. | Methods, models, systems, and apparatus for identifying target sequences for cas enzymes or crispr-cas systems for target sequences and conveying results thereof |
US20140179770A1 (en) | 2012-12-12 | 2014-06-26 | Massachusetts Institute Of Technology | Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications |
EP3567116A1 (en) | 2012-12-14 | 2019-11-13 | 10X Genomics, Inc. | Methods and systems for processing polynucleotides |
US10533221B2 (en) | 2012-12-14 | 2020-01-14 | 10X Genomics, Inc. | Methods and systems for processing polynucleotides |
CN113005148A (zh) | 2013-01-16 | 2021-06-22 | 爱默蕾大学 | Cas9-核酸复合物及其相关用途 |
EP4286517A3 (en) | 2013-04-04 | 2024-03-13 | President and Fellows of Harvard College | Therapeutic uses of genome editing with crispr/cas systems |
SG10201808935WA (en) | 2013-04-16 | 2018-11-29 | Regeneron Pharma | Targeted modification of rat genome |
WO2014204727A1 (en) | 2013-06-17 | 2014-12-24 | The Broad Institute Inc. | Functional genomics using crispr-cas systems, compositions methods, screens and applications thereof |
EP3011034B1 (en) | 2013-06-17 | 2019-08-07 | The Broad Institute, Inc. | Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using viral components |
JP6625971B2 (ja) | 2013-06-17 | 2019-12-25 | ザ・ブロード・インスティテュート・インコーポレイテッド | 配列操作のためのタンデムガイド系、方法および組成物の送達、エンジニアリングおよび最適化 |
DK3011031T3 (da) | 2013-06-17 | 2020-12-21 | Broad Inst Inc | Fremføring og anvendelse af crispr-cas-systemerne, vektorer og sammensætninger til levermålretning og -terapi |
CA2915837A1 (en) | 2013-06-17 | 2014-12-24 | The Broad Institute, Inc. | Optimized crispr-cas double nickase systems, methods and compositions for sequence manipulation |
JP6738729B2 (ja) * | 2013-06-17 | 2020-08-12 | ザ・ブロード・インスティテュート・インコーポレイテッド | 分裂終了細胞の疾患および障害をターゲティングおよびモデリングするための系、方法および組成物の送達、エンジニアリングおよび最適化 |
JP2016528890A (ja) * | 2013-07-09 | 2016-09-23 | 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 | CRISPR/Cas系を用いるゲノム編集の治療用の使用 |
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 |
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 |
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