CA2865129A1 - Compositions and methods for the treatment of hemoglobinopathies - Google Patents

Compositions and methods for the treatment of hemoglobinopathies Download PDF

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Publication number
CA2865129A1
CA2865129A1 CA2865129A CA2865129A CA2865129A1 CA 2865129 A1 CA2865129 A1 CA 2865129A1 CA 2865129 A CA2865129 A CA 2865129A CA 2865129 A CA2865129 A CA 2865129A CA 2865129 A1 CA2865129 A1 CA 2865129A1
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CA
Canada
Prior art keywords
seq
endonuclease
homing endonuclease
amino acid
nucleotide sequence
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Abandoned
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CA2865129A
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English (en)
French (fr)
Inventor
Michael A. Bender
Mark T. Groudine
Barry L. Stoddard
Ryo Takeuchi
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Fred Hutchinson Cancer Center
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Fred Hutchinson Cancer Center
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Application filed by Fred Hutchinson Cancer Center filed Critical Fred Hutchinson Cancer Center
Publication of CA2865129A1 publication Critical patent/CA2865129A1/en
Abandoned legal-status Critical Current

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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
    • 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]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/06—Antianaemics
    • 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)
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Microbiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • General Chemical & Material Sciences (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
CA2865129A 2012-02-24 2013-02-22 Compositions and methods for the treatment of hemoglobinopathies Abandoned CA2865129A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201261603231P 2012-02-24 2012-02-24
US61/603,231 2012-02-24
PCT/US2013/027459 WO2013126794A1 (en) 2012-02-24 2013-02-22 Compositions and methods for the treatment of hemoglobinopathies

Publications (1)

Publication Number Publication Date
CA2865129A1 true CA2865129A1 (en) 2013-08-29

Family

ID=49006265

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2865129A Abandoned CA2865129A1 (en) 2012-02-24 2013-02-22 Compositions and methods for the treatment of hemoglobinopathies

Country Status (18)

Country Link
US (2) US20150166969A1 (enExample)
EP (2) EP3272356A1 (enExample)
JP (2) JP6170080B2 (enExample)
KR (2) KR20180009383A (enExample)
CN (1) CN104284669A (enExample)
AU (2) AU2013222170B2 (enExample)
BR (1) BR112014020625A2 (enExample)
CA (1) CA2865129A1 (enExample)
DK (1) DK2836226T3 (enExample)
ES (1) ES2641840T3 (enExample)
HK (1) HK1249861A1 (enExample)
IL (2) IL234225B (enExample)
IN (1) IN2014DN07853A (enExample)
NZ (1) NZ630900A (enExample)
RU (2) RU2018109507A (enExample)
SG (2) SG11201405103SA (enExample)
WO (1) WO2013126794A1 (enExample)
ZA (1) ZA201406187B (enExample)

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN112469823A (zh) * 2018-04-27 2021-03-09 西雅图儿童医院d/b/a西雅图儿童研究所 布鲁顿氏酪氨酸激酶的基于talen和基于crispr/cas的基因编辑
CN114457119A (zh) * 2022-04-11 2022-05-10 中吉智药(南京)生物技术有限公司 慢病毒载体在制备治疗β-地中海贫血药物中的应用

Families Citing this family (187)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10323236B2 (en) 2011-07-22 2019-06-18 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
CA2850509C (en) 2011-10-14 2023-08-01 President And Fellows Of Harvard College Sequencing by structure assembly
WO2014163886A1 (en) 2013-03-12 2014-10-09 President And Fellows Of Harvard College Method of generating a three-dimensional nucleic acid containing matrix
ES2991004T3 (es) 2011-12-22 2024-12-02 Harvard College Métodos para la detección de analitos
GB201122458D0 (en) 2011-12-30 2012-02-08 Univ Wageningen Modified cascade ribonucleoproteins and uses thereof
RU2018109507A (ru) 2012-02-24 2019-02-28 Фред Хатчинсон Кэнсер Рисерч Сентер Композиции и способы лечения гемоглобинопатии
WO2013176772A1 (en) 2012-05-25 2013-11-28 The Regents Of The University Of California Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
RS60838B1 (sr) 2012-08-29 2020-10-30 Sangamo Therapeutics Inc Postupci i kompozicije za tretman genskog stanja
KR101656236B1 (ko) * 2012-10-23 2016-09-12 주식회사 툴젠 표적 DNA에 특이적인 가이드 RNA 및 Cas 단백질을 암호화하는 핵산 또는 Cas 단백질을 포함하는, 표적 DNA를 절단하기 위한 조성물 및 이의 용도
PT3502240T (pt) 2012-11-27 2021-08-11 Childrens Medical Ct Corp Elementos reguladores distais de bcl11a como alvo para a reindução de hemoglobina fetal
ES2713243T3 (es) 2012-12-06 2019-05-20 Sigma Aldrich Co Llc Modificación y regulación del genoma basada en CRISPR
AU2013359199C1 (en) 2012-12-12 2021-06-17 Massachusetts Institute Of Technology Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
EP4481048A3 (en) 2012-12-17 2025-02-26 President and Fellows of Harvard College Rna-guided human genome engineering
ES2901396T3 (es) 2013-03-14 2022-03-22 Caribou Biosciences Inc Composiciones y métodos de ácidos nucleicos dirigidos a ácido nucleico
US10760064B2 (en) 2013-03-15 2020-09-01 The General Hospital Corporation RNA-guided targeting of genetic and epigenomic regulatory proteins to specific genomic loci
AU2014228981B2 (en) 2013-03-15 2019-11-28 The General Hospital Corporation Using truncated guide RNAs (tru-gRNAs) to increase specificity for RNA-guided genome editing
WO2014204578A1 (en) 2013-06-21 2014-12-24 The General Hospital Corporation Using rna-guided foki nucleases (rfns) to increase specificity for rna-guided genome editing
WO2014165825A2 (en) 2013-04-04 2014-10-09 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
KR102884236B1 (ko) 2013-06-04 2025-11-12 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 Rna-가이드된 전사 조절
US20140356956A1 (en) 2013-06-04 2014-12-04 President And Fellows Of Harvard College RNA-Guided Transcriptional Regulation
SG11201510284XA (en) 2013-06-17 2016-01-28 Broad Inst Inc Delivery and use of the crispr-cas systems, vectors and compositions for hepatic targeting and therapy
RU2716421C2 (ru) 2013-06-17 2020-03-11 Те Брод Инститьют Инк. Доставка, применение и применения в терапии систем crispr-cas и композиций для целенаправленного воздействия на нарушения и заболевания с использованием вирусных компонентов
CA2915845A1 (en) 2013-06-17 2014-12-24 The Broad Institute, Inc. Delivery, engineering and optimization of systems, methods and compositions for targeting and modeling diseases and disorders of post mitotic cells
WO2015006498A2 (en) 2013-07-09 2015-01-15 President And Fellows Of Harvard College Therapeutic uses of genome editing with crispr/cas systems
CN105517579B (zh) 2013-07-10 2019-11-15 哈佛大学校长及研究员协会 用于RNA向导的基因调节和编辑的正交Cas9蛋白
US11306328B2 (en) 2013-07-26 2022-04-19 President And Fellows Of Harvard College Genome engineering
US9163284B2 (en) 2013-08-09 2015-10-20 President And Fellows Of Harvard College Methods for identifying a target site of a Cas9 nuclease
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI 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
EP4372090A3 (en) 2013-11-07 2024-08-07 Editas Medicine, Inc. Crispr-related methods and compositions with governing grnas
HRP20211706T1 (hr) * 2013-11-13 2022-02-04 The Children's Medical Center Corporation Nukleazom posredovana regulacija ekspresije gena
EP3071695A2 (en) * 2013-11-18 2016-09-28 Crispr Therapeutics AG Crispr-cas system materials and methods
US9074199B1 (en) 2013-11-19 2015-07-07 President And Fellows Of Harvard College Mutant Cas9 proteins
US10787684B2 (en) 2013-11-19 2020-09-29 President And Fellows Of Harvard College Large gene excision and insertion
US11053481B2 (en) 2013-12-12 2021-07-06 President And Fellows Of Harvard College Fusions of Cas9 domains and nucleic acid-editing domains
EP3470089A1 (en) 2013-12-12 2019-04-17 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using particle delivery components
KR20250068794A (ko) 2013-12-12 2025-05-16 더 브로드 인스티튜트, 인코퍼레이티드 게놈 편집을 위한 ｃｒｉｓｐｒ-ｃａｓ 시스템 및 조성물의 전달, 용도 및 치료적 응용
EP3089989B1 (en) * 2013-12-31 2020-06-24 The Regents of The University of California Cas9 crystals and methods of use thereof
EP3092310B1 (en) 2014-01-08 2019-12-25 President and Fellows of Harvard College Rna-guided gene drives
JP6416939B2 (ja) 2014-02-13 2018-10-31 タカラ バイオ ユーエスエー，インコーポレイティド 核酸の初期収集物から標的分子を減損させる方法、並びにそれを実施するための組成物及びキット
US11141493B2 (en) 2014-03-10 2021-10-12 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
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)
US11339437B2 (en) 2014-03-10 2022-05-24 Editas Medicine, Inc. Compositions and methods for treating CEP290-associated disease
US11242525B2 (en) 2014-03-26 2022-02-08 Editas Medicine, Inc. CRISPR/CAS-related methods and compositions for treating sickle cell disease
CN112063586A (zh) * 2014-03-26 2020-12-11 布里格姆及妇女医院股份有限公司 用于人造血干/祖细胞的离体扩增的组合物和方法
WO2015148860A1 (en) * 2014-03-26 2015-10-01 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating beta-thalassemia
JP6514717B2 (ja) 2014-04-25 2019-05-15 ザ チルドレンズ メディカル センター コーポレーション 異常ヘモグロビン症を治療するための組成物および方法
WO2016007839A1 (en) 2014-07-11 2016-01-14 President And Fellows Of Harvard College Methods for high-throughput labelling and detection of biological features in situ using microscopy
EP4079847A1 (en) 2014-07-30 2022-10-26 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
WO2016025759A1 (en) * 2014-08-14 2016-02-18 Shen Yuelei Dna knock-in system
CN106714826A (zh) 2014-09-07 2017-05-24 西莱克塔生物科技公司 用于减弱基因表达调节抗病毒转移载体免疫应答的方法和组合物
JP6722176B2 (ja) * 2014-09-16 2020-07-15 サンガモ セラピューティクス， インコーポレイテッド 造血幹細胞におけるヌクレアーゼ媒介性ゲノム工学および補正のための方法および組成物
US10370673B2 (en) 2014-09-26 2019-08-06 Purecircle Sdn Bhd Single nucleotide polymorphism (SNP) markers for stevia
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
KR102763527B1 (ko) 2014-12-03 2025-02-05 애질런트 테크놀로지스, 인크. 화학적 변형을 갖는 가이드 rna
WO2016094874A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Escorted and functionalized guides for crispr-cas systems
WO2016094872A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Dead guides for crispr transcription factors
EP3985115A1 (en) 2014-12-12 2022-04-20 The Broad Institute, Inc. Protected guide rnas (pgrnas)
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)
CA2970370A1 (en) 2014-12-24 2016-06-30 Massachusetts Institute Of Technology Crispr having or associated with destabilization domains
WO2016118726A2 (en) 2015-01-21 2016-07-28 Sangamo Biosciences, Inc. Methods and compositions for identification of highly specific nucleases
JP6800859B2 (ja) 2015-01-26 2020-12-16 フェイト セラピューティクス，インコーポレイテッド 造血細胞分化を誘導するための方法および組成物
WO2016135558A2 (en) * 2015-02-23 2016-09-01 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
US12129471B2 (en) 2015-02-23 2024-10-29 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of human genetic diseases including hemoglobinopathies
KR102648489B1 (ko) 2015-04-06 2024-03-15 더 보드 어브 트러스티스 어브 더 리랜드 스탠포드 주니어 유니버시티 Crispr/cas-매개 유전자 조절을 위한 화학적으로 변형된 가이드 rna
US10155938B2 (en) * 2015-04-14 2018-12-18 City Of Hope Coexpression of CAS9 and TREX2 for targeted mutagenesis
CN104805118A (zh) * 2015-04-22 2015-07-29 扬州大学 一种苏禽黄鸡胚胎干细胞特定基因进行靶向敲除方法
US11845928B2 (en) * 2015-05-04 2023-12-19 Tsinghua University Methods and kits for fragmenting DNA
AU2016261600B2 (en) 2015-05-08 2021-09-23 President And Fellows Of Harvard College Universal donor stem cells and related methods
ES2835861T5 (en) * 2015-05-08 2025-02-18 Childrens Medical Ct Corp Targeting bcl11a enhancer functional regions for fetal hemoglobin reinduction
JP7030522B2 (ja) * 2015-05-11 2022-03-07 エディタス・メディシン、インコーポレイテッド 幹細胞における遺伝子編集のための最適化ｃｒｉｓｐｒ／ｃａｓ９システムおよび方法
HK1251011A1 (zh) * 2015-05-12 2019-01-18 Sangamo Therapeutics, Inc. 核酸酶介导的基因表达调控
JP7396783B2 (ja) 2015-06-09 2023-12-12 エディタス・メディシン、インコーポレイテッド 移植を改善するためのｃｒｉｓｐｒ／ｃａｓ関連方法および組成物
JP2018518181A (ja) 2015-06-17 2018-07-12 ザ ユーエービー リサーチ ファンデーション 血液細胞系列の細胞に機能的ポリペプチドを導入するためのＣＲＩＳＰＲ／Ｃａｓ９複合体
CA2989831A1 (en) * 2015-06-17 2016-12-22 The Uab Research Foundation Crispr/cas9 complex for genomic editing
EP3436575A1 (en) 2015-06-18 2019-02-06 The Broad Institute Inc. Novel crispr enzymes and systems
US9926546B2 (en) 2015-08-28 2018-03-27 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
US9512446B1 (en) 2015-08-28 2016-12-06 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
AU2016316845B2 (en) 2015-08-28 2022-03-10 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
US9897378B2 (en) 2015-10-08 2018-02-20 Nyc Designed Inspirations Llc Cosmetic makeup sponge/blender container
IL294014B2 (en) 2015-10-23 2024-07-01 Harvard College Nucleobase editors and uses thereof
DK3368052T3 (da) * 2015-10-27 2022-03-14 Childrens Hospital Med Ct Anvendelse af mapk-hæmmere for at nedbringe tabet af hæmatopoietiske stamceller under ex vivo-dyrkning og -genmanipulation
GB2559526B (en) 2015-11-03 2021-02-17 Harvard College Method and apparatus for volumetric imaging of a three-dimensional nucleic acid containing matrix
CN115927199A (zh) * 2015-11-04 2023-04-07 菲特治疗公司 用于诱导造血细胞分化的方法和组合物
ES2942309T3 (es) 2015-11-04 2023-05-31 Vertex Pharma Materiales y métodos para el tratamiento de hemoglobinopatías
EP4249074A3 (en) 2015-11-04 2024-01-10 Fate Therapeutics, Inc. Genomic engineering of pluripotent cells
NZ783534A (en) * 2015-12-28 2025-09-26 Novartis Ag Compositions and methods for the treatment of hemoglobinopathies
AR107469A1 (es) 2016-01-27 2018-05-02 Oncorus Inc Vectores virales oncolíticos y sus usos
US11530253B2 (en) 2016-02-25 2022-12-20 The Children's Medical Center Corporation Customized class switch of immunoglobulin genes in lymphoma and hybridoma by CRISPR/CAS9 technology
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
EP3219799A1 (en) 2016-03-17 2017-09-20 IMBA-Institut für Molekulare Biotechnologie GmbH Conditional crispr sgrna expression
KR102351329B1 (ko) * 2016-04-18 2022-01-18 크리스퍼 테라퓨틱스 아게 혈색소병증의 치료를 위한 물질 및 방법
CN109415761B (zh) 2016-04-25 2022-09-20 哈佛学院董事及会员团体 用于原位分子检测的杂交链反应方法
WO2017191503A1 (en) * 2016-05-05 2017-11-09 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
US10767175B2 (en) 2016-06-08 2020-09-08 Agilent Technologies, Inc. High specificity genome editing using chemically modified guide RNAs
EP3487994A4 (en) * 2016-07-25 2020-01-29 Bluebird Bio, Inc. HOMING BCL11A ENDONUCLEASE VARIANTS, COMPOSITIONS AND METHODS OF USE
US11078481B1 (en) 2016-08-03 2021-08-03 KSQ Therapeutics, Inc. Methods for screening for cancer targets
WO2018027078A1 (en) 2016-08-03 2018-02-08 President And Fellows Of Harard College Adenosine nucleobase editors and uses thereof
US11661590B2 (en) 2016-08-09 2023-05-30 President And Fellows Of Harvard College Programmable CAS9-recombinase fusion proteins and uses thereof
CN106244555A (zh) * 2016-08-23 2016-12-21 广州医科大学附属第三医院 一种提高基因打靶的效率的方法及β‑球蛋白基因位点的碱基原位修复方法
US11542509B2 (en) 2016-08-24 2023-01-03 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
US11078483B1 (en) 2016-09-02 2021-08-03 KSQ Therapeutics, Inc. Methods for measuring and improving CRISPR reagent function
DK3510157T5 (da) 2016-09-08 2024-09-02 2Seventy Bio Inc Pd-1-homing-endonucleasevarianter, sammensætninger og fremgangsmåder til anvendelse
SG11201903089RA (en) 2016-10-14 2019-05-30 Harvard College Aav delivery of nucleobase editors
US10745677B2 (en) 2016-12-23 2020-08-18 President And Fellows Of Harvard College Editing of CCR5 receptor gene to protect against HIV infection
CA3050514A1 (en) 2017-01-27 2018-08-02 Children's Hospital Medical Center Methods of enhancing engraftment activity of hematopoietic stem cells
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
EP3592381A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Cancer vaccine
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
EP3596217A1 (en) 2017-03-14 2020-01-22 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
GB2575930A (en) 2017-03-23 2020-01-29 Harvard College Nucleobase editors comprising nucleic acid programmable DNA binding proteins
AU2018254547B2 (en) 2017-04-20 2024-06-13 Egenesis, Inc. Methods for generating genetically modified animals
AU2018254619B2 (en) 2017-04-21 2022-07-21 The General Hospital Corporation Variants of Cpf1 (CAS12a) with altered PAM specificity
US12110499B2 (en) 2017-04-24 2024-10-08 Seattle Children's Hospital 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
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
US20200140842A1 (en) 2017-05-25 2020-05-07 The General Hospital Corporation Bipartite base editor (bbe) architectures and type-ii-c-cas9 zinc finger editing
US11788087B2 (en) 2017-05-25 2023-10-17 The Children's Medical Center Corporation BCL11A guide delivery
WO2018220210A1 (en) * 2017-06-02 2018-12-06 Institut National De La Sante Et De La Recherche Medicale (Inserm) Recombinant lentiviral vector for stem cell-based gene therapy of sickle cell disorder
WO2019014564A1 (en) 2017-07-14 2019-01-17 Editas Medicine, Inc. SYSTEMS AND METHODS OF TARGETED INTEGRATION AND GENOME EDITING AND DETECTION THEREOF WITH INTEGRATED PRIMING SITES
CA3070299A1 (en) 2017-07-26 2019-01-31 Oncorus, Inc. Oncolytic viral vectors and uses thereof
US11732274B2 (en) 2017-07-28 2023-08-22 President And Fellows Of Harvard College Methods and compositions for evolving base editors using phage-assisted continuous evolution (PACE)
EP3676376B1 (en) 2017-08-30 2025-01-15 President and Fellows of Harvard College High efficiency base editors comprising gam
WO2019070974A1 (en) 2017-10-04 2019-04-11 Bluebird Bio, Inc. PCSK9 ENDONUCLEASE VARIANTS, COMPOSITIONS AND METHODS OF USE
AU2018347583A1 (en) 2017-10-13 2020-05-21 Selecta Biosciences, Inc. Methods and compositions for attenuating anti-viral transfer vector IgM responses
WO2019079347A1 (en) 2017-10-16 2019-04-25 The Broad Institute, Inc. USES OF BASIC EDITORS ADENOSINE
MA50849A (fr) * 2017-10-26 2020-09-02 Vertex Pharma Substances et procédés pour le traitement d'hémoglobinopathies
CN109722415B (zh) * 2017-10-27 2021-01-26 博雅辑因（北京）生物科技有限公司 一种造血干细胞的培养组合物、培养基以及造血干细胞的培养方法
EP3720473A1 (en) 2017-12-05 2020-10-14 Vertex Pharmaceuticals Incorporated Crispr-cas9 modified cd34+ human hematopoietic stem and progenitor cells and uses thereof
WO2019118949A1 (en) 2017-12-15 2019-06-20 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
WO2019150196A1 (en) 2018-02-05 2019-08-08 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
WO2019150203A1 (en) 2018-02-05 2019-08-08 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
CA3092372A1 (en) 2018-02-26 2019-08-29 AnTolRx, Inc. Tolerogenic liposomes and methods of use thereof
EP3765614A1 (en) 2018-03-14 2021-01-20 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
US10968257B2 (en) 2018-04-03 2021-04-06 The Broad Institute, Inc. Target recognition motifs and uses thereof
WO2019204226A1 (en) 2018-04-16 2019-10-24 University Of Massachusetts Compositions and methods for improved gene editing
WO2019213013A1 (en) 2018-05-02 2019-11-07 The Children's Medical Center Corporation Improved bcl11a micrornas for treating hemoglobinopathies
KR102852347B1 (ko) 2018-05-11 2025-09-01 빔 테라퓨틱스, 인크. 프로그래밍가능한 염기 편집기 시스템을 이용하여 병원성 아미노산을 치환하는 방법
WO2019226953A1 (en) 2018-05-23 2019-11-28 The Broad Institute, Inc. Base editors and uses thereof
BR112020025439A2 (pt) 2018-06-14 2021-03-16 Bluebird Bio, Inc. Receptores de antígenos quiméricos cd79a
KR20210058816A (ko) 2018-08-18 2021-05-24 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 원 위치에 유전자 편집
US12421507B2 (en) 2018-08-20 2025-09-23 The Broad Institute, Inc. Methods and compositions for optochemical control of CRISPR-CAS9
US20200102563A1 (en) * 2018-10-02 2020-04-02 Exosome Therapeutics, Inc. Exosome loaded therapeutics for treating sickle cell disease
US12312584B2 (en) 2018-10-02 2025-05-27 Exosome Therapeutics, Inc. cGMP exosome loaded therapeutics for treating sickle cell disease
WO2020076976A1 (en) 2018-10-10 2020-04-16 Readcoor, Inc. Three-dimensional spatial molecular indexing
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
EP3887521A1 (en) * 2018-11-29 2021-10-06 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
CA3122278A1 (en) 2018-12-10 2020-06-18 Bluebird Bio, Inc. Homing endonuclease variants
US12264323B2 (en) 2018-12-17 2025-04-01 The Broad Institute, Inc. CRISPR CPF1 direct repeat variants
CA3124110A1 (en) 2018-12-17 2020-06-25 The Broad Institute, Inc. Crispr-associated transposase systems and methods of use thereof
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
US11946040B2 (en) 2019-02-04 2024-04-02 The General Hospital Corporation Adenine DNA base editor variants with reduced off-target RNA editing
CA3128755C (en) * 2019-02-13 2024-06-04 Beam Therapeutics Inc. Compositions and methods for treating hemoglobinopathies
DE112020001342T5 (de) 2019-03-19 2022-01-13 President and Fellows of Harvard College Verfahren und Zusammensetzungen zum Editing von Nukleotidsequenzen
WO2020214842A1 (en) 2019-04-17 2020-10-22 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
CN110042124A (zh) * 2019-04-25 2019-07-23 国家卫生健康委科学技术研究所 基因组碱基编辑增加人红细胞中胎儿血红蛋白水平的试剂盒及应用
CN111939271A (zh) * 2019-04-30 2020-11-17 博雅辑因（北京）生物科技有限公司 一种血红蛋白病治疗有效性预测方法
EP3964579A4 (en) * 2019-04-30 2023-07-26 EdiGene (GuangZhou) Inc. Method for predicting effectiveness of treatment of hemoglobinopathy
CN114206396A (zh) 2019-05-28 2022-03-18 西莱克塔生物科技公司 用于减弱抗病毒转移载体免疫应答的方法和组合物
EP3990650A4 (en) * 2019-06-28 2024-03-20 ASC Therapeutics Inc. METHODS AND COMPOSITIONS FOR TREATING THALASSEMIA AND SICKLE CELL ANEMIA
CN115103910A (zh) 2019-10-03 2022-09-23 工匠开发实验室公司 具有工程化的双引导核酸的crispr系统
WO2021072328A1 (en) 2019-10-10 2021-04-15 The Broad Institute, Inc. Methods and compositions for prime editing rna
CN115175991A (zh) 2019-10-16 2022-10-11 博德研究所 工程化肌肉靶向组合物
CA3160172A1 (en) * 2019-11-15 2021-05-20 The Board Of Trustees Of The Leland Stanford Junior University Targeted integration at alpha-globin locus in human hematopoietic stem and progenitor cells
CN112979823B (zh) * 2019-12-18 2022-04-08 华东师范大学 一种用于治疗和/或预防β血红蛋白病的产品及融合蛋白
US12091681B2 (en) 2020-03-27 2024-09-17 Mendus B.V. Ex vivo use of modified cells of leukemic origin for enhancing the efficacy of adoptive cell therapy
GB2614813B (en) 2020-05-08 2025-05-07 Harvard College Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence
JP2023526007A (ja) * 2020-05-13 2023-06-20 アンスティチュ ナショナル ドゥ ラ サンテ エ ドゥ ラ ルシェルシュ メディカル β－ヘモグロビン異常症の処置のための塩基編集アプローチ
JP2023531384A (ja) * 2020-06-04 2023-07-24 エメンドバイオ・インコーポレイテッド 新規なｏｍｎｉ－５９、６１、６７、７６、７９、８０、８１及び８２クリスパーヌクレアーゼ
EP4171617A1 (en) 2020-06-30 2023-05-03 Mendus B.V. Use of leukemia-derived cells in ovarian cancer vaccines
US20220049303A1 (en) 2020-08-17 2022-02-17 Readcoor, Llc Methods and systems for spatial mapping of genetic variants
KR20230058482A (ko) * 2020-09-04 2023-05-03 고쿠리츠다이가쿠호진 히로시마다이가쿠 표적 dna의 편집 방법, 표적 dna가 편집된 세포의 제조 방법, 및 그것들에 사용하는 dna 편집 시스템
JP2024503249A (ja) * 2020-12-21 2024-01-25 ２セブンティ バイオ インコーポレイテッド 部位特異的変異導入のための組成物及び方法
WO2022182801A1 (en) 2021-02-25 2022-09-01 Artisan Development Labs, Inc. Compositions and methods for targeting, editing, or modifying genes
US20220305100A1 (en) 2021-03-12 2022-09-29 Dcprime B.V. Methods of vaccination and use of cd47 blockade
WO2022256448A2 (en) 2021-06-01 2022-12-08 Artisan Development Labs, Inc. Compositions and methods for targeting, editing, or modifying genes
CN113481184A (zh) * 2021-08-06 2021-10-08 北京大学 融合蛋白以及其使用方法
KR20240055811A (ko) 2021-09-10 2024-04-29 애질런트 테크놀로지스, 인크. 프라임 편집을 위한 화학적 변형을 갖는 가이드 rna
WO2023064367A1 (en) 2021-10-12 2023-04-20 Selecta Biosciences, Inc. Methods and compositions for attenuating anti-viral transfer vector igm responses
US20230279442A1 (en) 2021-12-15 2023-09-07 Versitech Limited Engineered cas9-nucleases and method of use thereof
EP4486881A1 (en) 2022-03-01 2025-01-08 Celyntra Therapeutics SA Composition and methods for transgene insertion
US20230357437A1 (en) 2022-03-09 2023-11-09 Selecta Biosciences, Inc. Immunosuppressants in combination with anti-igm agents and related dosing
EP4273242A1 (en) * 2022-05-03 2023-11-08 ETH Zurich Crispr-based modification of human hbd gene
CN117427169A (zh) * 2023-10-24 2024-01-23 中国医学科学院基础医学研究所 同时破坏HBB竞争和HBG表观沉默在治疗β-血红蛋白病中的应用

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20020182673A1 (en) * 1998-05-15 2002-12-05 Genentech, Inc. IL-17 homologous polypedies and therapeutic uses thereof
JP2006518372A (ja) 2003-01-28 2006-08-10 セレクティス 脊椎動物の体組織においてエクスビボかつイントトで相同的組換えを誘発するためのメガヌクレアーゼの使用およびその応用
WO2007049095A1 (en) 2005-10-25 2007-05-03 Cellectis Laglidadg homing endonuclease variants having mutations in two functional subdomains and use thereof
WO2008010009A1 (en) 2006-07-18 2008-01-24 Cellectis Meganuclease variants cleaving a dna target sequence from a rag gene and uses thereof
AU2007347328B2 (en) 2007-02-19 2013-03-07 Cellectis LAGLIDADG homing endonuclease variants having novel substrate specificity and use thereof
WO2009013559A1 (en) * 2007-07-23 2009-01-29 Cellectis Meganuclease variants cleaving a dna target sequence from the human hemoglobin beta gene and uses thereof
US8383604B2 (en) * 2008-09-15 2013-02-26 Children's Medical Center Corporation Modulation of BCL11A for treatment of hemoglobinopathies
US20140148361A1 (en) 2010-06-07 2014-05-29 Barry L. Stoddard Generation and Expression of Engineered I-ONUI Endonuclease and Its Homologues and Uses Thereof
US8673557B2 (en) * 2011-02-28 2014-03-18 Seattle Children's Research Institute Coupling endonucleases with end-processing enzymes drives high efficiency gene disruption
RU2018109507A (ru) 2012-02-24 2019-02-28 Фред Хатчинсон Кэнсер Рисерч Сентер Композиции и способы лечения гемоглобинопатии
RS60838B1 (sr) * 2012-08-29 2020-10-30 Sangamo Therapeutics Inc Postupci i kompozicije za tretman genskog stanja

Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN112469823A (zh) * 2018-04-27 2021-03-09 西雅图儿童医院d/b/a西雅图儿童研究所 布鲁顿氏酪氨酸激酶的基于talen和基于crispr/cas的基因编辑
CN114457119A (zh) * 2022-04-11 2022-05-10 中吉智药(南京)生物技术有限公司 慢病毒载体在制备治疗β-地中海贫血药物中的应用
CN114457119B (zh) * 2022-04-11 2022-08-12 中吉智药(南京)生物技术有限公司 慢病毒载体在制备治疗β-地中海贫血药物中的应用

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