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 (OSRAM) | 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 (OSRAM) |
| EP (1) | EP3004338B1 (OSRAM) |
| JP (1) | JP6450371B2 (OSRAM) |
| AU (1) | AU2014273091B2 (OSRAM) |
| CA (1) | CA2913872C (OSRAM) |
| DK (1) | DK3004338T3 (OSRAM) |
| ES (1) | ES2716867T3 (OSRAM) |
| WO (1) | WO2014191527A1 (OSRAM) |
Families Citing this family (78)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA3111953C (en) * | 2011-04-05 | 2023-10-24 | Cellectis | Method for the generation of compact tale-nucleases and uses thereof |
| EP3613852A3 (en) | 2011-07-22 | 2020-04-22 | 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 |
| 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 |
| US9737604B2 (en) | 2013-09-06 | 2017-08-22 | President And Fellows Of Harvard College | Use of cationic lipids to deliver CAS9 |
| US9388430B2 (en) | 2013-09-06 | 2016-07-12 | President And Fellows Of Harvard College | Cas9-recombinase fusion proteins and uses thereof |
| US9228207B2 (en) | 2013-09-06 | 2016-01-05 | President And Fellows Of Harvard College | Switchable gRNAs comprising aptamers |
| 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 |
| US11053481B2 (en) | 2013-12-12 | 2021-07-06 | President And Fellows Of Harvard College | Fusions of Cas9 domains and nucleic acid-editing domains |
| CN105939767B (zh) | 2014-01-08 | 2018-04-06 | 弗洛设计声能学公司 | 具有双声电泳腔的声电泳装置 |
| AU2015298571B2 (en) | 2014-07-30 | 2020-09-03 | 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 |
| CA3001008A1 (en) * | 2015-10-05 | 2017-04-13 | Precision Biosciences, Inc. | Engineered meganucleases with recognition sequences found in the human t cell receptor alpha constant region gene |
| CN108601821B (zh) | 2015-10-05 | 2023-09-19 | 精密生物科学公司 | 包含经修饰的人T细胞受体α恒定区基因的经遗传修饰的细胞 |
| US12043852B2 (en) | 2015-10-23 | 2024-07-23 | President And Fellows Of Harvard College | Evolved Cas9 proteins for gene editing |
| HK1257295A1 (zh) | 2015-12-04 | 2019-10-18 | Novartis Ag | 用於免疫肿瘤学的组合物和方法 |
| US20190241910A1 (en) * | 2016-03-11 | 2019-08-08 | Bluebird Bio, Inc. | Genome edited immune effector cells |
| CN109121413A (zh) * | 2016-04-14 | 2019-01-01 | 弗莱德哈钦森癌症研究中心 | 使用靶向核酸纳米载体编程治疗性细胞的组合物和方法 |
| 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 |
| US11214789B2 (en) | 2016-05-03 | 2022-01-04 | Flodesign Sonics, Inc. | Concentration and washing of particles with acoustics |
| CN109689865A (zh) * | 2016-07-25 | 2019-04-26 | 蓝鸟生物公司 | Bcl11a归巢核酸内切酶变体、组合物和使用方法 |
| GB2568182A (en) | 2016-08-03 | 2019-05-08 | Harvard College | Adenosine nucleobase editors and uses thereof |
| WO2018031683A1 (en) | 2016-08-09 | 2018-02-15 | President And Fellows Of Harvard College | Programmable cas9-recombinase fusion proteins and uses thereof |
| US20190309274A1 (en) | 2016-08-16 | 2019-10-10 | Bluebird Bio, Inc. | Il-10 receptor alpha homing endonuclease variants, compositions, and methods of use |
| US20190262398A1 (en) * | 2016-08-23 | 2019-08-29 | Bluebird Bio, Inc. | Tim3 homing endonuclease variants, compositions, and methods of use |
| US11542509B2 (en) | 2016-08-24 | 2023-01-03 | President And Fellows Of Harvard College | Incorporation of unnatural amino acids into proteins using base editing |
| IL265045B2 (en) | 2016-09-08 | 2023-09-01 | Bluebird Bio Inc | Variants of endonuclease pd-1, compositions and methods of use |
| JP7104710B2 (ja) | 2016-10-11 | 2022-07-21 | ２セブンティ バイオ インコーポレイテッド | ＴＣＲαホーミングエンドヌクレアーゼバリアント |
| GB2573062A (en) | 2016-10-14 | 2019-10-23 | 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 |
| US12269859B2 (en) | 2016-12-02 | 2025-04-08 | Angeles Therapeutics, Inc. | Synthetic immune receptors and methods of use thereof |
| WO2018119359A1 (en) | 2016-12-23 | 2018-06-28 | President And Fellows Of Harvard College | Editing of ccr5 receptor gene to protect against hiv infection |
| JP7348063B2 (ja) | 2017-01-05 | 2023-09-20 | フレッド ハッチンソン キャンサー センター | ワクチン効力を改善するためのシステム及び方法 |
| US11898179B2 (en) | 2017-03-09 | 2024-02-13 | President And Fellows Of Harvard College | Suppression of pain by gene editing |
| WO2018165631A1 (en) | 2017-03-09 | 2018-09-13 | President And Fellows Of Harvard College | Cancer vaccine |
| CN110914310A (zh) | 2017-03-10 | 2020-03-24 | 哈佛大学的校长及成员们 | 胞嘧啶至鸟嘌呤碱基编辑器 |
| EP3601561A2 (en) | 2017-03-22 | 2020-02-05 | Novartis AG | Compositions and methods for immunooncology |
| US11268082B2 (en) | 2017-03-23 | 2022-03-08 | President And Fellows Of Harvard College | Nucleobase editors comprising nucleic acid programmable DNA binding proteins |
| WO2018189360A1 (en) | 2017-04-13 | 2018-10-18 | Cellectis | New sequence specific reagents targeting ccr5 in primary hematopoietic cells |
| 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 |
| AU2018273979B2 (en) * | 2017-05-25 | 2024-10-17 | Regeneron Pharmaceuticals, Inc. | CBLB endonuclease variants, compositions, and methods of use |
| WO2019005957A1 (en) | 2017-06-30 | 2019-01-03 | Precision Biosciences, Inc. | GENETICALLY MODIFIED T CELLS COMPRISING A MODIFIED INTRON IN THE ALPHA T CELL RECEPTOR GENE |
| JP2020534795A (ja) | 2017-07-28 | 2020-12-03 | 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 | ファージによって支援される連続的進化（ｐａｃｅ）を用いて塩基編集因子を進化させるための方法および組成物 |
| WO2019139645A2 (en) | 2017-08-30 | 2019-07-18 | President And Fellows Of Harvard College | High efficiency base editors comprising gam |
| US11779654B2 (en) * | 2017-10-04 | 2023-10-10 | 2Seventy Bio, Inc. | PCSK9 endonuclease variants, compositions, and methods of use |
| US11788076B2 (en) | 2017-10-10 | 2023-10-17 | Hiroshima University | Full replacement technique for T cell receptor using platinum TALEN |
| US11773152B2 (en) | 2017-10-10 | 2023-10-03 | Hiroshima University | Technique for creating antigen-specific regulatory T cells (Treg) in which effector T cell (Teff) antigen receptors are used |
| CA3082251A1 (en) | 2017-10-16 | 2019-04-25 | The Broad Institute, Inc. | Uses of adenosine base editors |
| EP3725092A4 (en) | 2017-12-14 | 2021-09-22 | FloDesign Sonics, Inc. | DRIVE AND CONTROL UNIT FOR ACOUSTIC CONVERTER |
| US12406749B2 (en) | 2017-12-15 | 2025-09-02 | The Broad Institute, Inc. | Systems and methods for predicting repair outcomes in genetic engineering |
| US20210002621A1 (en) * | 2017-12-20 | 2021-01-07 | Bluebird Bio, Inc. | Ctla4 homing endonuclease variants, compositions, and methods of use |
| WO2019200122A1 (en) | 2018-04-12 | 2019-10-17 | Precision 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 |
| EP4365194A3 (en) | 2018-06-14 | 2024-07-24 | 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 |
| WO2020092453A1 (en) | 2018-10-29 | 2020-05-07 | The Broad Institute, Inc. | Nucleobase editors comprising geocas9 and uses thereof |
| CA3122278A1 (en) | 2018-12-10 | 2020-06-18 | Bluebird Bio, Inc. | Homing endonuclease variants |
| US12351837B2 (en) | 2019-01-23 | 2025-07-08 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| AU2020240109A1 (en) | 2019-03-19 | 2021-09-30 | President And Fellows Of Harvard College | Methods and compositions for editing nucleotide sequences |
| IL316764A (en) | 2019-04-03 | 2025-01-01 | Prec Biosciences Inc | Genetically modified immune cells containing shRNA-targeted microRNA (shRNAmir) |
| WO2020214842A1 (en) | 2019-04-17 | 2020-10-22 | 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 |
| WO2021035054A1 (en) | 2019-08-20 | 2021-02-25 | Precision Biosciences, Inc. | 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 |
| WO2021087305A1 (en) | 2019-10-30 | 2021-05-06 | Precision Biosciences, Inc. | Cd20 chimeric antigen receptors and methods of use for immunotherapy |
| BR112022022603A2 (pt) | 2020-05-08 | 2023-01-17 | Broad Inst Inc | Métodos e composições para edição simultânea de ambas as fitas de sequência alvo de nucleotídeos de fita dupla |
| EP4192875A1 (en) | 2020-08-10 | 2023-06-14 | Precision BioSciences, Inc. | Antibodies and fragments specific for b-cell maturation antigen and uses thereof |
| CA3202889A1 (en) * | 2020-12-21 | 2022-06-30 | Jordan JARJOUR | Compositions and methods for site-directed mutagenesis |
| EP4284823A1 (en) | 2021-01-28 | 2023-12-06 | Precision BioSciences, Inc. | Modulation of tgf beta signaling in genetically-modified eukaryotic cells |
| US20250043269A1 (en) | 2021-11-03 | 2025-02-06 | The J. David Gladstone Institutes, a testamentary trust established under the Will of J. David Glad | Precise Genome Editing Using Retrons |
| WO2023081767A1 (en) | 2021-11-05 | 2023-05-11 | Precision Biosciences, Inc. | Methods for immunotherapy |
| KR20240110844A (ko) | 2021-11-23 | 2024-07-16 | 셀렉티스 에스.에이. | 개선된 온-타겟/오프-타겟 활성 비율을 갖는 신규 tale 단백질 스캐폴드 |
| 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 |
| CA3111953C (en) * | 2011-04-05 | 2023-10-24 | Cellectis | Method for the generation of compact tale-nucleases and uses thereof |
| US9540623B2 (en) * | 2011-07-08 | 2017-01-10 | Cellectis | Method for increasing the efficiency of double-strand-break induced mutagenesis |
| EP2776560A2 (en) * | 2011-11-07 | 2014-09-17 | The University of Western Ontario | Endonuclease for genome editing |
-
2014
- 2014-05-28 WO PCT/EP2014/061189 patent/WO2014191527A1/en not_active Ceased
- 2014-05-28 AU AU2014273091A patent/AU2014273091B2/en active Active
- 2014-05-28 JP JP2016516173A patent/JP6450371B2/ja active Active
- 2014-05-28 US US14/891,216 patent/US10000746B2/en active Active
- 2014-05-28 DK DK14728899.7T patent/DK3004338T3/en active
- 2014-05-28 ES ES14728899T patent/ES2716867T3/es active Active
- 2014-05-28 CA CA2913872A patent/CA2913872C/en active Active
- 2014-05-28 EP EP14728899.7A patent/EP3004338B1/en active Active
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2016520320A5 (OSRAM) | ||
| JP2016520319A5 (OSRAM) | ||
| 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 (OSRAM) | ||
| JP2023002785A (ja) | 変異ｃａｓ９タンパク質 | |
| TW202028466A (zh) | 編輯RNAs的方法以及組成物 | |
| BR112019016205A2 (pt) | composições e métodos para o tratamento de hemoglobinopatias | |
| WO2013186563A3 (en) | Factor viii sequences | |
| JP2015533786A5 (OSRAM) | ||
| WO2017011773A3 (en) | Codon-optimized nucleic acids encoding antibodies | |
| JP2020530437A5 (OSRAM) | ||
| FI3352776T3 (fi) | Htt-repressoreita ja niiden käyttötapoja | |
| JP2017513472A5 (OSRAM) | ||
| 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 | |
| AU2021369494A1 (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 | |
| US20240287609A1 (en) | Compositions and methods for large-scale in vivo genetic screening | |
| Suresh | CRISPR-Cas9 Based Correction of a Pathogenic Microduplication and Engineering Artificial Transcription Factors for Gene Activation: Applications in Genome Engineering |