JP2016520320A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2016520320A5 JP2016520320A5 JP2016516173A JP2016516173A JP2016520320A5 JP 2016520320 A5 JP2016520320 A5 JP 2016520320A5 JP 2016516173 A JP2016516173 A JP 2016516173A JP 2016516173 A JP2016516173 A JP 2016516173A JP 2016520320 A5 JP2016520320 A5 JP 2016520320A5
- Authority
- JP
- Japan
- Prior art keywords
- activity
- cell
- variant
- seq
- onui
- 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
Links
- 230000000694 effects Effects 0.000 claims description 24
- 102100031780 Endonuclease Human genes 0.000 claims description 23
- 108010042407 Endonucleases Proteins 0.000 claims description 23
- 210000004027 cell Anatomy 0.000 claims description 22
- 238000000034 method Methods 0.000 claims description 20
- 108020004414 DNA Proteins 0.000 claims description 14
- 102000004190 Enzymes Human genes 0.000 claims description 10
- 108090000790 Enzymes Proteins 0.000 claims description 10
- 108090000623 proteins and genes Proteins 0.000 claims description 10
- 150000007523 nucleic acids Chemical group 0.000 claims description 8
- 108020001580 protein domains Proteins 0.000 claims description 8
- 108091028043 Nucleic acid sequence Proteins 0.000 claims description 7
- 206010028980 Neoplasm Diseases 0.000 claims description 6
- 102100029452 T cell receptor alpha chain constant Human genes 0.000 claims description 6
- 230000003197 catalytic effect Effects 0.000 claims description 6
- 108091007494 Nucleic acid- binding domains Proteins 0.000 claims description 5
- 210000001744 T-lymphocyte Anatomy 0.000 claims description 5
- 108060002716 Exonuclease Proteins 0.000 claims description 4
- 101000830950 Homo sapiens Three prime repair exonuclease 2 Proteins 0.000 claims description 4
- 102100024872 Three prime repair exonuclease 2 Human genes 0.000 claims description 4
- 201000011510 cancer Diseases 0.000 claims description 4
- 102000013165 exonuclease Human genes 0.000 claims description 4
- 108091033319 polynucleotide Proteins 0.000 claims description 4
- 102000040430 polynucleotide Human genes 0.000 claims description 4
- 239000002157 polynucleotide Substances 0.000 claims description 4
- 108090000765 processed proteins & peptides Proteins 0.000 claims description 4
- 102000004169 proteins and genes Human genes 0.000 claims description 4
- 102100031573 Hematopoietic progenitor cell antigen CD34 Human genes 0.000 claims description 3
- 101000777663 Homo sapiens Hematopoietic progenitor cell antigen CD34 Proteins 0.000 claims description 3
- 210000003958 hematopoietic stem cell Anatomy 0.000 claims description 3
- 210000004698 lymphocyte Anatomy 0.000 claims description 3
- 210000000130 stem cell Anatomy 0.000 claims description 3
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims description 2
- 108010019670 Chimeric Antigen Receptors Proteins 0.000 claims description 2
- 101000634853 Homo sapiens T cell receptor alpha chain constant Proteins 0.000 claims description 2
- 102100034343 Integrase Human genes 0.000 claims description 2
- 108010061833 Integrases Proteins 0.000 claims description 2
- 102000003960 Ligases Human genes 0.000 claims description 2
- 108090000364 Ligases Proteins 0.000 claims description 2
- 108060004795 Methyltransferase Proteins 0.000 claims description 2
- 101100508818 Mus musculus Inpp5k gene Proteins 0.000 claims description 2
- 101710163270 Nuclease Proteins 0.000 claims description 2
- 102000004160 Phosphoric Monoester Hydrolases Human genes 0.000 claims description 2
- 108090000608 Phosphoric Monoester Hydrolases Proteins 0.000 claims description 2
- 108091000080 Phosphotransferase Proteins 0.000 claims description 2
- 101100366438 Rattus norvegicus Sphkap gene Proteins 0.000 claims description 2
- 102000018120 Recombinases Human genes 0.000 claims description 2
- 108010091086 Recombinases Proteins 0.000 claims description 2
- 108091008874 T cell receptors Proteins 0.000 claims description 2
- 102000016266 T-Cell Antigen Receptors Human genes 0.000 claims description 2
- 101710183280 Topoisomerase Proteins 0.000 claims description 2
- 108700019146 Transgenes Proteins 0.000 claims description 2
- 102000008579 Transposases Human genes 0.000 claims description 2
- 108010020764 Transposases Proteins 0.000 claims description 2
- HCHKCACWOHOZIP-UHFFFAOYSA-N Zinc Chemical compound [Zn] HCHKCACWOHOZIP-UHFFFAOYSA-N 0.000 claims description 2
- 125000003275 alpha amino acid group Chemical group 0.000 claims description 2
- 239000000427 antigen Substances 0.000 claims description 2
- 108091007433 antigens Proteins 0.000 claims description 2
- 102000036639 antigens Human genes 0.000 claims description 2
- 238000003776 cleavage reaction Methods 0.000 claims description 2
- 102000034287 fluorescent proteins Human genes 0.000 claims description 2
- 108091006047 fluorescent proteins Proteins 0.000 claims description 2
- 239000011159 matrix material Substances 0.000 claims description 2
- 238000002703 mutagenesis Methods 0.000 claims description 2
- 231100000350 mutagenesis Toxicity 0.000 claims description 2
- 108020004707 nucleic acids Proteins 0.000 claims description 2
- 102000039446 nucleic acids Human genes 0.000 claims description 2
- 102000020233 phosphotransferase Human genes 0.000 claims description 2
- 229920001184 polypeptide Polymers 0.000 claims description 2
- 102000004196 processed proteins & peptides Human genes 0.000 claims description 2
- 230000007017 scission Effects 0.000 claims description 2
- 238000006467 substitution reaction Methods 0.000 claims description 2
- 239000011701 zinc Substances 0.000 claims description 2
- 229910052725 zinc Inorganic materials 0.000 claims description 2
- 239000003814 drug Substances 0.000 claims 4
- 230000019491 signal transduction Effects 0.000 claims 2
- 108700042076 T-Cell Receptor alpha Genes Proteins 0.000 description 2
- 230000011664 signaling Effects 0.000 description 2
- 239000003153 chemical reaction reagent Substances 0.000 description 1
- 201000010099 disease Diseases 0.000 description 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 description 1
- 238000010362 genome editing Methods 0.000 description 1
- 210000005260 human cell Anatomy 0.000 description 1
- 230000002452 interceptive effect Effects 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| DKPA201370303 | 2013-05-31 | ||
| DKPA201370303 | 2013-05-31 | ||
| PCT/EP2014/061189 WO2014191527A1 (en) | 2013-05-31 | 2014-05-28 | A laglidadg homing endonuclease cleaving the t cell receptor alpha gene and uses thereof |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2016520320A JP2016520320A (ja) | 2016-07-14 |
| JP2016520320A5 true JP2016520320A5 (cg-RX-API-DMAC7.html) | 2017-07-06 |
| JP6450371B2 JP6450371B2 (ja) | 2019-01-09 |
Family
ID=48628226
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016516173A Active JP6450371B2 (ja) | 2013-05-31 | 2014-05-28 | Ｔ細胞受容体α遺伝子を開裂するＬＡＧＬＩＤＡＤＧホーミングエンドヌクレアーゼおよびその用途 |
Country Status (8)
| Country | Link |
|---|---|
| US (1) | US10000746B2 (cg-RX-API-DMAC7.html) |
| EP (1) | EP3004338B1 (cg-RX-API-DMAC7.html) |
| JP (1) | JP6450371B2 (cg-RX-API-DMAC7.html) |
| AU (1) | AU2014273091B2 (cg-RX-API-DMAC7.html) |
| CA (1) | CA2913872C (cg-RX-API-DMAC7.html) |
| DK (1) | DK3004338T3 (cg-RX-API-DMAC7.html) |
| ES (1) | ES2716867T3 (cg-RX-API-DMAC7.html) |
| WO (1) | WO2014191527A1 (cg-RX-API-DMAC7.html) |
Families Citing this family (78)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3320910A1 (en) * | 2011-04-05 | 2018-05-16 | Cellectis | Method for the generation of compact tale-nucleases and uses thereof |
| CA2853829C (en) | 2011-07-22 | 2023-09-26 | President And Fellows Of Harvard College | Evaluation and improvement of nuclease cleavage specificity |
| US10704021B2 (en) | 2012-03-15 | 2020-07-07 | Flodesign Sonics, Inc. | Acoustic perfusion devices |
| 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 |
| US9526784B2 (en) | 2013-09-06 | 2016-12-27 | President And Fellows Of Harvard College | Delivery system for functional nucleases |
| US9228207B2 (en) | 2013-09-06 | 2016-01-05 | President And Fellows Of Harvard College | Switchable gRNAs comprising aptamers |
| US9388430B2 (en) | 2013-09-06 | 2016-07-12 | President And Fellows Of Harvard College | Cas9-recombinase fusion proteins and uses thereof |
| CN111218447B (zh) | 2013-11-07 | 2024-10-11 | 爱迪塔斯医药有限公司 | 使用统治型gRNA的CRISPR相关方法和组合物 |
| US20150166982A1 (en) | 2013-12-12 | 2015-06-18 | President And Fellows Of Harvard College | Methods for correcting pi3k point mutations |
| EP3092049A1 (en) | 2014-01-08 | 2016-11-16 | Flodesign Sonics Inc. | Acoustophoresis device with dual acoustophoretic chamber |
| EP3177718B1 (en) | 2014-07-30 | 2022-03-16 | President and Fellows of Harvard College | Cas9 proteins including ligand-dependent inteins |
| US11708572B2 (en) | 2015-04-29 | 2023-07-25 | Flodesign Sonics, Inc. | Acoustic cell separation techniques and processes |
| KR20250013291A (ko) | 2015-10-05 | 2025-01-31 | 프리시젼 바이오사이언시스 인코포레이티드 | 변형된 인간 t 세포 수용체 알파 불변 영역 유전자를 포함하는 유전자 변형된 세포 |
| EP3940070A1 (en) * | 2015-10-05 | 2022-01-19 | Precision Biosciences, Inc. | Engineered meganucleases with recognition sequences found in the human t cell receptor alpha constant region gene |
| SG11201803173VA (en) | 2015-10-23 | 2018-05-30 | Harvard College | Nucleobase editors and uses thereof |
| CN108699557B (zh) | 2015-12-04 | 2025-08-15 | 诺华股份有限公司 | 用于免疫肿瘤学的组合物和方法 |
| CN109311984A (zh) * | 2016-03-11 | 2019-02-05 | 蓝鸟生物公司 | 基因组编辑的免疫效应细胞 |
| US10188749B2 (en) | 2016-04-14 | 2019-01-29 | Fred Hutchinson Cancer Research Center | Compositions and methods to program therapeutic cells using targeted nucleic acid nanocarriers |
| NZ748378A (en) * | 2016-04-14 | 2025-08-29 | Fred Hutchinson Cancer Center | Compositions and methods to program therapeutic cells using targeted nucleic acid nanocarriers |
| US11214789B2 (en) | 2016-05-03 | 2022-01-04 | Flodesign Sonics, Inc. | Concentration and washing of particles with acoustics |
| AU2017301609A1 (en) * | 2016-07-25 | 2019-02-21 | Bluebird Bio, Inc. | BCL11A homing endonuclease variants, compositions, and methods of use |
| 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 |
| CA3033327A1 (en) | 2016-08-09 | 2018-02-15 | President And Fellows Of Harvard College | Programmable cas9-recombinase fusion proteins and uses thereof |
| CA3034094A1 (en) | 2016-08-16 | 2018-02-22 | Bluebird Bio, Inc. | Il-10 receptor alpha homing endonuclease variants, compositions, and methods of use |
| EP3504335A4 (en) * | 2016-08-23 | 2020-04-15 | Bluebird Bio, Inc. | HOMING TIM3 ENDONUCLEASE VARIANTS, COMPOSITIONS AND METHODS OF USE |
| WO2018039438A1 (en) | 2016-08-24 | 2018-03-01 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
| CA3035455A1 (en) | 2016-09-08 | 2018-03-15 | Bluebird Bio, Inc. | Pd-1 homing endonuclease variants, compositions, and methods of use |
| MX2019004156A (es) * | 2016-10-11 | 2019-09-26 | Bluebird Bio Inc | Variantes de endonucleasa homing de receptor de linfocitos t alfa (tcralpha). |
| WO2018071868A1 (en) | 2016-10-14 | 2018-04-19 | President And Fellows Of Harvard College | Aav delivery of nucleobase editors |
| AU2017346683B2 (en) * | 2016-10-17 | 2024-04-18 | Regeneron Pharmaceuticals, Inc. | TGFβR2 endonuclease variants, compositions, and methods of use |
| WO2018073393A2 (en) | 2016-10-19 | 2018-04-26 | Cellectis | Tal-effector nuclease (talen) -modified allogenic cells suitable for therapy |
| IL266892B2 (en) | 2016-12-02 | 2025-10-01 | Univ Southern California | Artificial immune receptors and methods of using them |
| US10745677B2 (en) | 2016-12-23 | 2020-08-18 | President And Fellows Of Harvard College | Editing of CCR5 receptor gene to protect against HIV infection |
| CN110121336A (zh) | 2017-01-05 | 2019-08-13 | 弗莱德哈钦森癌症研究中心 | 改善疫苗功效的系统和方法 |
| US12390514B2 (en) | 2017-03-09 | 2025-08-19 | President And Fellows Of Harvard College | Cancer vaccine |
| EP3592853A1 (en) | 2017-03-09 | 2020-01-15 | President and Fellows of Harvard College | Suppression of pain by gene editing |
| 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 |
| CN110832075A (zh) | 2017-03-22 | 2020-02-21 | 诺华股份有限公司 | 用于免疫肿瘤学的组合物和方法 |
| KR20240116572A (ko) | 2017-03-23 | 2024-07-29 | 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 | 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제 |
| CA3059643A1 (en) | 2017-04-13 | 2018-10-18 | Cellectis | New sequence specific reagents targeting ccr5 in primary hematopoietic cells |
| 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 |
| JP7191042B2 (ja) * | 2017-05-25 | 2022-12-16 | ２セブンティ バイオ インコーポレイテッド | Ｃｂｌｂエンドヌクレアーゼバリアント、組成物、および使用方法 |
| CA3068465A1 (en) | 2017-06-30 | 2019-01-03 | Precision Biosciences, Inc. | Genetically-modified t cells comprising a modified intron in the t cell receptor alpha gene |
| EP3658573A1 (en) | 2017-07-28 | 2020-06-03 | President and Fellows of Harvard College | Methods and compositions for evolving base editors using phage-assisted continuous evolution (pace) |
| WO2019139645A2 (en) | 2017-08-30 | 2019-07-18 | President And Fellows Of Harvard College | High efficiency base editors comprising gam |
| WO2019070974A1 (en) * | 2017-10-04 | 2019-04-11 | Bluebird Bio, Inc. | PCSK9 ENDONUCLEASE VARIANTS, COMPOSITIONS AND METHODS OF USE |
| EP3702453A4 (en) * | 2017-10-10 | 2021-07-21 | Hiroshima University | TOTAL REPLACEMENT TECHNIQUE FOR T-LYMPHOCYTE RECEPTOR USING PLATINUM TALENE |
| TWI814745B (zh) | 2017-10-10 | 2023-09-11 | 國立大學法人廣島大學 | 使用效應T細胞(Teff)抗原受體之抗原特異性控制性T細胞(Treg)之製出技術 |
| AU2018352592C1 (en) | 2017-10-16 | 2025-09-25 | Beam Therapeutics, Inc. | Uses of adenosine base editors |
| JP2021507561A (ja) | 2017-12-14 | 2021-02-22 | フロデザイン ソニックス， インク．Ｆｌｏｄｅｓｉｇｎ Ｓｏｎｉｃｓ， Ｉｎｃ． | 音響トランスデューサドライバ及びコントローラ |
| EP3724214A4 (en) | 2017-12-15 | 2021-09-01 | The Broad Institute Inc. | SYSTEMS AND METHODS FOR PREDICTING REPAIR RESULTS IN GENETIC ENGINEERING |
| US20210002621A1 (en) * | 2017-12-20 | 2021-01-07 | Bluebird Bio, Inc. | Ctla4 homing endonuclease variants, compositions, and methods of use |
| IL277883B2 (en) | 2018-04-12 | 2023-12-01 | Prec Biosciences Inc | Optimized engineered nucleases having specificity for the human t cell receptor alpha constant region gene |
| US12157760B2 (en) | 2018-05-23 | 2024-12-03 | The Broad Institute, Inc. | Base editors and uses thereof |
| AU2019287720B2 (en) | 2018-06-14 | 2025-08-28 | Regeneron Pharmaceuticals, Inc. | CD79A chimeric antigen receptors |
| WO2020072059A1 (en) | 2018-10-04 | 2020-04-09 | Bluebird Bio, Inc. | Cblb endonuclease variants, compositions, and methods of use |
| US12331320B2 (en) | 2018-10-10 | 2025-06-17 | The Research Foundation For The State University Of New York | Genome edited cancer cell vaccines |
| US12281338B2 (en) | 2018-10-29 | 2025-04-22 | The Broad Institute, Inc. | Nucleobase editors comprising GeoCas9 and uses thereof |
| US12404500B2 (en) | 2018-12-10 | 2025-09-02 | Novo Nordisk A/S | Homing endonuclease variants |
| US12351837B2 (en) | 2019-01-23 | 2025-07-08 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| GB2601617B (en) | 2019-03-19 | 2024-02-21 | Broad Inst Inc | Methods and compositions for editing nucleotide sequences |
| KR20240122580A (ko) | 2019-04-03 | 2024-08-12 | 프리시젼 바이오사이언시스 인코포레이티드 | 마이크로RNA-적응 shRNA(shRNAmiR)를 포함하는 유전자-변형 면역 세포 |
| US12473543B2 (en) | 2019-04-17 | 2025-11-18 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
| EP4004216A1 (en) | 2019-07-25 | 2022-06-01 | Precision BioSciences, Inc. | Compositions and methods for sequential stacking of nucleic acid sequences into a genomic locus |
| CA3148179A1 (en) | 2019-08-20 | 2021-02-25 | Bruce J. Mccreedy Jr. | Lymphodepletion dosing regimens for cellular immunotherapies |
| US12435330B2 (en) | 2019-10-10 | 2025-10-07 | The Broad Institute, Inc. | Methods and compositions for prime editing RNA |
| US20220411479A1 (en) | 2019-10-30 | 2022-12-29 | Precision Biosciences, Inc. | Cd20 chimeric antigen receptors and methods of use for immunotherapy |
| 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 |
| WO2022035793A1 (en) | 2020-08-10 | 2022-02-17 | Precision Biosciences, Inc. | Antibodies and fragments specific for b-cell maturation antigen and uses thereof |
| JP2024503249A (ja) * | 2020-12-21 | 2024-01-25 | ２セブンティ バイオ インコーポレイテッド | 部位特異的変異導入のための組成物及び方法 |
| US20250127811A1 (en) | 2021-01-28 | 2025-04-24 | Precision Biosciences, Inc. | Modulation of tgf beta signaling in genetically-modified eukaryotic cells |
| WO2023081756A1 (en) | 2021-11-03 | 2023-05-11 | The J. David Gladstone Institutes, A Testamentary Trust Established Under The Will Of J. David Gladstone | Precise genome editing using retrons |
| WO2023081767A1 (en) | 2021-11-05 | 2023-05-11 | Precision Biosciences, Inc. | Methods for immunotherapy |
| MX2024006051A (es) | 2021-11-23 | 2024-06-26 | Cellectis Sa | Nuevos andamios de proteinas tale con relaciones de actividad en objetivo/fuera de objetivo mejoradas. |
| WO2023141602A2 (en) | 2022-01-21 | 2023-07-27 | Renagade Therapeutics Management Inc. | Engineered retrons and methods of use |
| WO2024044723A1 (en) | 2022-08-25 | 2024-02-29 | Renagade Therapeutics Management Inc. | Engineered retrons and methods of use |
| EP4646223A1 (en) | 2023-01-05 | 2025-11-12 | Precision Biosciences, Inc. | Optimized engineered meganucleases having specificity for the human t cell receptor alpha constant region gene |
Family Cites Families (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US8956828B2 (en) * | 2009-11-10 | 2015-02-17 | Sangamo Biosciences, Inc. | Targeted disruption of T cell receptor genes using engineered zinc finger protein nucleases |
| US20140148361A1 (en) * | 2010-06-07 | 2014-05-29 | Barry L. Stoddard | Generation and Expression of Engineered I-ONUI Endonuclease and Its Homologues and Uses Thereof |
| EP3320910A1 (en) * | 2011-04-05 | 2018-05-16 | Cellectis | Method for the generation of compact tale-nucleases and uses thereof |
| WO2013009525A1 (en) * | 2011-07-08 | 2013-01-17 | Cellectis S.A. | Method for increasing the efficiency of double-strand break-induced mutagenssis |
| WO2013068845A2 (en) * | 2011-11-07 | 2013-05-16 | The University Of Western Ontario | Endonuclease for genome editing |
-
2014
- 2014-05-28 AU AU2014273091A patent/AU2014273091B2/en active Active
- 2014-05-28 ES ES14728899T patent/ES2716867T3/es active Active
- 2014-05-28 US US14/891,216 patent/US10000746B2/en active Active
- 2014-05-28 JP JP2016516173A patent/JP6450371B2/ja active Active
- 2014-05-28 EP EP14728899.7A patent/EP3004338B1/en active Active
- 2014-05-28 WO PCT/EP2014/061189 patent/WO2014191527A1/en not_active Ceased
- 2014-05-28 DK DK14728899.7T patent/DK3004338T3/en active
- 2014-05-28 CA CA2913872A patent/CA2913872C/en active Active
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2016520320A5 (cg-RX-API-DMAC7.html) | ||
| JP2016520319A5 (cg-RX-API-DMAC7.html) | ||
| US11155796B2 (en) | Compositions and methods for epigenome editing | |
| JP2025065213A (ja) | 免疫療法のための改変ナチュラルキラー（ｎｋ）細胞 | |
| JP2017018125A5 (cg-RX-API-DMAC7.html) | ||
| JP2023002785A (ja) | 変異ｃａｓ９タンパク質 | |
| BR112019016205A2 (pt) | composições e métodos para o tratamento de hemoglobinopatias | |
| WO2013186563A3 (en) | Factor viii sequences | |
| JP2015533786A5 (cg-RX-API-DMAC7.html) | ||
| WO2017011773A3 (en) | Codon-optimized nucleic acids encoding antibodies | |
| JP2020530437A5 (cg-RX-API-DMAC7.html) | ||
| FI3352776T3 (fi) | Htt-repressoreita ja niiden käyttötapoja | |
| CA3009727A1 (en) | Compositions and methods for the treatment of hemoglobinopathies | |
| JP2015503535A5 (cg-RX-API-DMAC7.html) | ||
| JP2021521855A (ja) | ヘモグロビン関連変異の編集のための相同組換え修復用鋳型の設計およびその送達 | |
| JP2017513472A5 (cg-RX-API-DMAC7.html) | ||
| US12297426B2 (en) | DNA damage response signature guided rational design of CRISPR-based systems and therapies | |
| US20240279629A1 (en) | Crispr-transposon systems for dna modification | |
| SG170819A1 (en) | Methods for the production of functional protein from dna having a nonsense mutation and the treatment of disorders associated therewith | |
| US11072782B2 (en) | Construct for epigenetic modification and its use in the silencing of genes | |
| CA3191148A1 (en) | Engineered immune cells with priming receptors | |
| EA201070108A1 (ru) | Cd44 сплайс-варианты в нейродегенеративных заболеваниях | |
| AU2021369494A9 (en) | Safe harbor loci | |
| KR20250134148A (ko) | Scn9a 또는 scn10a 유전자를 변형시키기 위한 유전자-편집 시스템 및 이의 방법 및 용도 | |
| WO2016086768A1 (zh) | 特异识别并修复β地中海贫血症beta-globin基因的嵌合核酸酶 |