LT2890780T - Būdai ir kompozicijos, skirti genetinės būklės gydymui - Google Patents

Būdai ir kompozicijos, skirti genetinės būklės gydymui

Info

Publication number
LT2890780T
LT2890780T LTEP13833658.1T LT13833658T LT2890780T LT 2890780 T LT2890780 T LT 2890780T LT 13833658 T LT13833658 T LT 13833658T LT 2890780 T LT2890780 T LT 2890780T
Authority
LT
Lithuania
Prior art keywords
compositions
treatment
methods
genetic condition
genetic
Prior art date
Application number
LTEP13833658.1T
Other languages
English (en)
Lithuanian (lt)
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.)
Filing date
Publication date
Application filed by Sangamo Therapeutics, Inc. filed Critical Sangamo Therapeutics, Inc.
Publication of LT2890780T publication Critical patent/LT2890780T/lt

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/06—Antianaemics
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/795—Porphyrin- or corrin-ring-containing peptides
    • C07K14/805—Haemoglobins; Myoglobins
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/52—Genes encoding for enzymes or proenzymes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/62—DNA sequences coding for fusion proteins
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0634—Cells from the blood or the immune system
    • C12N5/0647—Haematopoietic stem cells; Uncommitted or multipotent progenitors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • C07K2319/81—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor containing a Zn-finger domain for DNA binding
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2510/00—Genetically modified cells
    • C12N2510/02—Cells for production

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • 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)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (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)
LTEP13833658.1T 2012-08-29 2013-08-29 Būdai ir kompozicijos, skirti genetinės būklės gydymui LT2890780T (lt)

Applications Claiming Priority (2)

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

Publications (1)

Publication Number Publication Date
LT2890780T true LT2890780T (lt) 2020-11-10

Family

ID=50184630

Family Applications (1)

Application Number Title Priority Date Filing Date
LTEP13833658.1T LT2890780T (lt) 2012-08-29 2013-08-29 Būdai ir kompozicijos, skirti genetinės būklės gydymui

Country Status (26)

Country Link
US (4) US9650648B2 (en:Method)
EP (1) EP2890780B8 (en:Method)
JP (4) JP2015533786A (en:Method)
KR (3) KR102474010B1 (en:Method)
CN (1) CN104704110B (en:Method)
AU (1) AU2013308770B2 (en:Method)
CA (2) CA2882499C (en:Method)
CL (1) CL2015000450A1 (en:Method)
DK (1) DK2890780T3 (en:Method)
EA (1) EA039384B1 (en:Method)
ES (1) ES2812599T3 (en:Method)
HR (1) HRP20201443T1 (en:Method)
HU (1) HUE050516T2 (en:Method)
IL (1) IL237050B (en:Method)
IN (1) IN2015DN01480A (en:Method)
LT (1) LT2890780T (en:Method)
MX (1) MX367081B (en:Method)
PH (3) PH12021551661A1 (en:Method)
PL (1) PL2890780T3 (en:Method)
PT (1) PT2890780T (en:Method)
RS (1) RS60838B1 (en:Method)
SG (2) SG10201701601WA (en:Method)
SI (1) SI2890780T1 (en:Method)
UA (1) UA118957C2 (en:Method)
WO (1) WO2014036219A2 (en:Method)
ZA (1) ZA201500900B (en:Method)

Families Citing this family (127)

* 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
JP6170080B2 (ja) 2012-02-24 2017-07-26 フレッド ハッチンソン キャンサー リサーチ センター 異常ヘモグロビン症の治療のための組成物および方法
LT3401400T (lt) 2012-05-25 2019-06-10 The Regents Of The University Of California Būdai ir kompozicijos, skirtos rnr molekulės nukreipiamai tikslinės dnr modifikacijai ir rnr molekulės nukreipiamam transkripcijos moduliavimui
DE202013012610U1 (de) * 2012-10-23 2017-11-24 Toolgen, Inc. Zusammensetzung zum Spalten einer Ziel-DNA, umfassend eine für die Ziel-DNA spezifische guide-RNA und eine Cas-Protein-codierende Nukleinsäure oder ein Cas-Protein, sowie deren Verwendung
DK3138912T3 (en) 2012-12-06 2019-01-21 Sigma Aldrich Co Llc CRISPR-BASED RE-MODIFICATION AND REGULATION
JP6491113B2 (ja) 2013-02-25 2019-03-27 サンガモ セラピューティクス， インコーポレイテッド ヌクレアーゼ媒介性遺伝子破壊を増強するための方法および組成物
US20140315985A1 (en) 2013-03-14 2014-10-23 Caribou Biosciences, Inc. Compositions and methods of nucleic acid-targeting nucleic acids
JP2016522679A (ja) 2013-04-04 2016-08-04 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 ＣＲＩＳＰＲ／Ｃａｓ系を用いたゲノム編集の治療的使用
CA2910489A1 (en) 2013-05-15 2014-11-20 Sangamo Biosciences, Inc. Methods and compositions for treatment of a genetic condition
ES2991293T3 (es) * 2013-06-05 2024-12-03 Univ Duke Edición génica guiada por ARN y regulación génica
JP2016528890A (ja) 2013-07-09 2016-09-23 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 ＣＲＩＳＰＲ／Ｃａｓ系を用いるゲノム編集の治療用の使用
US9163284B2 (en) 2013-08-09 2015-10-20 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
EP3039136B8 (en) 2013-08-28 2020-12-16 Sangamo Therapeutics, 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
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
KR102523466B1 (ko) 2013-11-07 2023-04-20 에디타스 메디신, 인코포레이티드 지배적인 gRNA를 이용하는 CRISPR-관련 방법 및 조성물
LT3068881T (lt) 2013-11-13 2019-08-12 Children`S Medical Center Corporation Geno raiškos reguliavimas, tarpininkaujant nukleazei
US9840699B2 (en) 2013-12-12 2017-12-12 President And Fellows Of Harvard College Methods for nucleic acid editing
HUE051232T2 (hu) * 2014-02-03 2021-03-01 Sangamo Therapeutics Inc Béta-talasszémia kezelésére szolgáló eljárások és készítmények
CA2939942A1 (en) 2014-02-24 2015-08-27 Sangamo Biosciences, Inc. Methods and compositions for nuclease-mediated targeted integration
EP3114227B1 (en) 2014-03-05 2021-07-21 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating usher syndrome and retinitis pigmentosa
DK3116997T3 (da) 2014-03-10 2019-08-19 Editas Medicine Inc Crispr/cas-relaterede fremgangsmåder og sammensætninger til behandling af lebers kongenitale amaurose 10 (lca10)
US11141493B2 (en) 2014-03-10 2021-10-12 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
US11339437B2 (en) 2014-03-10 2022-05-24 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
WO2015143046A2 (en) 2014-03-18 2015-09-24 Sangamo Biosciences, Inc. Methods and compositions for regulation of zinc finger protein expression
WO2015148863A2 (en) * 2014-03-26 2015-10-01 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating sickle cell disease
US20170173083A1 (en) * 2014-03-26 2017-06-22 The Brigham And Women's Hospital, Inc. Compositions and methods for ex vivo expansion of human hematopoietic stem/progenitor cells
WO2015148860A1 (en) * 2014-03-26 2015-10-01 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating beta-thalassemia
WO2015153780A1 (en) 2014-04-02 2015-10-08 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating primary open angle glaucoma
AU2015298571B2 (en) 2014-07-30 2020-09-03 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
CA2959070C (en) 2014-08-27 2020-11-10 Caribou Biosciences, Inc. Methods for increasing cas9-mediated engineering efficiency
DK3194570T3 (da) * 2014-09-16 2021-09-13 Sangamo Therapeutics Inc Fremgangsmåder og sammensætninger til nukleasemedieret genommodificering og -korrektion i hæmatopoietiske stamceller
EP3215623A4 (en) 2014-11-06 2018-09-26 President and Fellows of Harvard College Cells lacking b2m surface expression and methods for allogeneic administration of such cells
JP2017536826A (ja) * 2014-11-24 2017-12-14 チルドレンズ メディカル センター コーポレーション 幹細胞および／または前駆細胞からの赤血球産生を改善するためのｓｈ２ｂ３の調節
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)
EP3262173A2 (en) * 2015-02-23 2018-01-03 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
US10395760B2 (en) 2015-04-13 2019-08-27 Invitae Corporation Methods, systems and processes of identifying genetic variation in highly similar genes
CA2982966C (en) 2015-04-24 2024-02-20 Editas Medicine, Inc. Evaluation of cas9 molecule/guide rna molecule complexes
US10179918B2 (en) 2015-05-07 2019-01-15 Sangamo Therapeutics, Inc. Methods and compositions for increasing transgene activity
CN107921148A (zh) 2015-05-08 2018-04-17 哈佛学院校长同事会 通用供体干细胞和相关方法
JP6873917B2 (ja) 2015-05-12 2021-05-19 サンガモ セラピューティクス， インコーポレイテッド ヌクレアーゼ介在性遺伝子発現調節
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
MX391052B (es) * 2015-09-23 2025-03-21 Sangamo Therapeutics Inc Represores de htt y usos de estos.
AU2016341041A1 (en) * 2015-10-20 2018-03-15 Pioneer Hi-Bred International, Inc. Methods and compositions for marker-free genome modification
IL294014B2 (en) 2015-10-23 2024-07-01 Harvard College Nucleobase editors and uses thereof
EP3368664A4 (en) 2015-10-28 2019-08-28 Sangamo Therapeutics, Inc. LIFE-SPECIFIC CONSTRUCTS, FACTOR VIII EXPRESSION CASSETTES AND METHOD OF USE THEREOF
WO2017077394A2 (en) * 2015-11-04 2017-05-11 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
ES2905558T3 (es) 2015-11-13 2022-04-11 Avellino Lab Usa Inc Procedimientos para el tratamiento de las distrofias corneales
EP3390437B1 (en) * 2015-12-18 2025-02-26 Sangamo Therapeutics, Inc. Targeted disruption of the mhc cell receptor
US20190249172A1 (en) 2016-02-18 2019-08-15 The Regents Of The University Of California Methods and compositions for gene editing in stem cells
JP2019508051A (ja) * 2016-03-14 2019-03-28 エディタス・メディシン、インコーポレイテッド β異常ヘモグロビン症を治療するためのＣＲＩＳＰＲ／ＣＡＳ関連方法および組成物
EP3433364A1 (en) 2016-03-25 2019-01-30 Editas Medicine, Inc. Systems and methods for treating alpha 1-antitrypsin (a1at) deficiency
US11566263B2 (en) 2016-08-02 2023-01-31 Editas Medicine, Inc. Compositions and methods for treating CEP290 associated disease
KR102827276B1 (ko) 2016-08-03 2025-07-01 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
CN109804066A (zh) 2016-08-09 2019-05-24 哈佛大学的校长及成员们 可编程cas9-重组酶融合蛋白及其用途
WO2020225754A1 (en) 2019-05-06 2020-11-12 Mcmullen Tara Crispr gene editing for autosomal dominant diseases
EP3500677A4 (en) 2016-08-20 2020-04-01 Avellino Lab USA, Inc. Single guide rna, crispr/cas9 systems, and methods of use thereof
JP7272948B2 (ja) 2016-08-24 2023-05-12 サンガモ セラピューティクス， インコーポレイテッド 操作された標的特異的ヌクレアーゼ
SI3504229T1 (sl) * 2016-08-24 2022-04-29 Sangamo Therapeutics, Inc. Regulacija izražanja genov z uporabo modificiranih nukleaz
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
WO2018049009A2 (en) 2016-09-07 2018-03-15 Sangamo Therapeutics, Inc. Modulation of liver genes
WO2018071663A1 (en) * 2016-10-14 2018-04-19 Emendobio Inc. Rna compositions for genome editing
KR20240007715A (ko) 2016-10-14 2024-01-16 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵염기 에디터의 aav 전달
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
WO2018165629A1 (en) 2017-03-10 2018-09-13 President And Fellows Of Harvard College Cytosine to guanine base editor
WO2018170184A1 (en) 2017-03-14 2018-09-20 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
KR20240116572A (ko) 2017-03-23 2024-07-29 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제
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
EP3622070A2 (en) 2017-05-10 2020-03-18 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
CA3070242A1 (en) * 2017-07-18 2019-01-24 Calimmune, Inc. Compositions and methods for treating beta-hemoglobinopathies
WO2019023680A1 (en) 2017-07-28 2019-01-31 President And Fellows Of Harvard College METHODS AND COMPOSITIONS FOR EVOLUTION OF BASIC EDITORS USING PHAGE-ASSISTED CONTINUOUS EVOLUTION (PACE)
WO2019139645A2 (en) 2017-08-30 2019-07-18 President And Fellows Of Harvard College High efficiency base editors comprising gam
CN111757937A (zh) 2017-10-16 2020-10-09 布罗德研究所股份有限公司 腺苷碱基编辑器的用途
US20210180091A1 (en) 2017-10-26 2021-06-17 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of hemoglobinopathies
KR102730214B1 (ko) * 2017-11-09 2024-11-13 상가모 테라퓨틱스, 인코포레이티드 시토카인 유도성 sh2-함유 단백질 (cish) 유전자의 유전자 변형
EP3724214A4 (en) 2017-12-15 2021-09-01 The Broad Institute Inc. SYSTEMS AND METHODS FOR PREDICTING REPAIR RESULTS 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
WO2019157324A1 (en) * 2018-02-08 2019-08-15 Sangamo Therapeutics, Inc. Engineered target specific nucleases
KR20200123201A (ko) 2018-02-21 2020-10-28 알시온 라이프사이언스 인크. 유체 전달 시스템 및 방법
WO2019178426A1 (en) 2018-03-14 2019-09-19 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
US12133884B2 (en) 2018-05-11 2024-11-05 Beam Therapeutics Inc. Methods of substituting pathogenic amino acids using programmable base editor systems
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
EP3820495A4 (en) 2018-07-09 2022-07-20 The Broad Institute Inc. RNA PROGRAMMABLE EPIGENETIC RNA MODIFIERS AND THEIR USES
CN112867529B (zh) 2018-08-27 2023-06-02 亚克安娜医疗有限公司 流体递送系统和方法
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
JP7657726B2 (ja) 2019-03-19 2025-04-07 ザ ブロード インスティテュート，インコーポレーテッド 編集ヌクレオチド配列を編集するための方法および組成物
AU2020253362A1 (en) 2019-04-02 2021-11-04 Sangamo Therapeutics, Inc. Methods for the treatment of beta-thalassemia
US20220184137A1 (en) * 2019-04-12 2022-06-16 INSERM (Institut National de la Santé et de la Recherche Médicale) Correction of Beta-Thalassemia Phenotype by Genetically Engineered Hematopoietic Stem Cell
WO2020214842A1 (en) 2019-04-17 2020-10-22 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
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
EP4007610A4 (en) * 2019-08-01 2023-09-13 The Regents of the University of California COMPOSITIONS AND METHODS OF TREATMENT OF ALPHA-THALASSEMIA
WO2021026336A2 (en) * 2019-08-07 2021-02-11 I Altius Institute For Biomedical Sciences Compositions and methods for modulation of gene expression
JP2022548399A (ja) * 2019-09-23 2022-11-18 オメガ セラピューティクス， インコーポレイテッド 肝細胞核因子４－アルファ（ＨＮＦ４α）遺伝子発現をモジュレートするための組成物および方法
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
EP4045649A4 (en) * 2019-10-15 2023-11-22 Altius Institute For Biomedical Sciences DNA-BINDING PROTEINS FOR DISPLACING ENDOGENOUS TRANSCRIPTION FACTORS LINKED TO REGULATORY REGIONS OF GENES
WO2021216622A1 (en) 2020-04-21 2021-10-28 Aspen Neuroscience, Inc. Gene editing of gba1 in stem cells and method of use of cells differentiated therefrom
US20230165909A1 (en) 2020-04-21 2023-06-01 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
WO2023115041A1 (en) 2021-12-17 2023-06-22 Sana Biotechnology, Inc. Modified paramyxoviridae attachment glycoproteins
TW202342498A (zh) 2021-12-17 2023-11-01 美商薩那生物科技公司 經修飾副黏液病毒科融合醣蛋白
US20250082777A1 (en) 2022-01-10 2025-03-13 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
EP4473097A1 (en) 2022-02-02 2024-12-11 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
EP4590688A1 (en) 2022-09-21 2025-07-30 Sana Biotechnology, Inc. Lipid particles comprising variant paramyxovirus attachment glycoproteins and uses thereof
EP4602174A1 (en) 2022-10-13 2025-08-20 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
WO2024220560A1 (en) 2023-04-18 2024-10-24 Sana Biotechnology, Inc. Engineered protein g fusogens and related lipid particles and methods thereof
WO2024220598A2 (en) 2023-04-18 2024-10-24 Sana Biotechnology, Inc. Lentiviral vectors with two or more genomes
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
AU2024274792A1 (en) 2023-05-23 2025-10-23 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
WO2025151838A1 (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
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
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
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
US5436150A (en) 1992-04-03 1995-07-25 The Johns Hopkins University Functional domains in flavobacterium okeanokoities (foki) restriction endonuclease
US5356802A (en) 1992-04-03 1994-10-18 The Johns Hopkins University Functional domains in flavobacterium okeanokoites (FokI) restriction endonuclease
US5487994A (en) 1992-04-03 1996-01-30 The Johns Hopkins University Insertion and deletion mutants of 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
CA2181548C (en) 1994-01-18 2009-11-03 Carlos F. Barbas, Iii Zinc finger protein derivatives and methods therefor
US6140466A (en) 1994-01-18 2000-10-31 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
AU696455C (en) 1994-03-23 2006-03-02 Case Western Reserve 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
USRE45795E1 (en) 1994-08-20 2015-11-10 Gendaq, Ltd. 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
GB9703369D0 (en) 1997-02-18 1997-04-09 Lindqvist Bjorn H Process
GB2338237B (en) 1997-02-18 2001-02-28 Actinova Ltd In vitro peptide or protein expression library
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
GB9710807D0 (en) 1997-05-23 1997-07-23 Medical Res Council Nucleic acid binding proteins
GB9710809D0 (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
ATE466952T1 (de) 1998-03-02 2010-05-15 Massachusetts Inst Technology Poly-zinkfinger-proteine mit verbesserten linkern
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
US7070934B2 (en) 1999-01-12 2006-07-04 Sangamo Biosciences, Inc. Ligand-controlled regulation of endogenous gene expression
US6599692B1 (en) 1999-09-14 2003-07-29 Sangamo Bioscience, Inc. Functional genomics using zinc finger proteins
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
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
US7013219B2 (en) 1999-01-12 2006-03-14 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
WO2000046386A2 (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
US6794136B1 (en) 2000-11-20 2004-09-21 Sangamo Biosciences, Inc. Iterative optimization in the design of binding proteins
US7030215B2 (en) 1999-03-24 2006-04-18 Sangamo Biosciences, Inc. Position dependent recognition of GNN nucleotide triplets by zinc fingers
EP1236045B1 (en) 1999-12-06 2005-11-09 Sangamo Biosciences Inc. Methods of using randomized libraries of zinc finger proteins for the identification of gene function
WO2001059450A2 (en) 2000-02-08 2001-08-16 Sangamo Biosciences, Inc. Cells expressing zinc finger protein for drug discovery
US20020061512A1 (en) 2000-02-18 2002-05-23 Kim Jin-Soo Zinc finger domains and methods of identifying same
WO2001088197A2 (en) 2000-05-16 2001-11-22 Massachusetts Institute Of Technology 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
GB0108491D0 (en) 2001-04-04 2001-05-23 Gendaq Ltd Engineering zinc fingers
JP2005500061A (ja) 2001-08-20 2005-01-06 ザ スクリップス リサーチ インスティテュート Ｃｎｎについての亜鉛フィンガー結合ドメイン
US7262054B2 (en) 2002-01-22 2007-08-28 Sangamo Biosciences, Inc. Zinc finger proteins for DNA binding and gene regulation in plants
CN100575485C (zh) 2002-01-23 2009-12-30 犹他大学研究基金会 使用锌指核酸酶的定向染色体诱变
EP2368982A3 (en) 2002-03-21 2011-10-12 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
EP2806025B1 (en) 2002-09-05 2019-04-03 California Institute of Technology Use of zinc finger nucleases to stimulate gene targeting
US8409861B2 (en) 2003-08-08 2013-04-02 Sangamo Biosciences, Inc. Targeted deletion of cellular DNA sequences
US7888121B2 (en) 2003-08-08 2011-02-15 Sangamo Biosciences, Inc. Methods and compositions for targeted cleavage and recombination
AU2004263865B2 (en) 2003-08-08 2007-05-17 Sangamo Therapeutics, Inc. Methods and compositions for targeted cleavage and recombination
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
AU2005232665B2 (en) 2004-04-08 2010-05-13 Sangamo Therapeutics, Inc. Methods and compositions for treating neuropathic and neurodegenerative conditions
EP1789095A2 (en) 2004-09-16 2007-05-30 Sangamo Biosciences Inc. Compositions and methods for protein production
EP1869075B1 (en) * 2005-02-28 2012-04-11 Sangamo BioSciences, Inc. Anti-angiogenic methods and compositions
MX2007013757A (es) 2005-05-05 2008-01-24 Univ Arizona Reagrupacion permitida de secuencia (seer)-met odo de novedad para visualizar secuencias de adn especificas.
CN101273141B (zh) 2005-07-26 2013-03-27 桑格摩生物科学股份有限公司 外源核酸序列的靶向整合和表达
ES2440801T3 (es) 2005-10-18 2014-01-30 Precision Biosciences Meganucleasas racionalmente diseñadas con especificidad de secuencia y afinidad de unión a ADN alteradas
EP2447279B1 (en) 2006-05-25 2014-04-09 Sangamo BioSciences, Inc. Methods and compositions for gene inactivation
WO2007139898A2 (en) 2006-05-25 2007-12-06 Sangamo Biosciences, Inc. Variant foki cleavage half-domains
ATE489465T1 (de) 2007-04-26 2010-12-15 Sangamo Biosciences Inc Gezielte integration in die ppp1r12c-position
US8790345B2 (en) 2007-08-21 2014-07-29 Zimmer, Inc. Titanium alloy with oxidized zirconium for a prosthetic implant
EP2188384B1 (en) 2007-09-27 2015-07-15 Sangamo BioSciences, Inc. Rapid in vivo identification of biologically active nucleases
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
ES2459880T3 (es) 2008-04-14 2014-05-12 Sangamo Biosciences, Inc. Construcciones donantes lineales para integración dirigida
AU2009260888B2 (en) 2008-05-28 2014-09-11 Sangamo Therapeutics, Inc. Compositions for linking DNA-binding domains and cleavage domains
SG191561A1 (en) 2008-08-22 2013-07-31 Sangamo Biosciences Inc Methods and compositions for targeted single-stranded cleavage and targeted integration
DK2334794T3 (en) * 2008-09-15 2017-02-20 Children's Medical Center Corp MODULATION OF BCL11A FOR TREATMENT OF HEMOGLOBINOPATHIES
KR101803737B1 (ko) 2008-12-04 2017-12-01 상가모 테라퓨틱스, 인코포레이티드 징크 핑거 뉴클레아제를 이용한 랫트의 유전체 편집
EP2206723A1 (en) 2009-01-12 2010-07-14 Bonas, Ulla Modular DNA-binding domains
BR112012003257A2 (pt) 2009-08-11 2016-11-22 Sangamo Biosciences Inc organismos homozigotos para modificação direcionada
US8586526B2 (en) 2010-05-17 2013-11-19 Sangamo Biosciences, Inc. DNA-binding proteins and uses thereof
PT2510096E (pt) 2009-12-10 2015-02-04 Univ Iowa State Res Found Inc Modificação do adn modificada pelo efector tal
CN102812034B (zh) 2010-01-22 2016-08-03 陶氏益农公司 靶向基因组改造
AU2011213242B2 (en) * 2010-02-08 2015-01-29 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
CN102959087B (zh) 2010-05-03 2015-04-29 桑格摩生物科学股份有限公司 用于连接锌指模块的组合物
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
CA2848417C (en) 2011-09-21 2023-05-02 Sangamo Biosciences, Inc. Methods and compositions for regulation of transgene expression
WO2013063315A2 (en) 2011-10-27 2013-05-02 Sangamo Biosciences, Inc. Methods and compositions for modification of the hprt locus
JP6170080B2 (ja) * 2012-02-24 2017-07-26 フレッド ハッチンソン キャンサー リサーチ センター 異常ヘモグロビン症の治療のための組成物および方法
US10174331B2 (en) 2012-05-07 2019-01-08 Sangamo Therapeutics, Inc. Methods and compositions for nuclease-mediated targeted integration of transgenes
CA2910489A1 (en) 2013-05-15 2014-11-20 Sangamo Biosciences, Inc. Methods and compositions for treatment of a genetic condition

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