JP6866385B2 - 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 - Google Patents

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 Download PDF

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JP6866385B2
JP6866385B2 JP2018543036A JP2018543036A JP6866385B2 JP 6866385 B2 JP6866385 B2 JP 6866385B2 JP 2018543036 A JP2018543036 A JP 2018543036A JP 2018543036 A JP2018543036 A JP 2018543036A JP 6866385 B2 JP6866385 B2 JP 6866385B2
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cells
hspc
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JP2018533625A5 (enExample
JP2018533625A (ja
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ザール ジル
ザール ジル
ミリアム キム
ミリアム キム
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ザ トラスティーズ オブ ザ ユニバーシティ オブ ペンシルバニア
ザ トラスティーズ オブ ザ ユニバーシティ オブ ペンシルバニア
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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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Families Citing this family (60)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013066438A2 (en) 2011-07-22 2013-05-10 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
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US20150166984A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting alpha-antitrypsin point mutations
WO2016022363A2 (en) 2014-07-30 2016-02-11 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
EP3025719B1 (en) 2014-11-26 2018-09-26 Miltenyi Biotec GmbH Combination immunotherapy of antigen-recognizing receptors and hematopoietic cells for the treatment of diseases
AU2016337525B2 (en) 2015-10-16 2022-01-20 The Trustees Of Columbia University In The City Of New York Compositions and methods for inhibition of lineage specific antigens
SG10202104041PA (en) 2015-10-23 2021-06-29 Harvard College Nucleobase editors and uses thereof
EP3370741B1 (en) * 2015-11-04 2025-10-08 The Trustees of the University of Pennsylvania Methods and compositions for gene editing in hematopoietic stem cells
HK1257668A1 (zh) * 2016-02-19 2019-10-25 The Regents Of The University Of California 短发夹rna(shrna734)及其用於阳性选择和消除遗传修饰的细胞的用途
KR20250103795A (ko) 2016-08-03 2025-07-07 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
US11661590B2 (en) 2016-08-09 2023-05-30 President And Fellows Of Harvard College Programmable CAS9-recombinase fusion proteins and uses thereof
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
SG11201903089RA (en) 2016-10-14 2019-05-30 Harvard College Aav delivery of nucleobase editors
HUE067685T2 (hu) * 2016-11-02 2024-11-28 Univ Basel Immunológiailag megkülönböztethetõ sejtfelszíni variánsok sejtterápiában történõ alkalmazásra
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
IL268895B2 (en) * 2017-02-28 2025-05-01 Vor Biopharma Inc Compositions and methods for inhibition of lineage specific proteins
US11898179B2 (en) 2017-03-09 2024-02-13 President And Fellows Of Harvard College Suppression of pain by gene editing
EP3592381A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Cancer vaccine
WO2018165629A1 (en) 2017-03-10 2018-09-13 President And Fellows Of Harvard College Cytosine to guanine base editor
WO2018176009A1 (en) 2017-03-23 2018-09-27 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
CN111801345A (zh) 2017-07-28 2020-10-20 哈佛大学的校长及成员们 使用噬菌体辅助连续进化(pace)的进化碱基编辑器的方法和组合物
EP3676376B1 (en) 2017-08-30 2025-01-15 President and Fellows of Harvard College High efficiency base editors comprising gam
US11795443B2 (en) 2017-10-16 2023-10-24 The Broad Institute, Inc. Uses of adenosine base editors
WO2019118949A1 (en) 2017-12-15 2019-06-20 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
EP3794130A4 (en) 2018-05-16 2022-07-27 Synthego Corporation METHODS AND SYSTEMS FOR DESIGNING AND USING GUIDE RNA
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
EP3820495A4 (en) 2018-07-09 2022-07-20 The Broad Institute Inc. RNA-PROGRAMMABLE EPIGENETIC RNA MODIFIERS AND THEIR USES
WO2020047164A1 (en) 2018-08-28 2020-03-05 Vor Biopharma, Inc Genetically engineered hematopoietic stem cells and uses thereof
JP7716976B2 (ja) * 2018-09-19 2025-08-01 フジフィルム セルラー ダイナミクス，インコーポレイテッド キメラ抗原受容体改変免疫細胞の活性化及び拡大培養のためのプロテインｌ
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
CA3116412A1 (en) * 2018-10-31 2020-05-07 Humanigen, Inc. Materials and methods for treating 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
AU2020207962A1 (en) * 2019-01-18 2021-07-22 University Of Southern California Methods and compositions to improve the safety and efficacy of cellular therapies
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
EP4219700A1 (en) * 2019-03-07 2023-08-02 The Regents of the University of California Crispr-cas effector polypeptides and methods of use thereof
EP3942043A2 (en) 2019-03-19 2022-01-26 The Broad Institute, Inc. 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
US20220333116A1 (en) * 2019-08-28 2022-10-20 Vor Biopharma Inc. Compositions and methods for cd123 modification
WO2021041971A1 (en) * 2019-08-28 2021-03-04 Vor Biopharma, Inc. Compositions and methods for cll1 modification
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
AU2020361683A1 (en) 2019-10-11 2022-05-19 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
KR102923195B1 (ko) 2020-02-28 2026-02-04 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
EP3909612A1 (en) * 2020-05-12 2021-11-17 Life Science Inkubator Betriebs GmbH & Co. KG Composition of nanoparticles
AU2021413252A1 (en) * 2020-12-31 2023-06-08 Vor Biopharma Inc. Compositions and methods for cd34 gene modification
CN115443333B (zh) * 2021-04-27 2025-04-25 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
CA3218208A1 (en) * 2021-04-28 2022-11-03 Vinod VATHIPADIEKAL Antisense oligonucleotides and their use for treatment of neurodegenerative disorders
CA3236152A1 (en) * 2021-11-09 2023-05-19 Vor Biopharma Inc. Compositions and methods for erm2 modification
EP4433512A4 (en) 2021-11-19 2025-12-31 Univ Pennsylvania Genetically modified pan-leukocyte-specific CD45 antigen to facilitate T-lymphocyte therapy
EP4463190A4 (en) * 2022-01-11 2026-01-14 Actinium Pharmaceuticals Inc TREATMENT METHODS FOR CD33-POSITIVE HEMATOLOGICAL MALIGNITIES
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
US9573988B2 (en) * 2013-02-20 2017-02-21 Novartis Ag Effective targeting of primary human leukemia using anti-CD123 chimeric antigen receptor engineered T cells
DK3309248T3 (da) * 2013-05-29 2021-08-02 Cellectis Fremgangsmåde til manipulering af T-celler til immunterapi under anvendelse af et RNA-guidet CAS-nuklease-system
IL322264A (en) * 2014-04-07 2025-09-01 Novartis Ag Cancer treatment using chimeric antigen receptor (CAR) against CD19
AU2016337525B2 (en) * 2015-10-16 2022-01-20 The Trustees Of Columbia University In The City Of New York Compositions and methods for inhibition of lineage specific antigens
EP3370741B1 (en) * 2015-11-04 2025-10-08 The Trustees of the University of Pennsylvania Methods and compositions for gene editing in hematopoietic stem cells

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