PL2890780T3 - Sposoby i kompozycje do leczenia zaburzeń genetycznych - Google Patents

Sposoby i kompozycje do leczenia zaburzeń genetycznych

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Publication number
PL2890780T3
PL2890780T3 PL13833658T PL13833658T PL2890780T3 PL 2890780 T3 PL2890780 T3 PL 2890780T3 PL 13833658 T PL13833658 T PL 13833658T PL 13833658 T PL13833658 T PL 13833658T PL 2890780 T3 PL2890780 T3 PL 2890780T3
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PL
Poland
Prior art keywords
compositions
treatment
methods
genetic condition
genetic
Prior art date
Application number
PL13833658T
Other languages
English (en)
Polish (pl)
Inventor
Gregory J. Cost
Philip D. Gregory
Dmitry Guschin
Michael C. Holmes
Jeffrey C. Miller
David Paschon
Edward J. Rebar
Andreas Reik
Fyodor Urnov
Lei Zhang
Original Assignee
Sangamo Therapeutics, Inc.
Priority date (The priority date 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 date listed.)
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Publication date
Application filed by Sangamo Therapeutics, Inc. filed Critical Sangamo Therapeutics, Inc.
Publication of PL2890780T3 publication Critical patent/PL2890780T3/pl

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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • A61P7/06Antianaemics
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    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702Regulators; Modulating activity
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    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/795Porphyrin- or corrin-ring-containing peptides
    • C07K14/805Haemoglobins; Myoglobins
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/52Genes encoding for enzymes or proenzymes
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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/62DNA sequences coding for fusion proteins
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    • C12N5/00Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06Animal cells or tissues; Human cells or tissues
    • C12N5/0602Vertebrate cells
    • C12N5/0634Cells from the blood or the immune system
    • C12N5/0647Haematopoietic stem cells; Uncommitted or multipotent progenitors
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    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/80Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • C07K2319/81Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor containing a Zn-finger domain for DNA binding
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    • C12N2510/00Genetically modified cells
    • C12N2510/02Cells for production

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  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Hematology (AREA)
  • Toxicology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Diabetes (AREA)
  • Immunology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Cell Biology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Enzymes And Modification Thereof (AREA)
PL13833658T 2012-08-29 2013-08-29 Sposoby i kompozycje do leczenia zaburzeń genetycznych PL2890780T3 (pl)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201261694693P 2012-08-29 2012-08-29
PCT/US2013/057214 WO2014036219A2 (en) 2012-08-29 2013-08-29 Methods and compositions for treatment of a genetic condition
EP13833658.1A EP2890780B8 (en) 2012-08-29 2013-08-29 Methods and compositions for treatment of a genetic condition

Publications (1)

Publication Number Publication Date
PL2890780T3 true PL2890780T3 (pl) 2021-02-08

Family

ID=50184630

Family Applications (1)

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PL13833658T PL2890780T3 (pl) 2012-08-29 2013-08-29 Sposoby i kompozycje do leczenia zaburzeń genetycznych

Country Status (26)

Country Link
US (4) US9650648B2 (h)
EP (1) EP2890780B8 (h)
JP (4) JP2015533786A (h)
KR (3) KR102474010B1 (h)
CN (1) CN104704110B (h)
AU (1) AU2013308770B2 (h)
CA (2) CA3171494A1 (h)
CL (1) CL2015000450A1 (h)
DK (1) DK2890780T3 (h)
EA (1) EA039384B1 (h)
ES (1) ES2812599T3 (h)
HR (1) HRP20201443T1 (h)
HU (1) HUE050516T2 (h)
IL (1) IL237050B (h)
IN (1) IN2015DN01480A (h)
LT (1) LT2890780T (h)
MX (1) MX367081B (h)
PH (3) PH12021551661A1 (h)
PL (1) PL2890780T3 (h)
PT (1) PT2890780T (h)
RS (1) RS60838B1 (h)
SG (2) SG10201701601WA (h)
SI (1) SI2890780T1 (h)
UA (1) UA118957C2 (h)
WO (1) WO2014036219A2 (h)
ZA (1) ZA201500900B (h)

Families Citing this family (124)

* 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
GB201122458D0 (en) 2011-12-30 2012-02-08 Univ Wageningen Modified cascade ribonucleoproteins and uses thereof
BR112014020625A2 (pt) 2012-02-24 2017-07-04 Hutchinson Fred Cancer Res polinucleotídeo, polipeptídeo, composição, célula e célula tronco editada por genoma
SG10201809817UA (en) 2012-05-25 2018-12-28 Univ California Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
CN110669746B (zh) * 2012-10-23 2024-04-16 基因工具股份有限公司 用于切割靶dna的组合物及其用途
CA2977152C (en) 2012-12-06 2019-04-09 Sigma-Aldrich Co. Llc Crispr-based genome modification and regulation
CA2901676C (en) 2013-02-25 2023-08-22 Sangamo Biosciences, Inc. Methods and compositions for enhancing nuclease-mediated gene disruption
KR20170108172A (ko) 2013-03-14 2017-09-26 카리부 바이오사이언시스 인코포레이티드 핵산-표적화 핵산의 조성물 및 방법
EP2981617B1 (en) 2013-04-04 2023-07-05 President and Fellows of Harvard College Therapeutic uses of genome editing with crispr/cas systems
WO2014186585A2 (en) * 2013-05-15 2014-11-20 Sangamo Biosciences, Inc. Methods and compositions for treatment of a genetic condition
KR102551324B1 (ko) * 2013-06-05 2023-07-05 듀크 유니버시티 Rna-가이드 유전자 편집 및 유전자 조절
EP3019595A4 (en) 2013-07-09 2016-11-30 THERAPEUTIC USES OF A GENERIC CHANGE WITH CRISPR / CAS SYSTEMS
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
US9567609B2 (en) 2013-08-28 2017-02-14 Sangamo Biosciences, Inc. Compositions for linking DNA-binding domains and cleavage domains
US9737604B2 (en) 2013-09-06 2017-08-22 President And Fellows Of Harvard College Use of cationic lipids to deliver CAS9
US9340800B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College Extended DNA-sensing GRNAS
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
JP2016536021A (ja) 2013-11-07 2016-11-24 エディタス・メディシン,インコーポレイテッド CRISPR関連方法および支配gRNAのある組成物
PT3492593T (pt) * 2013-11-13 2021-10-18 Childrens Medical Center Regulação da expressão de genes mediada por nucleases
US20150166982A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting pi3k point mutations
ES2796087T3 (es) 2014-02-03 2020-11-25 Sangamo Therapeutics Inc Métodos y composiciones para el tratamiento de beta talasemia
US10370680B2 (en) 2014-02-24 2019-08-06 Sangamo Therapeutics, Inc. Method of treating factor IX deficiency using nuclease-mediated targeted integration
WO2015134812A1 (en) 2014-03-05 2015-09-11 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating usher syndrome and retinitis pigmentosa
US11339437B2 (en) 2014-03-10 2022-05-24 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
US11141493B2 (en) 2014-03-10 2021-10-12 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
EP3116997B1 (en) 2014-03-10 2019-05-15 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating leber's congenital amaurosis 10 (lca10)
WO2015143046A2 (en) 2014-03-18 2015-09-24 Sangamo Biosciences, Inc. Methods and compositions for regulation of zinc finger protein expression
WO2015148860A1 (en) * 2014-03-26 2015-10-01 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating beta-thalassemia
US11242525B2 (en) 2014-03-26 2022-02-08 Editas Medicine, Inc. CRISPR/CAS-related methods and compositions for treating sickle cell disease
EP3777534A1 (en) * 2014-03-26 2021-02-17 The Brigham & Women's Hospital, Inc. Compositions and methods for ex vivo expansion of human hematopoietic stem/progenitor cells
US10077453B2 (en) 2014-07-30 2018-09-18 President And Fellows Of Harvard College CAS9 proteins including ligand-dependent inteins
SG11201701245QA (en) 2014-08-27 2017-03-30 Caribou Biosciences Inc Methods for increasing cas9-mediated engineering efficiency
EP3194570B1 (en) * 2014-09-16 2021-06-30 Sangamo Therapeutics, Inc. Methods and compositions for nuclease-mediated genome engineering and correction in hematopoietic stem cells
US12180263B2 (en) 2014-11-06 2024-12-31 President And Fellows Of Harvard College Cells lacking B2M surface expression and methods for allogeneic administration of such cells
WO2016085934A1 (en) * 2014-11-24 2016-06-02 Children's Medical Center Corporation Modulation of sh2b3 to improve red blood cell production from stem cells and/or progenitor cells
WO2016094880A1 (en) * 2014-12-12 2016-06-16 The Broad Institute Inc. Delivery, use and therapeutic applications of crispr systems and compositions for genome editing as to hematopoietic stem cells (hscs)
WO2016135559A2 (en) * 2015-02-23 2016-09-01 Crispr Therapeutics Ag Materials and methods for treatment of human genetic diseases including hemoglobinopathies
SG11201706766WA (en) * 2015-02-23 2017-09-28 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
EP3910641B1 (en) * 2015-04-13 2022-10-05 Invitae Corporation Methods, systems and processes of identifying genetic variation in highly similar genes
CN107690480B (zh) 2015-04-24 2022-03-22 爱迪塔斯医药公司 Cas9分子/指导rna分子复合物的评价
US10179918B2 (en) 2015-05-07 2019-01-15 Sangamo Therapeutics, Inc. Methods and compositions for increasing transgene activity
AU2016261600B2 (en) 2015-05-08 2021-09-23 President And Fellows Of Harvard College Universal donor stem cells and related methods
HK1251011A1 (zh) 2015-05-12 2019-01-18 Sangamo Therapeutics, Inc. 核酸酶介导的基因表达调控
US9957501B2 (en) 2015-06-18 2018-05-01 Sangamo Therapeutics, Inc. Nuclease-mediated regulation of gene expression
US10676735B2 (en) * 2015-07-22 2020-06-09 Duke University High-throughput screening of regulatory element function with epigenome editing technologies
AU2016325613B2 (en) * 2015-09-23 2020-06-25 Sangamo Therapeutics, Inc. Htt repressors and uses thereof
EP3365440B1 (en) * 2015-10-20 2022-09-14 Pioneer Hi-Bred International, Inc. Restoring function to a non-functional gene product via guided cas systems and methods of use
AU2016342380B2 (en) 2015-10-23 2022-04-07 President And Fellows Of Harvard College Nucleobase editors and uses thereof
MY189674A (en) 2015-10-28 2022-02-24 Sangamo Therapeutics Inc Liver-specific constructs, factor viii expression cassettes and methods of use thereof
US12043843B2 (en) 2015-11-04 2024-07-23 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of hemoglobinopathies
CN108699542A (zh) 2015-11-13 2018-10-23 阿维利诺美国实验室股份有限公司 用于治疗角膜营养不良的方法
CN117137947A (zh) 2015-12-18 2023-12-01 桑格摩生物治疗股份有限公司 Mhc细胞受体的靶向破坏
WO2017143071A1 (en) 2016-02-18 2017-08-24 The Regents Of The University Of California Methods and compositions for gene editing in stem cells
AU2017235333B2 (en) * 2016-03-14 2023-08-24 Editas Medicine, Inc. CRISPR/CAS-related methods and compositions for treating beta hemoglobinopathies
WO2017165862A1 (en) 2016-03-25 2017-09-28 Editas Medicine, Inc. Systems and methods for treating alpha 1-antitrypsin (a1at) deficiency
BR112019001887A2 (pt) 2016-08-02 2019-07-09 Editas Medicine Inc composições e métodos para o tratamento de doença associada a cep290
KR102827276B1 (ko) 2016-08-03 2025-07-01 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
WO2018031683A1 (en) 2016-08-09 2018-02-15 President And Fellows Of Harvard College Programmable cas9-recombinase fusion proteins and uses thereof
WO2020225754A1 (en) 2019-05-06 2020-11-12 Mcmullen Tara Crispr gene editing for autosomal dominant diseases
US20190185850A1 (en) 2016-08-20 2019-06-20 Avellino Lab Usa, Inc. Single guide rna/crispr/cas9 systems, and methods of use 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
HRP20212025T1 (hr) * 2016-08-24 2022-04-01 Sangamo Therapeutics, Inc. Regulacija ekspresije gena pomoću projektiranih nukleaza
SG10201913948PA (en) 2016-08-24 2020-03-30 Sangamo Therapeutics Inc Engineered target specific nucleases
WO2018049009A2 (en) 2016-09-07 2018-03-15 Sangamo Therapeutics, Inc. Modulation of liver genes
US20190330620A1 (en) * 2016-10-14 2019-10-31 Emendobio Inc. Rna compositions for genome editing
JP7588390B2 (ja) 2016-10-14 2024-11-22 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 核酸塩基エディターのaav送達
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
TWI758316B (zh) * 2017-01-09 2022-03-21 美商聖加莫治療股份有限公司 使用經工程改造之核酸酶調控基因表現
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
EP3592853A1 (en) 2017-03-09 2020-01-15 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
US11542496B2 (en) 2017-03-10 2023-01-03 President And Fellows Of Harvard College Cytosine to guanine base editor
EP3596217A1 (en) 2017-03-14 2020-01-22 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
EP3601562A1 (en) 2017-03-23 2020-02-05 President and Fellows of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
EP3615664A4 (en) * 2017-04-24 2021-01-27 Seattle Children's Hospital (DBA Seattle Children's Research Institute) HOMOLOGY-DIRECTED REPAIR COMPOSITIONS FOR THE TREATMENT OF HEMOGLOBINOPATHIES
WO2018209158A2 (en) 2017-05-10 2018-11-15 Editas Medicine, Inc. Crispr/rna-guided nuclease systems and methods
US11560566B2 (en) 2017-05-12 2023-01-24 President And Fellows Of Harvard College Aptazyme-embedded guide RNAs for use with CRISPR-Cas9 in genome editing and transcriptional activation
MX2020000676A (es) * 2017-07-18 2021-05-14 Csl Behring Gene Therapy Inc Composiciones y metodos para tratar beta-hemoglobinopatias.
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
WO2019139645A2 (en) 2017-08-30 2019-07-18 President And Fellows Of Harvard College High efficiency base editors comprising gam
AU2018352592C1 (en) 2017-10-16 2025-09-25 Beam Therapeutics, Inc. Uses of adenosine base editors
EP3701029A1 (en) 2017-10-26 2020-09-02 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of hemoglobinopathies
BR112020008568A2 (pt) * 2017-11-09 2020-10-06 Sangamo Therapeutics, Inc. modificação genética de gene de proteína contendo sh2 indutível por citoquina (cish)
US12406749B2 (en) 2017-12-15 2025-09-02 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
EP3511412A1 (en) * 2018-01-12 2019-07-17 Genethon Genetically engineered hematopoietic stem cell as a platform for systemic protein expression
MA51788A (fr) 2018-02-05 2020-12-16 Vertex Pharma Substances et méthodes pour traiter des hémoglobinopathies
BR112020015488A2 (pt) * 2018-02-08 2020-12-08 Sangamo Therapeutics, Inc. Nucleases alvo-específicas modificadas
JP2021513903A (ja) 2018-02-21 2021-06-03 アルキオーネ・ライフサイエンシズ・インコーポレイテッドAlcyone Lifesciences, Inc. 流体送達システムおよび方法
EP3765614A1 (en) 2018-03-14 2021-01-20 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
KR20250134703A (ko) 2018-05-11 2025-09-11 빔 테라퓨틱스, 인크. 프로그래밍가능한 염기 편집기 시스템을 이용하여 병원성 아미노산을 치환하는 방법
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
AU2019327386A1 (en) 2018-08-27 2021-03-18 Alcyone Therapeutics, Inc. Fluid delivery systems and methods
US12281338B2 (en) 2018-10-29 2025-04-22 The Broad Institute, Inc. Nucleobase editors comprising GeoCas9 and uses thereof
WO2020154500A1 (en) 2019-01-23 2020-07-30 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
US20230078265A1 (en) 2019-03-19 2023-03-16 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
CN115141807A (zh) 2019-04-02 2022-10-04 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
WO2020208223A1 (en) * 2019-04-12 2020-10-15 Institut National De La Sante Et De La Recherche Medicale (Inserm) Correction of βeta-thalassemia phenotype by genetically engineered hematopoietic stem cell
CN112391410B (zh) * 2020-02-17 2024-04-02 华东师范大学 一种sgRNA及其在修复内含子异常剪接中的应用
US20220280571A1 (en) * 2019-08-01 2022-09-08 The Regents Of The University Of California Compositions and methods for treating alpha thalassemia
WO2021026336A2 (en) * 2019-08-07 2021-02-11 I Altius Institute For Biomedical Sciences Compositions and methods for modulation of gene expression
CA3147643A1 (en) * 2019-09-23 2021-04-01 Omega Therapeutics, Inc. Compositions and methods for modulating hepatocyte nuclear factor 4-alpha (hnf4.alpha.) gene expression
WO2021072328A1 (en) 2019-10-10 2021-04-15 The Broad Institute, Inc. Methods and compositions for prime editing rna
US20230227513A1 (en) * 2019-10-15 2023-07-20 Altius Institute For Biomedical Sciences DNA Binding Proteins for Displacing Endogenous Transcription Factors Bound to Gene Regulatory Regions
US20250263752A1 (en) 2020-04-21 2025-08-21 Aspen Neuroscience, Inc. Gene editing of gba1 in stem cells and method of use of cells differentiated therefrom
WO2021216623A1 (en) 2020-04-21 2021-10-28 Aspen Neuroscience, Inc. Gene editing of lrrk2 in stem cells and method of use of cells differentiated therefrom
JP2023525304A (ja) 2020-05-08 2023-06-15 ザ ブロード インスティテュート,インコーポレーテッド 標的二本鎖ヌクレオチド配列の両鎖同時編集のための方法および組成物
WO2023115039A2 (en) 2021-12-17 2023-06-22 Sana Biotechnology, Inc. Modified paramyxoviridae fusion glycoproteins
US20240408192A1 (en) 2021-12-17 2024-12-12 Sana Biotechnology, Inc. Modified paramyxoviridae attachment glycoproteins
EP4463135A2 (en) 2022-01-10 2024-11-20 Sana Biotechnology, Inc. Methods of ex vivo dosing and administration of lipid particles or viral vectors and related systems and uses
EP4472646A1 (en) 2022-02-01 2024-12-11 Sana Biotechnology, Inc. Cd3-targeted lentiviral vectors and uses thereof
WO2023150647A1 (en) 2022-02-02 2023-08-10 Sana Biotechnology, Inc. Methods of repeat dosing and administration of lipid particles or viral vectors and related systems and uses
WO2024044655A1 (en) 2022-08-24 2024-02-29 Sana Biotechnology, Inc. Delivery of heterologous proteins
WO2024064838A1 (en) 2022-09-21 2024-03-28 Sana Biotechnology, Inc. Lipid particles comprising variant paramyxovirus attachment glycoproteins and uses thereof
WO2024081820A1 (en) 2022-10-13 2024-04-18 Sana Biotechnology, Inc. Viral particles targeting hematopoietic stem cells
WO2024119157A1 (en) 2022-12-02 2024-06-06 Sana Biotechnology, Inc. Lipid particles with cofusogens and methods of producing and using the same
WO2024220574A1 (en) 2023-04-18 2024-10-24 Sana Biotechnology, Inc. Universal protein g fusogens and adapter systems thereof and related lipid particles and uses
WO2024220598A2 (en) 2023-04-18 2024-10-24 Sana Biotechnology, Inc. Lentiviral vectors with two or more genomes
WO2024220560A1 (en) 2023-04-18 2024-10-24 Sana Biotechnology, Inc. Engineered protein g fusogens and related lipid particles and methods thereof
WO2024243340A1 (en) 2023-05-23 2024-11-28 Sana Biotechnology, Inc. Tandem fusogens and related lipid particles
WO2025054202A1 (en) 2023-09-05 2025-03-13 Sana Biotechnology, Inc. Method of screening a sample comprising a transgene with a unique barcode
US20250230470A1 (en) 2024-01-12 2025-07-17 Sana Biotechnology, Inc. Safety switches to control in vitro and in vivo proliferation of cell therapy products
WO2025153530A1 (en) 2024-01-16 2025-07-24 Novo Nordisk A/S Albumin-targeted endonucleases, compositions, and methods of use
WO2025184529A1 (en) 2024-03-01 2025-09-04 Sana Biotechnology, Inc. Viral particles with fusogen display and related compositions and methods

Family Cites Families (105)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US789538A (en) 1904-11-11 1905-05-09 Colin E Ham Dumb-bell.
US4217344A (en) 1976-06-23 1980-08-12 L'oreal Compositions containing aqueous dispersions of lipid spheres
US4235871A (en) 1978-02-24 1980-11-25 Papahadjopoulos Demetrios P Method of encapsulating biologically active materials in lipid vesicles
US4186183A (en) 1978-03-29 1980-01-29 The United States Of America As Represented By The Secretary Of The Army Liposome carriers in chemotherapy of leishmaniasis
US4261975A (en) 1979-09-19 1981-04-14 Merck & Co., Inc. Viral liposome particle
US4485054A (en) 1982-10-04 1984-11-27 Lipoderm Pharmaceuticals Limited Method of encapsulating biologically active materials in multilamellar lipid vesicles (MLV)
US4501728A (en) 1983-01-06 1985-02-26 Technology Unlimited, Inc. Masking of liposomes from RES recognition
US4897355A (en) 1985-01-07 1990-01-30 Syntex (U.S.A.) Inc. N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US4946787A (en) 1985-01-07 1990-08-07 Syntex (U.S.A.) Inc. N-(ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US5049386A (en) 1985-01-07 1991-09-17 Syntex (U.S.A.) Inc. N-ω,(ω-1)-dialkyloxy)- and N-(ω,(ω-1)-dialkenyloxy)Alk-1-YL-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US4797368A (en) 1985-03-15 1989-01-10 The United States Of America As Represented By The Department Of Health And Human Services Adeno-associated virus as eukaryotic expression vector
US4774085A (en) 1985-07-09 1988-09-27 501 Board of Regents, Univ. of Texas Pharmaceutical administration systems containing a mixture of immunomodulators
US5422251A (en) 1986-11-26 1995-06-06 Princeton University Triple-stranded nucleic acids
US4837028A (en) 1986-12-24 1989-06-06 Liposome Technology, Inc. Liposomes with enhanced circulation time
US5176996A (en) 1988-12-20 1993-01-05 Baylor College Of Medicine Method for making synthetic oligonucleotides which bind specifically to target sites on duplex DNA molecules, by forming a colinear triplex, the synthetic oligonucleotides and methods of use
US5264618A (en) 1990-04-19 1993-11-23 Vical, Inc. Cationic lipids for intracellular delivery of biologically active molecules
AU7979491A (en) 1990-05-03 1991-11-27 Vical, Inc. Intracellular delivery of biologically active substances by means of self-assembling lipid complexes
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
US5420032A (en) 1991-12-23 1995-05-30 Universitge Laval Homing endonuclease which originates from chlamydomonas eugametos and recognizes and cleaves a 15, 17 or 19 degenerate double stranded nucleotide sequence
US5487994A (en) 1992-04-03 1996-01-30 The Johns Hopkins University Insertion and deletion mutants of FokI restriction endonuclease
US5356802A (en) 1992-04-03 1994-10-18 The Johns Hopkins University Functional domains in flavobacterium okeanokoites (FokI) restriction endonuclease
US5436150A (en) 1992-04-03 1995-07-25 The Johns Hopkins University Functional domains in flavobacterium okeanokoities (foki) restriction endonuclease
US5792632A (en) 1992-05-05 1998-08-11 Institut Pasteur Nucleotide sequence encoding the enzyme I-SceI and the uses thereof
US5587308A (en) 1992-06-02 1996-12-24 The United States Of America As Represented By The Department Of Health & Human Services Modified adeno-associated virus vector capable of expression from a novel promoter
US6140466A (en) 1994-01-18 2000-10-31 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
CA2681922C (en) 1994-01-18 2012-05-15 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
US6242568B1 (en) 1994-01-18 2001-06-05 The Scripps Research Institute Zinc finger protein derivatives and methods therefor
EP0752005B1 (en) 1994-03-23 2008-10-08 Ohio University Compacted nucleic acids and their delivery to cells
US5585245A (en) 1994-04-22 1996-12-17 California Institute Of Technology Ubiquitin-based split protein sensor
GB9824544D0 (en) 1998-11-09 1999-01-06 Medical Res Council Screening system
USRE39229E1 (en) 1994-08-20 2006-08-08 Gendaq Limited Binding proteins for recognition of DNA
US5789538A (en) 1995-02-03 1998-08-04 Massachusetts Institute Of Technology Zinc finger proteins with high affinity new DNA binding specificities
US5925523A (en) 1996-08-23 1999-07-20 President & Fellows Of Harvard College Intraction trap assay, reagents and uses thereof
GB2338237B (en) 1997-02-18 2001-02-28 Actinova Ltd In vitro peptide or protein expression library
GB9703369D0 (en) 1997-02-18 1997-04-09 Lindqvist Bjorn H Process
US6342345B1 (en) 1997-04-02 2002-01-29 The Board Of Trustees Of The Leland Stanford Junior University Detection of molecular interactions by reporter subunit complementation
GB9710809D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
GB9710807D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
US6410248B1 (en) 1998-01-30 2002-06-25 Massachusetts Institute Of Technology General strategy for selecting high-affinity zinc finger proteins for diverse DNA target sites
WO1999045132A1 (en) 1998-03-02 1999-09-10 Massachusetts Institute Of Technology Poly zinc finger proteins with improved linkers
CO5070700A1 (es) 1998-06-05 2001-08-28 Basf Ag Genes novedosos de (adp-ribosa) polimerasa
US6140815A (en) 1998-06-17 2000-10-31 Dover Instrument Corporation High stability spin stand platform
US6532231B1 (en) 1998-08-29 2003-03-11 Lucent Technologies Inc. Arrangement for changing the destination of single link, single destination data messages
US6140081A (en) 1998-10-16 2000-10-31 The Scripps Research Institute Zinc finger binding domains for GNN
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US7013219B2 (en) 1999-01-12 2006-03-14 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
US7070934B2 (en) 1999-01-12 2006-07-04 Sangamo Biosciences, Inc. Ligand-controlled regulation of endogenous gene expression
US6453242B1 (en) 1999-01-12 2002-09-17 Sangamo Biosciences, Inc. Selection of sites for targeting by zinc finger proteins and methods of designing zinc finger proteins to bind to preselected sites
US6599692B1 (en) 1999-09-14 2003-07-29 Sangamo Bioscience, Inc. Functional genomics using zinc finger proteins
CA2361191A1 (en) 1999-02-03 2000-08-10 The Children's Medical Center Corporation Gene repair involving the induction of double-stranded dna cleavage at a chromosomal target site
US7030215B2 (en) 1999-03-24 2006-04-18 Sangamo Biosciences, Inc. Position dependent recognition of GNN nucleotide triplets by zinc fingers
US6794136B1 (en) 2000-11-20 2004-09-21 Sangamo Biosciences, Inc. Iterative optimization in the design of binding proteins
IL150069A0 (en) 1999-12-06 2002-12-01 Sangamo Biosciences Inc Methods of using randomized libraries of zinc finger proteins for the identification of gene function
AU5077401A (en) 2000-02-08 2001-08-20 Sangamo Biosciences Inc Cells for drug discovery
US20020061512A1 (en) 2000-02-18 2002-05-23 Kim Jin-Soo Zinc finger domains and methods of identifying same
US20030044787A1 (en) 2000-05-16 2003-03-06 Joung J. Keith Methods and compositions for interaction trap assays
JP2002060786A (ja) 2000-08-23 2002-02-26 Kao Corp 硬質表面用殺菌防汚剤
US7067317B2 (en) 2000-12-07 2006-06-27 Sangamo Biosciences, Inc. Regulation of angiogenesis with zinc finger proteins
US20050267061A1 (en) 2004-04-08 2005-12-01 Sangamo Biosciences, Inc. Methods and compositions for treating neuropathic and neurodegenerative conditions
GB0108491D0 (en) 2001-04-04 2001-05-23 Gendaq Ltd Engineering zinc fingers
WO2003016496A2 (en) 2001-08-20 2003-02-27 The Scripps Research Institute Zinc finger binding domains for cnn
US7262054B2 (en) 2002-01-22 2007-08-28 Sangamo Biosciences, Inc. Zinc finger proteins for DNA binding and gene regulation in plants
BRPI0307383B1 (pt) 2002-01-23 2019-12-31 The Univ Of Utah Research Foundation método de recombinação genética direcionada em célula de planta hospedeira
EP1504092B2 (en) 2002-03-21 2014-06-25 Sangamo BioSciences, Inc. Methods and compositions for using zinc finger endonucleases to enhance homologous recombination
US7361635B2 (en) 2002-08-29 2008-04-22 Sangamo Biosciences, Inc. Simultaneous modulation of multiple genes
AU2003298574B2 (en) 2002-09-05 2008-04-24 California Institute Of Technology Use of chimeric nucleases to stimulate gene targeting
WO2005014791A2 (en) 2003-08-08 2005-02-17 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
US7888121B2 (en) 2003-08-08 2011-02-15 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
US8409861B2 (en) 2003-08-08 2013-04-02 Sangamo Biosciences, Inc. Targeted deletion of cellular DNA sequences
US7972854B2 (en) 2004-02-05 2011-07-05 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
WO2005100392A2 (en) 2004-04-08 2005-10-27 Sangamo Biosciences, Inc. Treatment of neuropathic pain with zinc finger proteins
AU2005287278B2 (en) 2004-09-16 2011-08-04 Sangamo Biosciences, Inc. Compositions and methods for protein production
JP4988606B2 (ja) * 2005-02-28 2012-08-01 サンガモ バイオサイエンシズ インコーポレイテッド 抗血管新生方法及び組成物
US20090068164A1 (en) 2005-05-05 2009-03-12 The Ariz Bd Of Regents On Behald Of The Univ Of Az Sequence enabled reassembly (seer) - a novel method for visualizing specific dna sequences
JP2009502170A (ja) 2005-07-26 2009-01-29 サンガモ バイオサイエンシズ インコーポレイテッド 外来核酸配列の標的化された組込み及び発現
ES2626025T3 (es) 2005-10-18 2017-07-21 Precision Biosciences Meganucleasas diseñadas racionalmente con especificidad de secuencia y afinidad de unión a ADN alteradas
JP5266210B2 (ja) 2006-05-25 2013-08-21 サンガモ バイオサイエンシズ インコーポレイテッド 改変開裂ハーフドメイン
AU2007267874B2 (en) 2006-05-25 2012-03-15 Sangamo Therapeutics, Inc. Methods and compositions for gene inactivation
CA2684378C (en) 2007-04-26 2016-11-29 Sangamo Biosciences, Inc. Targeted integration into the ppp1r12c locus
US8790345B2 (en) 2007-08-21 2014-07-29 Zimmer, Inc. Titanium alloy with oxidized zirconium for a prosthetic implant
CN101883863B (zh) 2007-09-27 2018-01-23 桑格摩生物科学股份有限公司 生物活性核酸酶的快速体内鉴定
WO2009054985A1 (en) 2007-10-25 2009-04-30 Sangamo Biosciences, Inc. Methods and compositions for targeted integration
BE1018531A4 (nl) 2007-10-31 2011-03-01 Gielen Cornelis Het gebruik van niet-pathogene bacterien in sporenvorm of in slapende vorm voor het verwijderen van huismijtuitwerpselen en organische allergieverwekkende materialen van alle soorten textiel.
DE102007056956B4 (de) 2007-11-27 2009-10-29 Moosbauer, Peter, Dipl.-Ing.(FH) Schaltung zur Regelung der Stromversorgung eines Verbrauchers und Verfahren zum Betrieb einer Schaltung
JP2011518555A (ja) 2008-04-14 2011-06-30 サンガモ バイオサイエンシーズ, インコーポレイテッド 標的組込みのための線形ドナーコンストラクト
JP2011521643A (ja) 2008-05-28 2011-07-28 サンガモ バイオサイエンシーズ, インコーポレイテッド Dna結合ドメインおよび切断ドメインを連結するための組成物
AU2009283194B2 (en) 2008-08-22 2014-10-16 Sangamo Therapeutics, Inc. Methods and compositions for targeted single-stranded cleavage and targeted integration
CA2737180C (en) 2008-09-15 2019-02-19 Children's Medical Center Corporation Modulation of bcl11a for treatment of hemoglobinopathies
CN102625655B (zh) 2008-12-04 2016-07-06 桑格摩生物科学股份有限公司 使用锌指核酸酶在大鼠中进行基因组编辑
EP2206723A1 (en) 2009-01-12 2010-07-14 Bonas, Ulla Modular DNA-binding domains
US20110041195A1 (en) 2009-08-11 2011-02-17 Sangamo Biosciences, Inc. Organisms homozygous for targeted modification
SG181601A1 (en) 2009-12-10 2012-07-30 Univ Minnesota Tal effector-mediated dna modification
WO2011090804A1 (en) 2010-01-22 2011-07-28 Dow Agrosciences Llc Targeted genomic alteration
EP4328304A3 (en) 2010-02-08 2024-08-28 Sangamo Therapeutics, Inc. Engineered cleavage half-domains
WO2011100058A1 (en) 2010-02-09 2011-08-18 Sangamo Biosciences, Inc. Targeted genomic modification with partially single-stranded donor molecules
ES2773052T3 (es) 2010-05-03 2020-07-09 Sangamo Therapeutics Inc Composiciones para unir módulos de dedos de cinc
CA2798988C (en) 2010-05-17 2020-03-10 Sangamo Biosciences, Inc. Tal-effector (tale) dna-binding polypeptides and uses thereof
WO2012021632A2 (en) 2010-08-10 2012-02-16 The Johns Hopkins University Generation and use of pluripotent stem cells
US9405700B2 (en) 2010-11-04 2016-08-02 Sonics, Inc. Methods and apparatus for virtualization in an integrated circuit
WO2012073047A2 (en) 2010-12-03 2012-06-07 Genome Research Limited Compositions and methods
AU2012312260B2 (en) 2011-09-21 2017-08-31 Sangamo Therapeutics, Inc. Methods and compositions for regulation of transgene expression
EP2771457B1 (en) 2011-10-27 2017-11-22 Sangamo Therapeutics, Inc. Methods and compositions for modification of the hprt locus
BR112014020625A2 (pt) * 2012-02-24 2017-07-04 Hutchinson Fred Cancer Res polinucleotídeo, polipeptídeo, composição, célula e célula tronco editada por genoma
HK1208051A1 (en) 2012-05-07 2016-02-19 桑格摩生物科学股份有限公司 Methods and compositions for nuclease-mediated targeted integration of transgenes
WO2014186585A2 (en) * 2013-05-15 2014-11-20 Sangamo Biosciences, Inc. Methods and compositions for treatment of a genetic condition

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