JP2019513407A5 - - Google Patents

Download PDF

Info

Publication number
JP2019513407A5
JP2019513407A5 JP2018555646A JP2018555646A JP2019513407A5 JP 2019513407 A5 JP2019513407 A5 JP 2019513407A5 JP 2018555646 A JP2018555646 A JP 2018555646A JP 2018555646 A JP2018555646 A JP 2018555646A JP 2019513407 A5 JP2019513407 A5 JP 2019513407A5
Authority
JP
Japan
Prior art keywords
dna
modified
grna
bcl11a gene
gene
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
Application number
JP2018555646A
Other languages
English (en)
Japanese (ja)
Other versions
JP2019513407A (ja
JP6974349B2 (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/IB2017/000577 external-priority patent/WO2017182881A2/en
Publication of JP2019513407A publication Critical patent/JP2019513407A/ja
Publication of JP2019513407A5 publication Critical patent/JP2019513407A5/ja
Application granted granted Critical
Publication of JP6974349B2 publication Critical patent/JP6974349B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

JP2018555646A 2016-04-18 2017-04-18 ヘモグロビン異常症の処置のための材料及び方法 Active JP6974349B2 (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201662324024P 2016-04-18 2016-04-18
US62/324,024 2016-04-18
US201662382522P 2016-09-01 2016-09-01
US62/382,522 2016-09-01
US201662429428P 2016-12-02 2016-12-02
US62/429,428 2016-12-02
PCT/IB2017/000577 WO2017182881A2 (en) 2016-04-18 2017-04-18 Materials and methods for treatment of hemoglobinopathies

Publications (3)

Publication Number Publication Date
JP2019513407A JP2019513407A (ja) 2019-05-30
JP2019513407A5 true JP2019513407A5 (enExample) 2020-05-28
JP6974349B2 JP6974349B2 (ja) 2021-12-01

Family

ID=58993163

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2018555646A Active JP6974349B2 (ja) 2016-04-18 2017-04-18 ヘモグロビン異常症の処置のための材料及び方法

Country Status (25)

Country Link
US (5) US20200330609A1 (enExample)
EP (2) EP3445388B1 (enExample)
JP (1) JP6974349B2 (enExample)
KR (1) KR102351329B1 (enExample)
CN (1) CN109715198B (enExample)
AU (1) AU2017252023B2 (enExample)
BR (1) BR112018071321A2 (enExample)
CA (1) CA3021467A1 (enExample)
DK (1) DK3445388T3 (enExample)
ES (1) ES2979222T3 (enExample)
FI (1) FI3445388T3 (enExample)
HR (1) HRP20240657T1 (enExample)
HU (1) HUE066768T2 (enExample)
IL (1) IL262416B2 (enExample)
LT (1) LT3445388T (enExample)
MA (1) MA71705A (enExample)
MD (1) MD3445388T2 (enExample)
MX (1) MX2018012729A (enExample)
PL (1) PL3445388T3 (enExample)
PT (1) PT3445388T (enExample)
RS (1) RS65592B1 (enExample)
SG (2) SG11201809144XA (enExample)
SI (1) SI3445388T1 (enExample)
SM (1) SMT202400195T1 (enExample)
WO (1) WO2017182881A2 (enExample)

Families Citing this family (31)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12129471B2 (en) 2015-02-23 2024-10-29 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of human genetic diseases including hemoglobinopathies
US20180030438A1 (en) 2015-02-23 2018-02-01 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
WO2016182917A1 (en) * 2015-05-08 2016-11-17 Children's Medical Center Corporation Targeting bcl11a enhancer functional regions for fetal hemoglobin reinduction
EP3294896A1 (en) 2015-05-11 2018-03-21 Editas Medicine, Inc. Optimized crispr/cas9 systems and methods for gene editing in stem cells
MX382223B (es) 2015-05-12 2025-03-13 Sangamo Therapeutics Inc Regulacion de expresion genica mediada por nucleasa.
JP7396783B2 (ja) 2015-06-09 2023-12-12 エディタス・メディシン、インコーポレイテッド 移植を改善するためのcrispr/cas関連方法および組成物
ES2942309T3 (es) 2015-11-04 2023-05-31 Vertex Pharma Materiales y métodos para el tratamiento de hemoglobinopatías
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
WO2018218135A1 (en) 2017-05-25 2018-11-29 The Children's Medical Center Corporation Bcl11a guide delivery
WO2019014564A1 (en) 2017-07-14 2019-01-17 Editas Medicine, Inc. SYSTEMS AND METHODS OF TARGETED INTEGRATION AND GENOME EDITING AND DETECTION THEREOF WITH INTEGRATED PRIMING SITES
WO2019081982A1 (en) * 2017-10-26 2019-05-02 Crispr Therapeutics Ag SUBSTANCES AND METHODS FOR THE TREATMENT OF HEMOGLOBINOPATHIES
CN109722415B (zh) * 2017-10-27 2021-01-26 博雅辑因(北京)生物科技有限公司 一种造血干细胞的培养组合物、培养基以及造血干细胞的培养方法
SG11202005147WA (en) 2017-12-05 2020-06-29 Vertex Pharma Crispr-cas9 modified cd34+ human hematopoietic stem and progenitor cells and uses thereof
WO2019118516A1 (en) * 2017-12-11 2019-06-20 Editas Medicine, Inc. Cpf1-related methods and compositions for gene editing
US12098363B2 (en) * 2018-01-26 2024-09-24 The Children's Medical Center Corporation Targeting BCL11A distal regulatory elements with a CAS9-CAS9 fusion for fetal hemoglobin reinduction
EP3749767A1 (en) 2018-02-05 2020-12-16 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of hemoglobinopathies
US11268077B2 (en) 2018-02-05 2022-03-08 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of hemoglobinopathies
US12350284B2 (en) 2018-05-02 2025-07-08 The Children's Medical Center Corporation BCL11A microRNAs for treating hemoglobinopathies
CN113939586A (zh) * 2019-04-02 2022-01-14 桑格摩生物治疗股份有限公司 治疗β-地中海贫血症的方法
CN110117659B (zh) * 2019-06-18 2022-10-11 上海奕谱生物科技有限公司 一种新型的肿瘤标记物stamp-ep10及其应用
WO2021037232A1 (zh) * 2019-08-28 2021-03-04 甘李药业股份有限公司 编辑造血干/祖细胞中bcl11a基因的方法
JP2023549080A (ja) * 2020-10-30 2023-11-22 アーバー バイオテクノロジーズ, インコーポレイテッド Bcl11aを標的とするrnaガイドを含む組成物及びその使用
GB202017555D0 (en) * 2020-11-06 2020-12-23 Harbour Antibodies Bv Antibody-conjugated nanoparticles
CN116801913A (zh) * 2020-12-03 2023-09-22 斯克里贝治疗公司 用于靶向bcl11a的组合物和方法
WO2022206986A1 (en) * 2021-04-02 2022-10-06 Shanghaitech University Gene therapy for treating beta-hemoglobinopathies
EP4337232A4 (en) * 2021-05-13 2025-03-19 The Children's Medical Center Corporation Methods for stratifying subjects for fetal hemoglobin reinduction
EP4399307A2 (en) * 2021-09-08 2024-07-17 Flagship Pioneering Innovations VI, LLC Hbb-modulating compositions and methods
WO2024050450A1 (en) 2022-08-31 2024-03-07 Gigamune, Inc. Engineered enveloped vectors and methods of use thereof
WO2024073751A1 (en) 2022-09-29 2024-04-04 Vor Biopharma Inc. Methods and compositions for gene modification and enrichment
AU2023375263A1 (en) * 2022-11-02 2025-04-10 Incisive Genetics, Inc. Compositions and methods for preventing, ameliorating, or treating sickle cell disease and compositions and methods for disrupting genes and gene segments
WO2025094917A1 (ja) * 2023-10-30 2025-05-08 C4U株式会社 Bcl11a遺伝子のエンハンサーの一部又は全部が破壊された造血幹細胞の製造方法、及びその方法に用いるためのキット

Family Cites Families (139)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US3687808A (en) 1969-08-14 1972-08-29 Univ Leland Stanford Junior Synthetic polynucleotides
US4469863A (en) 1980-11-12 1984-09-04 Ts O Paul O P Nonionic nucleic acid alkyl and aryl phosphonates and processes for manufacture and use thereof
US5023243A (en) 1981-10-23 1991-06-11 Molecular Biosystems, Inc. Oligonucleotide therapeutic agent and method of making same
US4476301A (en) 1982-04-29 1984-10-09 Centre National De La Recherche Scientifique Oligonucleotides, a process for preparing the same and their application as mediators of the action of interferon
JPS5927900A (ja) 1982-08-09 1984-02-14 Wakunaga Seiyaku Kk 固定化オリゴヌクレオチド
FR2540122B1 (fr) 1983-01-27 1985-11-29 Centre Nat Rech Scient Nouveaux composes comportant une sequence d'oligonucleotide liee a un agent d'intercalation, leur procede de synthese et leur application
US4605735A (en) 1983-02-14 1986-08-12 Wakunaga Seiyaku Kabushiki Kaisha Oligonucleotide derivatives
US4948882A (en) 1983-02-22 1990-08-14 Syngene, Inc. Single-stranded labelled oligonucleotides, reactive monomers and methods of synthesis
US4824941A (en) 1983-03-10 1989-04-25 Julian Gordon Specific antibody to the native form of 2'5'-oligonucleotides, the method of preparation and the use as reagents in immunoassays or for binding 2'5'-oligonucleotides in biological systems
US4587044A (en) 1983-09-01 1986-05-06 The Johns Hopkins University Linkage of proteins to nucleic acids
US5118802A (en) 1983-12-20 1992-06-02 California Institute Of Technology DNA-reporter conjugates linked via the 2' or 5'-primary amino group of the 5'-terminal nucleoside
US5550111A (en) 1984-07-11 1996-08-27 Temple University-Of The Commonwealth System Of Higher Education Dual action 2',5'-oligoadenylate antiviral derivatives and uses thereof
US5258506A (en) 1984-10-16 1993-11-02 Chiron Corporation Photolabile reagents for incorporation into oligonucleotide chains
US5430136A (en) 1984-10-16 1995-07-04 Chiron Corporation Oligonucleotides having selectably cleavable and/or abasic sites
US5367066A (en) 1984-10-16 1994-11-22 Chiron Corporation Oligonucleotides with selectably cleavable and/or abasic sites
US4828979A (en) 1984-11-08 1989-05-09 Life Technologies, Inc. Nucleotide analogs for nucleic acid labeling and detection
FR2575751B1 (fr) 1985-01-08 1987-04-03 Pasteur Institut Nouveaux nucleosides de derives de l'adenosine, leur preparation et leurs applications biologiques
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US5405938A (en) 1989-12-20 1995-04-11 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US5185444A (en) 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
US5235033A (en) 1985-03-15 1993-08-10 Anti-Gene Development Group Alpha-morpholino ribonucleoside derivatives and polymers thereof
US4762779A (en) 1985-06-13 1988-08-09 Amgen Inc. Compositions and methods for functionalizing nucleic acids
US5317098A (en) 1986-03-17 1994-05-31 Hiroaki Shizuya Non-radioisotope tagging of fragments
JPS638396A (ja) 1986-06-30 1988-01-14 Wakunaga Pharmaceut Co Ltd ポリ標識化オリゴヌクレオチド誘導体
US5276019A (en) 1987-03-25 1994-01-04 The United States Of America As Represented By The Department Of Health And Human Services Inhibitors for replication of retroviruses and for the expression of oncogene products
US5264423A (en) 1987-03-25 1993-11-23 The United States Of America As Represented By The Department Of Health And Human Services Inhibitors for replication of retroviruses and for the expression of oncogene products
US4904582A (en) 1987-06-11 1990-02-27 Synthetic Genetics Novel amphiphilic nucleic acid conjugates
ATE113059T1 (de) 1987-06-24 1994-11-15 Florey Howard Inst Nukleosid-derivate.
US5585481A (en) 1987-09-21 1996-12-17 Gen-Probe Incorporated Linking reagents for nucleotide probes
US4924624A (en) 1987-10-22 1990-05-15 Temple University-Of The Commonwealth System Of Higher Education 2,',5'-phosphorothioate oligoadenylates and plant antiviral uses thereof
US5188897A (en) 1987-10-22 1993-02-23 Temple University Of The Commonwealth System Of Higher Education Encapsulated 2',5'-phosphorothioate oligoadenylates
US5525465A (en) 1987-10-28 1996-06-11 Howard Florey Institute Of Experimental Physiology And Medicine Oligonucleotide-polyamide conjugates and methods of production and applications of the same
DE3738460A1 (de) 1987-11-12 1989-05-24 Max Planck Gesellschaft Modifizierte oligonukleotide
US5082830A (en) 1988-02-26 1992-01-21 Enzo Biochem, Inc. End labeled nucleotide probe
JPH03503894A (ja) 1988-03-25 1991-08-29 ユニバーシィティ オブ バージニア アランミ パテンツ ファウンデイション オリゴヌクレオチド n‐アルキルホスホラミデート
US5278302A (en) 1988-05-26 1994-01-11 University Patents, Inc. Polynucleotide phosphorodithioates
US5109124A (en) 1988-06-01 1992-04-28 Biogen, Inc. Nucleic acid probe linked to a label having a terminal cysteine
US5216141A (en) 1988-06-06 1993-06-01 Benner Steven A Oligonucleotide analogs containing sulfur linkages
US5175273A (en) 1988-07-01 1992-12-29 Genentech, Inc. Nucleic acid intercalating agents
US5262536A (en) 1988-09-15 1993-11-16 E. I. Du Pont De Nemours And Company Reagents for the preparation of 5'-tagged oligonucleotides
US5512439A (en) 1988-11-21 1996-04-30 Dynal As Oligonucleotide-linked magnetic particles and uses thereof
US5599923A (en) 1989-03-06 1997-02-04 Board Of Regents, University Of Tx Texaphyrin metal complexes having improved functionalization
US5457183A (en) 1989-03-06 1995-10-10 Board Of Regents, The University Of Texas System Hydroxylated texaphyrins
US5391723A (en) 1989-05-31 1995-02-21 Neorx Corporation Oligonucleotide conjugates
US4958013A (en) 1989-06-06 1990-09-18 Northwestern University Cholesteryl modified oligonucleotides
US5451463A (en) 1989-08-28 1995-09-19 Clontech Laboratories, Inc. Non-nucleoside 1,3-diol reagents for labeling synthetic oligonucleotides
US5134066A (en) 1989-08-29 1992-07-28 Monsanto Company Improved probes using nucleosides containing 3-dezauracil analogs
US5254469A (en) 1989-09-12 1993-10-19 Eastman Kodak Company Oligonucleotide-enzyme conjugate that can be used as a probe in hybridization assays and polymerase chain reaction procedures
US5399676A (en) 1989-10-23 1995-03-21 Gilead Sciences Oligonucleotides with inverted polarity
US5264564A (en) 1989-10-24 1993-11-23 Gilead Sciences Oligonucleotide analogs with novel linkages
US5264562A (en) 1989-10-24 1993-11-23 Gilead Sciences, Inc. Oligonucleotide analogs with novel linkages
US5292873A (en) 1989-11-29 1994-03-08 The Research Foundation Of State University Of New York Nucleic acids labeled with naphthoquinone probe
US5177198A (en) 1989-11-30 1993-01-05 University Of N.C. At Chapel Hill Process for preparing oligoribonucleoside and oligodeoxyribonucleoside boranophosphates
US5130302A (en) 1989-12-20 1992-07-14 Boron Bilogicals, Inc. Boronated nucleoside, nucleotide and oligonucleotide compounds, compositions and methods for using same
US5486603A (en) 1990-01-08 1996-01-23 Gilead Sciences, Inc. Oligonucleotide having enhanced binding affinity
US5578718A (en) 1990-01-11 1996-11-26 Isis Pharmaceuticals, Inc. Thiol-derivatized nucleosides
US5681941A (en) 1990-01-11 1997-10-28 Isis Pharmaceuticals, Inc. Substituted purines and oligonucleotide cross-linking
US5587361A (en) 1991-10-15 1996-12-24 Isis Pharmaceuticals, Inc. Oligonucleotides having phosphorothioate linkages of high chiral purity
US5587470A (en) 1990-01-11 1996-12-24 Isis Pharmaceuticals, Inc. 3-deazapurines
US5459255A (en) 1990-01-11 1995-10-17 Isis Pharmaceuticals, Inc. N-2 substituted purines
AU7579991A (en) 1990-02-20 1991-09-18 Gilead Sciences, Inc. Pseudonucleosides and pseudonucleotides and their polymers
US5214136A (en) 1990-02-20 1993-05-25 Gilead Sciences, Inc. Anthraquinone-derivatives oligonucleotides
US5321131A (en) 1990-03-08 1994-06-14 Hybridon, Inc. Site-specific functionalization of oligodeoxynucleotides for non-radioactive labelling
US5470967A (en) 1990-04-10 1995-11-28 The Dupont Merck Pharmaceutical Company Oligonucleotide analogs with sulfamate linkages
ES2116977T3 (es) 1990-05-11 1998-08-01 Microprobe Corp Soportes solidos para ensayos de hibridacion de acidos nucleicos y metodos para inmovilizar oligonucleotidos de modo covalente.
US5602240A (en) 1990-07-27 1997-02-11 Ciba Geigy Ag. Backbone modified oligonucleotide analogs
DE69126530T2 (de) 1990-07-27 1998-02-05 Isis Pharmaceutical, Inc., Carlsbad, Calif. Nuklease resistente, pyrimidin modifizierte oligonukleotide, die die gen-expression detektieren und modulieren
US5489677A (en) 1990-07-27 1996-02-06 Isis Pharmaceuticals, Inc. Oligonucleoside linkages containing adjacent oxygen and nitrogen atoms
US5138045A (en) 1990-07-27 1992-08-11 Isis Pharmaceuticals Polyamine conjugated oligonucleotides
US5608046A (en) 1990-07-27 1997-03-04 Isis Pharmaceuticals, Inc. Conjugated 4'-desmethyl nucleoside analog compounds
US5618704A (en) 1990-07-27 1997-04-08 Isis Pharmacueticals, Inc. Backbone-modified oligonucleotide analogs and preparation thereof through radical coupling
US5541307A (en) 1990-07-27 1996-07-30 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogs and solid phase synthesis thereof
US5610289A (en) 1990-07-27 1997-03-11 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogues
US5218105A (en) 1990-07-27 1993-06-08 Isis Pharmaceuticals Polyamine conjugated oligonucleotides
US5623070A (en) 1990-07-27 1997-04-22 Isis Pharmaceuticals, Inc. Heteroatomic oligonucleoside linkages
US5688941A (en) 1990-07-27 1997-11-18 Isis Pharmaceuticals, Inc. Methods of making conjugated 4' desmethyl nucleoside analog compounds
US5677437A (en) 1990-07-27 1997-10-14 Isis Pharmaceuticals, Inc. Heteroatomic oligonucleoside linkages
KR100211552B1 (ko) 1990-08-03 1999-08-02 디. 꼬쉬 유전자 발현 억제용 화합물 및 방법
US5245022A (en) 1990-08-03 1993-09-14 Sterling Drug, Inc. Exonuclease resistant terminally substituted oligonucleotides
US5177196A (en) 1990-08-16 1993-01-05 Microprobe Corporation Oligo (α-arabinofuranosyl nucleotides) and α-arabinofuranosyl precursors thereof
US5512667A (en) 1990-08-28 1996-04-30 Reed; Michael W. Trifunctional intermediates for preparing 3'-tailed oligonucleotides
US5214134A (en) 1990-09-12 1993-05-25 Sterling Winthrop Inc. Process of linking nucleosides with a siloxane bridge
US5561225A (en) 1990-09-19 1996-10-01 Southern Research Institute Polynucleotide analogs containing sulfonate and sulfonamide internucleoside linkages
US5596086A (en) 1990-09-20 1997-01-21 Gilead Sciences, Inc. Modified internucleoside linkages having one nitrogen and two carbon atoms
US5432272A (en) 1990-10-09 1995-07-11 Benner; Steven A. Method for incorporating into a DNA or RNA oligonucleotide using nucleotides bearing heterocyclic bases
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
CA2095212A1 (en) 1990-11-08 1992-05-09 Sudhir Agrawal Incorporation of multiple reporter groups on synthetic oligonucleotides
US5714331A (en) 1991-05-24 1998-02-03 Buchardt, Deceased; Ole Peptide nucleic acids having enhanced binding affinity, sequence specificity and solubility
US5719262A (en) 1993-11-22 1998-02-17 Buchardt, Deceased; Ole Peptide nucleic acids having amino acid side chains
US5539082A (en) 1993-04-26 1996-07-23 Nielsen; Peter E. Peptide nucleic acids
US5371241A (en) 1991-07-19 1994-12-06 Pharmacia P-L Biochemicals Inc. Fluorescein labelled phosphoramidites
US5571799A (en) 1991-08-12 1996-11-05 Basco, Ltd. (2'-5') oligoadenylate analogues useful as inhibitors of host-v5.-graft response
TW393513B (en) 1991-11-26 2000-06-11 Isis Pharmaceuticals Inc Enhanced triple-helix and double-helix formation with oligomers containing modified pyrimidines
US5484908A (en) 1991-11-26 1996-01-16 Gilead Sciences, Inc. Oligonucleotides containing 5-propynyl pyrimidines
US5565552A (en) 1992-01-21 1996-10-15 Pharmacyclics, Inc. Method of expanded porphyrin-oligonucleotide conjugate synthesis
US5595726A (en) 1992-01-21 1997-01-21 Pharmacyclics, Inc. Chromophore probe for detection of nucleic acid
US5633360A (en) 1992-04-14 1997-05-27 Gilead Sciences, Inc. Oligonucleotide analogs capable of passive cell membrane permeation
US5434257A (en) 1992-06-01 1995-07-18 Gilead Sciences, Inc. Binding compentent oligomers containing unsaturated 3',5' and 2',5' linkages
US5272250A (en) 1992-07-10 1993-12-21 Spielvogel Bernard F Boronated phosphoramidate compounds
US5574142A (en) 1992-12-15 1996-11-12 Microprobe Corporation Peptide linkers for improved oligonucleotide delivery
US5476925A (en) 1993-02-01 1995-12-19 Northwestern University Oligodeoxyribonucleotides including 3'-aminonucleoside-phosphoramidate linkages and terminal 3'-amino groups
GB9304618D0 (en) 1993-03-06 1993-04-21 Ciba Geigy Ag Chemical compounds
JPH08508491A (ja) 1993-03-31 1996-09-10 スターリング ウインスロップ インコーポレイティド ホスホジエステル結合をアミド結合に置き換えたオリゴヌクレオチド
US5502177A (en) 1993-09-17 1996-03-26 Gilead Sciences, Inc. Pyrimidine derivatives for labeled binding partners
ES2216005T3 (es) 1993-11-09 2004-10-16 Targeted Genetics Corporation Produccion de titulos elevados de vectores de aav recombinantes.
EP0728214B1 (en) 1993-11-09 2004-07-28 Medical College Of Ohio Stable cell lines capable of expressing the adeno-associated virus replication gene
US5457187A (en) 1993-12-08 1995-10-10 Board Of Regents University Of Nebraska Oligonucleotides containing 5-fluorouracil
US5596091A (en) 1994-03-18 1997-01-21 The Regents Of The University Of California Antisense oligonucleotides comprising 5-aminoalkyl pyrimidine nucleotides
US5625050A (en) 1994-03-31 1997-04-29 Amgen Inc. Modified oligonucleotides and intermediates useful in nucleic acid therapeutics
US5525711A (en) 1994-05-18 1996-06-11 The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services Pteridine nucleotide analogs as fluorescent DNA probes
US5658785A (en) 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
US5597696A (en) 1994-07-18 1997-01-28 Becton Dickinson And Company Covalent cyanine dye oligonucleotide conjugates
US5580731A (en) 1994-08-25 1996-12-03 Chiron Corporation N-4 modified pyrimidine deoxynucleotides and oligonucleotide probes synthesized therewith
US5856152A (en) 1994-10-28 1999-01-05 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV vector and methods of use therefor
AU707866B2 (en) 1994-12-06 1999-07-22 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant AAV vectors
FR2737730B1 (fr) 1995-08-10 1997-09-05 Pasteur Merieux Serums Vacc Procede de purification de virus par chromatographie
CA2625279A1 (en) 1995-08-30 1997-03-06 Genzyme Corporation Chromatographic purification of adenovirus and aav
ES2317646T3 (es) 1995-09-08 2009-04-16 Genzyme Corporation Vectores aav mejorados para terapia genica.
US5910434A (en) 1995-12-15 1999-06-08 Systemix, Inc. Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant
ES2399232T3 (es) 1997-09-05 2013-03-26 Genzyme Corporation Procedimientos para la generación de preparaciones de vectores de AAV recombinantes de títulos altos exentas de auxiliares
US6258595B1 (en) 1999-03-18 2001-07-10 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
US6287860B1 (en) 2000-01-20 2001-09-11 Isis Pharmaceuticals, Inc. Antisense inhibition of MEKK2 expression
AU2001255575B2 (en) 2000-04-28 2006-08-31 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with aav5 capsids and aav5 vectors pseudotyped in heterologous capsids
US20030158403A1 (en) 2001-07-03 2003-08-21 Isis Pharmaceuticals, Inc. Nuclease resistant chimeric oligonucleotides
US9209196B2 (en) 2011-11-30 2015-12-08 Sharp Kabushiki Kaisha Memory circuit, method of driving the same, nonvolatile storage device using the same, and liquid crystal display device
BR112014020625A2 (pt) * 2012-02-24 2017-07-04 Hutchinson Fred Cancer Res polinucleotídeo, polipeptídeo, composição, célula e célula tronco editada por genoma
DE102012007232B4 (de) 2012-04-07 2014-03-13 Susanne Weller Verfahren zur Herstellung von rotierenden elektrischen Maschinen
RS59199B1 (sr) 2012-05-25 2019-10-31 Univ California Metode i jedinjenja za rnk-upravljanu ciljanu dnk modifikaciju i za rnk- upravljanu modulaciju transkripta
DK2925864T3 (en) * 2012-11-27 2019-02-11 Childrens Medical Ct Corp DIRECTIONAL TARGETING OF DISTANT BCL11A CONTROLS FOR FETAL HEMOGLOBIN REINUCTION
WO2015021426A1 (en) * 2013-08-09 2015-02-12 Sage Labs, Inc. A crispr/cas system-based novel fusion protein and its application in genome editing
JP2015092462A (ja) 2013-09-30 2015-05-14 Tdk株式会社 正極及びそれを用いたリチウムイオン二次電池
WO2015141521A1 (ja) 2014-03-21 2015-09-24 株式会社日立国際電気 基板処理装置、半導体装置の製造方法及び記録媒体
CN111647627A (zh) * 2014-04-28 2020-09-11 重组股份有限公司 多重基因编辑
US20170037431A1 (en) * 2014-05-01 2017-02-09 University Of Washington In vivo Gene Engineering with Adenoviral Vectors
US20170191123A1 (en) * 2014-05-28 2017-07-06 Toolgen Incorporated Method for Sensitive Detection of Target DNA Using Target-Specific Nuclease
JP6197169B2 (ja) 2014-09-29 2017-09-20 東芝メモリ株式会社 半導体装置の製造方法
WO2019081982A1 (en) * 2017-10-26 2019-05-02 Crispr Therapeutics Ag SUBSTANCES AND METHODS FOR THE TREATMENT OF HEMOGLOBINOPATHIES
SG11202005147WA (en) * 2017-12-05 2020-06-29 Vertex Pharma Crispr-cas9 modified cd34+ human hematopoietic stem and progenitor cells and uses thereof

Similar Documents

Publication Publication Date Title
JP2019513407A5 (enExample)
CN108026526B (zh) 用于改善移植的crispr/cas相关方法和组合物
EP4086345A1 (en) Leaper technology based method for treating mps ih and composition
EP3487523B1 (en) Therapeutic applications of cpf1-based genome editing
JP6974349B2 (ja) ヘモグロビン異常症の処置のための材料及び方法
Kay State-of-the-art gene-based therapies: the road ahead
Ribeiro et al. Plasmid DNA size does affect nonviral gene delivery efficiency in stem cells
JP2020508659A5 (enExample)
CN110869498A (zh) 经由核递送crispr/cas9导向编辑细胞rna
JP2019520079A5 (enExample)
US20240123088A1 (en) USE OF A SPLIT dCAS FUSION PROTEIN SYSTEM FOR EPIGENETIC EDITING
AU2018353012A1 (en) Compositions and methods for gene editing for Hemophilia A
CN117802102A (zh) 用于治疗β-血红蛋白病的CRISPR/CAS相关方法和组合物
Locatelli et al. Autologous gene therapy for hemoglobinopathies: From bench to patient’s bedside
JP2019520080A5 (enExample)
EP4506461A1 (en) Method for repairing hba2 gene mutations by single base editing and use thereof
EP3640334A1 (en) Genome editing system for repeat expansion mutation
KR20220025757A (ko) 진핵 세포에 유전자 편집 시스템을 전달하기 위한 박테리아 플랫폼
Peng et al. Short noncoding DNA fragment improve efficiencies of in vivo electroporation‐mediated gene transfer
Hu et al. Co-opting regulation bypass repair as a gene-correction strategy for monogenic diseases
CN117279671A (zh) 用于在c3安全港位点处敲入的策略
CN115873850A (zh) 腺嘌呤碱基编辑系统及其应用
WO2025190256A1 (en) Type ii cas protein and uses thereof
WO2025036482A1 (en) Type ii cas protein, crispr-cas system and uses thereof
Fang et al. Gene editing in regenerative medicine