JP2019525759A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2019525759A5 JP2019525759A5 JP2019503701A JP2019503701A JP2019525759A5 JP 2019525759 A5 JP2019525759 A5 JP 2019525759A5 JP 2019503701 A JP2019503701 A JP 2019503701A JP 2019503701 A JP2019503701 A JP 2019503701A JP 2019525759 A5 JP2019525759 A5 JP 2019525759A5
- Authority
- JP
- Japan
- Prior art keywords
- amino acid
- seq
- item
- nos
- cell
- 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.)
- Pending
Links
- 125000003275 alpha amino acid group Chemical group 0.000 claims description 88
- 108090000765 processed proteins & peptides Proteins 0.000 claims description 76
- 229920001184 polypeptide Polymers 0.000 claims description 70
- 102000004196 processed proteins & peptides Human genes 0.000 claims description 70
- 239000012634 fragment Substances 0.000 claims description 48
- 210000004027 cell Anatomy 0.000 claims description 38
- 238000006467 substitution reaction Methods 0.000 claims description 32
- 102220485790 Destrin_S40R_mutation Human genes 0.000 claims description 29
- 102220471474 Protein MON2 homolog_S36A_mutation Human genes 0.000 claims description 29
- 102220370154 c.104C>A Human genes 0.000 claims description 29
- 102200018963 rs35470366 Human genes 0.000 claims description 29
- 102200104655 rs11555566 Human genes 0.000 claims description 27
- 102200034226 rs6441 Human genes 0.000 claims description 27
- 102220559237 Voltage-dependent L-type calcium channel subunit alpha-1C_N32R_mutation Human genes 0.000 claims description 25
- 102200012954 rs121918642 Human genes 0.000 claims description 25
- 102220130981 rs774149422 Human genes 0.000 claims description 25
- 102000040430 polynucleotide Human genes 0.000 claims description 18
- 108091033319 polynucleotide Proteins 0.000 claims description 18
- 239000002157 polynucleotide Substances 0.000 claims description 18
- 102200081901 rs137854510 Human genes 0.000 claims description 17
- 102220256808 rs149398412 Human genes 0.000 claims description 17
- 102220645900 Protein pitchfork_T48R_mutation Human genes 0.000 claims description 12
- 101100493741 Homo sapiens BCL11A gene Proteins 0.000 claims description 11
- 102220064266 rs535608443 Human genes 0.000 claims description 11
- 230000004568 DNA-binding Effects 0.000 claims description 10
- 210000003958 hematopoietic stem cell Anatomy 0.000 claims description 9
- 102000004190 Enzymes Human genes 0.000 claims description 7
- 108090000790 Enzymes Proteins 0.000 claims description 7
- 108060002716 Exonuclease Proteins 0.000 claims description 7
- 102000013165 exonuclease Human genes 0.000 claims description 7
- 102220631830 AH receptor-interacting protein_E42R_mutation Human genes 0.000 claims description 5
- 230000002159 abnormal effect Effects 0.000 claims description 5
- 102220097833 rs876660426 Human genes 0.000 claims description 5
- 239000013598 vector Substances 0.000 claims description 5
- 102000016928 DNA-directed DNA polymerase Human genes 0.000 claims description 4
- 108010014303 DNA-directed DNA polymerase Proteins 0.000 claims description 4
- 101000903703 Homo sapiens B-cell lymphoma/leukemia 11A Proteins 0.000 claims description 4
- 208000002903 Thalassemia Diseases 0.000 claims description 4
- 201000006288 alpha thalassemia Diseases 0.000 claims description 4
- 208000005980 beta thalassemia Diseases 0.000 claims description 4
- 208000007056 sickle cell anemia Diseases 0.000 claims description 4
- 208000024891 symptom Diseases 0.000 claims description 4
- 102220559695 Differentially expressed in FDCP 8 homolog_V68R_mutation Human genes 0.000 claims description 3
- 102000004533 Endonucleases Human genes 0.000 claims description 3
- 108010042407 Endonucleases Proteins 0.000 claims description 3
- 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
- 102220543096 Protein pitchfork_T48V_mutation Human genes 0.000 claims description 3
- 102220559236 Voltage-dependent L-type calcium channel subunit alpha-1C_N32K_mutation Human genes 0.000 claims description 3
- 102220347004 c.89G>A Human genes 0.000 claims description 3
- 108020004999 messenger RNA Proteins 0.000 claims description 3
- 102200057181 rs1064793683 Human genes 0.000 claims description 3
- 102200070537 rs11214077 Human genes 0.000 claims description 3
- 102200005897 rs137852377 Human genes 0.000 claims description 3
- 102220139511 rs144140226 Human genes 0.000 claims description 3
- 102220049541 rs200283315 Human genes 0.000 claims description 3
- 102220223774 rs375605948 Human genes 0.000 claims description 3
- 102220139828 rs587782481 Human genes 0.000 claims description 3
- 102200001739 rs72552725 Human genes 0.000 claims description 3
- 101710145992 B-cell lymphoma/leukemia 11A Proteins 0.000 claims description 2
- 102100022976 B-cell lymphoma/leukemia 11A Human genes 0.000 claims description 2
- 108010044495 Fetal Hemoglobin Proteins 0.000 claims description 2
- 108090000652 Flap endonucleases Proteins 0.000 claims description 2
- 102000004150 Flap endonucleases Human genes 0.000 claims description 2
- 108091005886 Hemoglobin subunit gamma Proteins 0.000 claims description 2
- 102100038617 Hemoglobin subunit gamma-2 Human genes 0.000 claims description 2
- 108060004795 Methyltransferase Proteins 0.000 claims description 2
- 239000002299 complementary DNA Substances 0.000 claims description 2
- 230000001419 dependent effect Effects 0.000 claims description 2
- 230000000694 effects Effects 0.000 claims description 2
- 108090000623 proteins and genes Proteins 0.000 claims description 2
- 230000003612 virological effect Effects 0.000 claims description 2
- 102220224000 rs1060502077 Human genes 0.000 claims 6
- 102220536706 Hemoglobin subunit epsilon_K34R_mutation Human genes 0.000 claims 1
- 208000034737 hemoglobinopathy Diseases 0.000 claims 1
- 230000002265 prevention Effects 0.000 claims 1
- 238000000034 method Methods 0.000 description 40
- 102220256728 rs545107676 Human genes 0.000 description 7
- 108091005904 Hemoglobin subunit beta Proteins 0.000 description 6
- 230000005782 double-strand break Effects 0.000 description 6
- 102100021519 Hemoglobin subunit beta Human genes 0.000 description 5
- 241000702423 Adeno-associated virus - 2 Species 0.000 description 3
- 125000001433 C-terminal amino-acid group Chemical group 0.000 description 3
- 125000001429 N-terminal alpha-amino-acid group Chemical group 0.000 description 3
- 241000700605 Viruses Species 0.000 description 3
- HCHKCACWOHOZIP-UHFFFAOYSA-N Zinc Chemical compound [Zn] HCHKCACWOHOZIP-UHFFFAOYSA-N 0.000 description 3
- 239000011701 zinc Substances 0.000 description 3
- 229910052725 zinc Inorganic materials 0.000 description 3
- 241000972680 Adeno-associated virus - 6 Species 0.000 description 2
- 102220085953 rs864622049 Human genes 0.000 description 2
- 241001430294 unidentified retrovirus Species 0.000 description 2
- 239000013603 viral vector Substances 0.000 description 2
- 241001655883 Adeno-associated virus - 1 Species 0.000 description 1
- 241000202702 Adeno-associated virus - 3 Species 0.000 description 1
- 241000580270 Adeno-associated virus - 4 Species 0.000 description 1
- 241001634120 Adeno-associated virus - 5 Species 0.000 description 1
- 241001164823 Adeno-associated virus - 7 Species 0.000 description 1
- 241001164825 Adeno-associated virus - 8 Species 0.000 description 1
- 241000649045 Adeno-associated virus 10 Species 0.000 description 1
- 241000702421 Dependoparvovirus Species 0.000 description 1
- 101100162704 Emericella nidulans I-AniI gene Proteins 0.000 description 1
- 102100034343 Integrase Human genes 0.000 description 1
- 108010061833 Integrases Proteins 0.000 description 1
- 230000027455 binding Effects 0.000 description 1
- 230000002950 deficient Effects 0.000 description 1
- 230000011559 double-strand break repair via nonhomologous end joining Effects 0.000 description 1
- 238000010362 genome editing Methods 0.000 description 1
Applications Claiming Priority (9)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662366530P | 2016-07-25 | 2016-07-25 | |
| US62/366,530 | 2016-07-25 | ||
| US201662367465P | 2016-07-27 | 2016-07-27 | |
| US62/367,465 | 2016-07-27 | ||
| US201662375829P | 2016-08-16 | 2016-08-16 | |
| US62/375,829 | 2016-08-16 | ||
| US201662414273P | 2016-10-28 | 2016-10-28 | |
| US62/414,273 | 2016-10-28 | ||
| PCT/US2017/043726 WO2018022619A1 (en) | 2016-07-25 | 2017-07-25 | Bcl11a homing endonuclease variants, compositions, and methods of use |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2019525759A JP2019525759A (ja) | 2019-09-12 |
| JP2019525759A5 true JP2019525759A5 (enExample) | 2020-09-03 |
Family
ID=61017227
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019503701A Pending JP2019525759A (ja) | 2016-07-25 | 2017-07-25 | Ｂｃｌ１１ａホーミングエンドヌクレアーゼバリアント、組成物、および使用方法 |
Country Status (7)
| Country | Link |
|---|---|
| US (1) | US20190184035A1 (enExample) |
| EP (1) | EP3487994A4 (enExample) |
| JP (1) | JP2019525759A (enExample) |
| CN (1) | CN109689865A (enExample) |
| AU (1) | AU2017301609A1 (enExample) |
| CA (1) | CA3031785A1 (enExample) |
| WO (1) | WO2018022619A1 (enExample) |
Families Citing this family (25)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US9458450B2 (en) | 2012-03-15 | 2016-10-04 | Flodesign Sonics, Inc. | Acoustophoretic separation technology using multi-dimensional standing waves |
| US10967298B2 (en) | 2012-03-15 | 2021-04-06 | Flodesign Sonics, Inc. | Driver and control for variable impedence load |
| US9950282B2 (en) | 2012-03-15 | 2018-04-24 | Flodesign Sonics, Inc. | Electronic configuration and control for acoustic standing wave generation |
| US10704021B2 (en) | 2012-03-15 | 2020-07-07 | Flodesign Sonics, Inc. | Acoustic perfusion devices |
| WO2015105955A1 (en) | 2014-01-08 | 2015-07-16 | Flodesign Sonics, Inc. | Acoustophoresis device with dual acoustophoretic chamber |
| US11377651B2 (en) | 2016-10-19 | 2022-07-05 | Flodesign Sonics, Inc. | Cell therapy processes utilizing acoustophoresis |
| US11021699B2 (en) | 2015-04-29 | 2021-06-01 | FioDesign Sonics, Inc. | Separation using angled acoustic waves |
| US11708572B2 (en) | 2015-04-29 | 2023-07-25 | Flodesign Sonics, Inc. | Acoustic cell separation techniques and processes |
| US11474085B2 (en) | 2015-07-28 | 2022-10-18 | Flodesign Sonics, Inc. | Expanded bed affinity selection |
| US11459540B2 (en) | 2015-07-28 | 2022-10-04 | Flodesign Sonics, Inc. | Expanded bed affinity selection |
| US11214789B2 (en) | 2016-05-03 | 2022-01-04 | Flodesign Sonics, Inc. | Concentration and washing of particles with acoustics |
| US11085035B2 (en) | 2016-05-03 | 2021-08-10 | Flodesign Sonics, Inc. | Therapeutic cell washing, concentration, and separation utilizing acoustophoresis |
| KR102622910B1 (ko) | 2016-09-08 | 2024-01-10 | 2세븐티 바이오, 인코포레이티드 | Pd-1 호밍 엔도뉴클레아제 변이체, 조성물 및 사용 방법 |
| WO2018075541A1 (en) | 2016-10-17 | 2018-04-26 | Bluebird Bio, Inc. | TGFβR2 ENDONUCLEASE VARIANTS, COMPOSITIONS, AND METHODS OF USE |
| EP3529347A1 (en) | 2016-10-19 | 2019-08-28 | Flodesign Sonics, Inc. | Affinity cell extraction by acoustics |
| TW201839136A (zh) | 2017-02-06 | 2018-11-01 | 瑞士商諾華公司 | 治療血色素異常症之組合物及方法 |
| CN110799644A (zh) | 2017-05-25 | 2020-02-14 | 蓝鸟生物公司 | Cblb核酸内切酶变体、组合物及使用方法 |
| KR20220066413A (ko) | 2017-12-14 | 2022-05-24 | 프로디자인 소닉스, 인크. | 음향 트랜스듀서 구동기 및 제어기 |
| EP3784790A4 (en) * | 2018-04-27 | 2022-02-09 | Seattle Children's Hospital d/b/a Seattle Children's Research Institute | BRUTON TYROSINE KINASE HOMING ENDONUCLEAR VARIANTS, COMPOSITIONS AND METHOD OF USE |
| WO2020072059A1 (en) | 2018-10-04 | 2020-04-09 | Bluebird Bio, Inc. | Cblb endonuclease variants, compositions, and methods of use |
| US12404500B2 (en) | 2018-12-10 | 2025-09-02 | Novo Nordisk A/S | Homing endonuclease variants |
| US20220364123A1 (en) * | 2019-04-24 | 2022-11-17 | Seattle Children's Hospital D/B/A Seattle Children's Research Institute | Wiskott-aldrich syndrome gene homing endonuclease variants, compositions, and methods of use |
| WO2025128871A2 (en) | 2023-12-13 | 2025-06-19 | Renagade Therapeutics Management Inc. | Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents |
| WO2025153530A1 (en) * | 2024-01-16 | 2025-07-24 | Novo Nordisk A/S | Albumin-targeted endonucleases, compositions, and methods of use |
| WO2025174765A1 (en) | 2024-02-12 | 2025-08-21 | Renagade Therapeutics Management Inc. | Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents |
Family Cites Families (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2009095742A1 (en) * | 2008-01-31 | 2009-08-06 | Cellectis | New i-crei derived single-chain meganuclease and uses thereof |
| WO2009059195A2 (en) * | 2007-10-31 | 2009-05-07 | Precision Biosciences | Rationally-designed single-chain meganucleases with non-palindromic recognition sequences |
| WO2011156430A2 (en) * | 2010-06-07 | 2011-12-15 | Fred Hutchinson Cancer Research Center | Generation and expression of engineered i-onui endonuclease and its homologues and uses thereof |
| EP3272356A1 (en) * | 2012-02-24 | 2018-01-24 | Fred Hutchinson Cancer Research Center | Compositions and methods for the treatment of hemoglobinopathies |
| DK3004338T3 (en) * | 2013-05-31 | 2019-04-08 | Cellectis Sa | LAGLIDADG HOMING ENDONUCLEASE DIVERSE T-CELL RECEPTOR ALPHA GENET AND APPLICATIONS THEREOF |
| JP6488283B2 (ja) * | 2013-05-31 | 2019-03-20 | セレクティスＣｅｌｌｅｃｔｉｓ | Ｃ−ｃケモカイン受容体５型（ｃｃｒ５）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 |
-
2017
- 2017-07-25 EP EP17835117.7A patent/EP3487994A4/en not_active Withdrawn
- 2017-07-25 CN CN201780054710.4A patent/CN109689865A/zh active Pending
- 2017-07-25 WO PCT/US2017/043726 patent/WO2018022619A1/en not_active Ceased
- 2017-07-25 AU AU2017301609A patent/AU2017301609A1/en not_active Abandoned
- 2017-07-25 CA CA3031785A patent/CA3031785A1/en not_active Abandoned
- 2017-07-25 JP JP2019503701A patent/JP2019525759A/ja active Pending
- 2017-07-25 US US16/320,280 patent/US20190184035A1/en not_active Abandoned
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2019525759A5 (enExample) | ||
| IL292999A (en) | Compositions and methods of treating amyotrophic lateral sclerosis (als) | |
| US11773409B2 (en) | CRISPR/Cas 9-mediated integration of polynucleotides by sequential homologous recombination of AAV donor vectors | |
| US20210017509A1 (en) | Gene Editing for Autosomal Dominant Diseases | |
| FI3313991T3 (fi) | Muunnettu tekijä IX sekä koostumukset, menetelmät ja käytöt geeninsiirtoa varten soluihin, elimiin ja kudoksiin | |
| JP7575950B2 (ja) | 遺伝子編集されたｃｄ３４＋細胞におけるｆｏｘｐ３の発現 | |
| JP2017535266A5 (enExample) | ||
| JP2018522529A5 (enExample) | ||
| JP2021505133A5 (enExample) | ||
| AU2019250249A1 (en) | Promoters, expression cassettes, vectors, kits, and methods for the treatment of achromatopsia and other diseases | |
| JP2021522273A (ja) | Ｃｎｓ変性のための遺伝子治療法 | |
| JP2019513393A5 (enExample) | ||
| JP2018516978A5 (enExample) | ||
| JP2020500541A5 (enExample) | ||
| IL299325B1 (en) | Variants of acid alpha-glucosidase and their uses | |
| HRP20231077T1 (hr) | Genska terapija za pigmentni retinitis | |
| JP2020514286A5 (enExample) | ||
| JP2017510298A5 (enExample) | ||
| JP2020509786A5 (enExample) | ||
| FI3373980T3 (fi) | Menetelmiä lihasdystrofian hoitamiseksi | |
| JPWO2020214613A5 (enExample) | ||
| CN119403823A (zh) | 具有增强的肌肉转导效率的肽修饰的aav衣壳 | |
| WO2021127057A1 (en) | Methods and compositions for correction of dmd mutations | |
| US20240398985A1 (en) | Novel transcription factors | |
| US20240425877A1 (en) | Abca4 genome editing |