JP2018533625A5 - - Google Patents

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JP2018533625A5
JP2018533625A5 JP2018543036A JP2018543036A JP2018533625A5 JP 2018533625 A5 JP2018533625 A5 JP 2018533625A5 JP 2018543036 A JP2018543036 A JP 2018543036A JP 2018543036 A JP2018543036 A JP 2018543036A JP 2018533625 A5 JP2018533625 A5 JP 2018533625A5
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hspc
cancer
seq
cells
deletion
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JP2018543036A
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JP2018533625A (ja
JP6866385B2 (ja
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Priority claimed from PCT/US2016/060273 external-priority patent/WO2017079400A1/en
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JP2018543036A 2015-11-04 2016-11-03 造血幹細胞における遺伝子編集のための方法および組成物 Expired - Fee Related JP6866385B2 (ja)

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JP2021065361A JP7378439B2 (ja) 2015-11-04 2021-04-07 造血幹細胞における遺伝子編集のための方法および組成物
JP2023097365A JP2023115069A (ja) 2015-11-04 2023-06-14 造血幹細胞における遺伝子編集のための方法および組成物

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US201562250561P 2015-11-04 2015-11-04
US62/250,561 2015-11-04
PCT/US2016/060273 WO2017079400A1 (en) 2015-11-04 2016-11-03 Methods and compositions for gene editing in hematopoietic stem cells

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JP2018533625A JP2018533625A (ja) 2018-11-15
JP2018533625A5 true JP2018533625A5 (OSRAM) 2019-12-05
JP6866385B2 JP6866385B2 (ja) 2021-04-28

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JP2018543036A Expired - Fee Related JP6866385B2 (ja) 2015-11-04 2016-11-03 造血幹細胞における遺伝子編集のための方法および組成物
JP2021065361A Active JP7378439B2 (ja) 2015-11-04 2021-04-07 造血幹細胞における遺伝子編集のための方法および組成物
JP2023097365A Pending JP2023115069A (ja) 2015-11-04 2023-06-14 造血幹細胞における遺伝子編集のための方法および組成物

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JP2021065361A Active JP7378439B2 (ja) 2015-11-04 2021-04-07 造血幹細胞における遺伝子編集のための方法および組成物
JP2023097365A Pending JP2023115069A (ja) 2015-11-04 2023-06-14 造血幹細胞における遺伝子編集のための方法および組成物

Country Status (17)

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US (3) US10548922B2 (OSRAM)
EP (1) EP3370741B1 (OSRAM)
JP (3) JP6866385B2 (OSRAM)
KR (1) KR20180073679A (OSRAM)
CN (1) CN108463227A (OSRAM)
AU (2) AU2016349280B2 (OSRAM)
BR (1) BR112018009098A8 (OSRAM)
CA (1) CA3004053A1 (OSRAM)
CL (1) CL2018001198A1 (OSRAM)
EA (1) EA201891092A1 (OSRAM)
HK (1) HK1259151A1 (OSRAM)
IL (1) IL259084A (OSRAM)
MX (1) MX2018005612A (OSRAM)
MY (1) MY185961A (OSRAM)
PH (1) PH12018500965A1 (OSRAM)
SG (2) SG10202107602XA (OSRAM)
WO (1) WO2017079400A1 (OSRAM)

Families Citing this family (58)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3613852A3 (en) 2011-07-22 2020-04-22 President and Fellows of Harvard College Evaluation and improvement of nuclease cleavage specificity
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
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
US11053481B2 (en) 2013-12-12 2021-07-06 President And Fellows Of Harvard College Fusions of Cas9 domains and nucleic acid-editing domains
AU2015298571B2 (en) 2014-07-30 2020-09-03 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
ES2692206T3 (es) 2014-11-26 2018-11-30 Miltenyi Biotec Gmbh Inmunoterapia combinada de receptores de reconocimiento de antígenos y células hematopoyéticas para el tratamiento de enfermedades
CN116712535A (zh) 2015-10-16 2023-09-08 纽约市哥伦比亚大学理事会 用于抑制谱系特异性抗原的组合物和方法
US12043852B2 (en) 2015-10-23 2024-07-23 President And Fellows Of Harvard College Evolved Cas9 proteins for gene editing
SG10202107602XA (en) * 2015-11-04 2021-08-30 Univ Pennsylvania Methods and compositions for gene editing in hematopoietic stem cells
CN108495640B (zh) * 2016-02-19 2022-11-04 加利福尼亚大学董事会 短发夹rna(shrna734)及其用于阳性选择和消除遗传修饰的细胞的用途
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
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
GB2573062A (en) 2016-10-14 2019-10-23 Harvard College AAV delivery of nucleobase editors
SG11201903454VA (en) * 2016-11-02 2019-05-30 Univ Basel Immunologically discernible cell surface variants for use in cell therapy
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
AU2018229351B2 (en) 2017-02-28 2024-01-04 Vor Biopharma, Inc. Compositions and methods for inhibition of lineage specific proteins
WO2018165631A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Cancer vaccine
US11898179B2 (en) 2017-03-09 2024-02-13 President And Fellows Of Harvard College Suppression of pain by gene editing
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
US11268082B2 (en) 2017-03-23 2022-03-08 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable DNA binding proteins
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
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
CA3082251A1 (en) 2017-10-16 2019-04-25 The Broad Institute, Inc. Uses of adenosine base editors
US12406749B2 (en) 2017-12-15 2025-09-02 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
CN112654710A (zh) 2018-05-16 2021-04-13 辛瑟高公司 用于指导rna设计和使用的方法和系统
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
IL260445B (en) * 2018-07-05 2019-08-29 Lempo Therapeutics Ltd Methods and compositions for modulating myeloperoxidase (mpo) expression
WO2020047164A1 (en) 2018-08-28 2020-03-05 Vor Biopharma, Inc Genetically engineered hematopoietic stem cells and uses thereof
IL281413B2 (en) * 2018-09-19 2024-09-01 Fujifilm Cellular Dynamics Inc Protein l for activation and expansion of chimeric antigen receptor-modified immune cells
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
EP3873205A4 (en) * 2018-10-31 2022-05-11 Humanigen, Inc. MATERIALS AND METHODS FOR TREATMENT OF CANCER
CN109852627B (zh) * 2018-12-26 2022-07-05 中国人民解放军军事科学院军事医学研究院 用于CRISPRi系统的质粒、其构建方法及其在使鼠疫菌目的基因定向沉默中的应用
EP3911371A4 (en) * 2019-01-18 2023-04-26 University of Southern California METHODS AND COMPOSITIONS FOR IMPROVING THE SAFETY AND EFFICACY OF CELL THERAPY
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
WO2020172638A1 (en) * 2019-02-22 2020-08-27 The Trustees Of The University Of Pennsylvania Crispr/cas9 knock-out of cd33 in human hematopoietic stem/ progenitor cells for allogenic transplantation
WO2020181101A1 (en) * 2019-03-07 2020-09-10 The Regents Of The University Of California Crispr-cas effector polypeptides and methods of use thereof
AU2020240109A1 (en) 2019-03-19 2021-09-30 President And Fellows Of Harvard College Methods and compositions for editing nucleotide sequences
WO2020214842A1 (en) 2019-04-17 2020-10-22 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
KR20220047381A (ko) * 2019-08-28 2022-04-15 보르 바이오파마 인크. Cd123 변형을 위한 조성물 및 방법
MX2022002462A (es) * 2019-08-28 2022-06-02 Vor Biopharma Inc Composiciones y métodos para la modificación de cll1.
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
US12383581B2 (en) 2019-10-11 2025-08-12 The Trustees Of The University Of Pennsylvania Compositions and methods for targeting CD13 and TIM-3 with CAR T cells to treat acute myeloid leukemia
KR20210109971A (ko) 2020-02-28 2021-09-07 현대자동차주식회사 리튬이온 전지 음극 제조방법 및 이에 의해 제조된 리튬이온 전지 음극을 포함하는 리튬이온 전지
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
US20240366675A1 (en) * 2020-12-31 2024-11-07 Vor Biopharma Inc. Compositions and methods for cd34 gene modification
CN120249215A (zh) * 2021-04-27 2025-07-04 上海驯鹿生物技术有限公司 一种基因编辑的造血干细胞及其与car-t细胞的联合应用
KR20240004702A (ko) * 2021-04-28 2024-01-11 에자이 알앤드디 매니지먼트 가부시키가이샤 안티센스 올리고뉴클레오티드 및 신경퇴행성 장애의 치료를 위한 이들의 용도
JP2024543369A (ja) * 2021-11-09 2024-11-21 ブイオーアール バイオファーマ インコーポレーテッド Emr2修飾のための化合物及び方法
WO2023091954A2 (en) 2021-11-19 2023-05-25 The Trustees Of The University Of Pennsylvania Engineered pan-leukocyte antigen cd45 to facilitate car t cell therapy
EP4463190A2 (en) * 2022-01-11 2024-11-20 Actinium Pharmaceuticals, Inc. Methods for treating cd33-positive hematological malignancies
WO2025030010A1 (en) 2023-08-01 2025-02-06 Vor Biopharma Inc. Compositions comprising genetically engineered hematopoietic stem cells and methods of use thereof
WO2025059062A1 (en) * 2023-09-11 2025-03-20 The Trustees Of The University Of Pennsylvania Genetic engineering of human hematopoietic stem/progenitor cells (hspcs) for locus-specific expression of therapeutic proteins

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2008086529A2 (en) * 2007-01-11 2008-07-17 Yale University Compositions and methods for targeted inactivation of hiv cell surface receptors
EP3763810A3 (en) * 2012-10-10 2021-07-14 Sangamo Therapeutics, Inc. T cell modifying compounds and uses thereof
WO2014130635A1 (en) * 2013-02-20 2014-08-28 Novartis Ag Effective targeting of primary human leukemia using anti-cd123 chimeric antigen receptor engineered t cells
AU2014273490B2 (en) * 2013-05-29 2019-05-09 Cellectis Methods for engineering T cells for immunotherapy by using RNA-guided Cas nuclease system
PL3888674T3 (pl) * 2014-04-07 2024-09-23 Novartis Ag Leczenie nowotworu złośliwego z zastosowaniem chimerycznego receptora antygenowego anty-CD19
CN116712535A (zh) * 2015-10-16 2023-09-08 纽约市哥伦比亚大学理事会 用于抑制谱系特异性抗原的组合物和方法
SG10202107602XA (en) * 2015-11-04 2021-08-30 Univ Pennsylvania Methods and compositions for gene editing in hematopoietic stem cells

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