AU2008346801A1 - RNA interference for the treatment of heart failure - Google Patents

RNA interference for the treatment of heart failure Download PDF

Info

Publication number
AU2008346801A1
AU2008346801A1 AU2008346801A AU2008346801A AU2008346801A1 AU 2008346801 A1 AU2008346801 A1 AU 2008346801A1 AU 2008346801 A AU2008346801 A AU 2008346801A AU 2008346801 A AU2008346801 A AU 2008346801A AU 2008346801 A1 AU2008346801 A1 AU 2008346801A1
Authority
AU
Australia
Prior art keywords
rnai
vector
aav
expression
plb
Prior art date
Legal status (The legal status 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 status listed.)
Abandoned
Application number
AU2008346801A
Other languages
English (en)
Inventor
Henry Fechner
Roger J. Hajjar
Wolfgang Ch. Poller
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Nanocor Therapeutics Inc
Original Assignee
Nanocor 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 Nanocor Therapeutics Inc filed Critical Nanocor Therapeutics Inc
Publication of AU2008346801A1 publication Critical patent/AU2008346801A1/en
Abandoned legal-status Critical Current

Links

Classifications

    • 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
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • 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
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • 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
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/04—Inotropic agents, i.e. stimulants of cardiac contraction; Drugs for heart failure
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/06—Antiarrhythmics
    • 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
    • C12N15/86—Viral vectors
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Biochemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Plant Pathology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Cardiology (AREA)
  • Physics & Mathematics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Virology (AREA)
  • Hospice & Palliative Care (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
AU2008346801A 2007-12-31 2008-12-23 RNA interference for the treatment of heart failure Abandoned AU2008346801A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US1832607P 2007-12-31 2007-12-31
US61/018,326 2007-12-31
PCT/US2008/088248 WO2009088786A1 (en) 2007-12-31 2008-12-23 Rna interference for the treatment of heart failure

Publications (1)

Publication Number Publication Date
AU2008346801A1 true AU2008346801A1 (en) 2009-07-16

Family

ID=40853375

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2008346801A Abandoned AU2008346801A1 (en) 2007-12-31 2008-12-23 RNA interference for the treatment of heart failure

Country Status (8)

Country Link
US (2) US8404658B2 (enExample)
EP (1) EP2225002A4 (enExample)
JP (1) JP2011512326A (enExample)
CN (1) CN101970051A (enExample)
AU (1) AU2008346801A1 (enExample)
CA (1) CA2711179A1 (enExample)
NZ (1) NZ587060A (enExample)
WO (1) WO2009088786A1 (enExample)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN108192922A (zh) * 2018-01-03 2018-06-22 李江 受磷蛋白基因反义rna重组aav载体、病毒、构建方法及应用

Families Citing this family (173)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3075867B1 (en) * 2008-09-03 2019-03-13 Quantumdx Group Limited Sensing strategies and methods for nucleic acid detection using biosensors
EP2652151A2 (en) 2010-12-15 2013-10-23 Miragen Therapeutics Microrna inhibitors comprising locked nucleotides
CN107320734A (zh) 2011-04-29 2017-11-07 西莱克塔生物科技公司 从合成纳米载体中控制释放免疫抑制剂
EP2763703B1 (en) 2011-10-06 2018-02-14 Miragen Therapeutics, Inc. Control of whole body energy homeostasis by microrna regulation
JP6562632B2 (ja) * 2012-02-14 2019-08-21 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 心血管疾患および他の状態のためのパラクリン遺伝子の全身送達および制御された発現
EP2864482A4 (en) 2012-06-21 2016-02-10 Miragen Therapeutics Inc OLIGONUCLEOTIDE INHIBITORS COMPRISING A BLOCKED NUCLEIC ACID PATTERN
CN105209621B (zh) 2012-12-12 2021-05-25 布罗德研究所有限公司 对用于序列操纵的改进的系统、方法和酶组合物进行的工程化和优化
EP3064585B1 (en) 2012-12-12 2020-02-05 The Broad Institute, Inc. Engineering and optimization of improved systems, methods and enzyme compositions for sequence manipulation
EP2931899A1 (en) 2012-12-12 2015-10-21 The Broad Institute, Inc. Functional genomics using crispr-cas systems, compositions, methods, knock out libraries and applications thereof
SG10201707569YA (en) 2012-12-12 2017-10-30 Broad Inst Inc Delivery, Engineering and Optimization of Systems, Methods and Compositions for Sequence Manipulation and Therapeutic Applications
CN105307641A (zh) 2013-05-03 2016-02-03 西莱克塔生物科技公司 用于降低不期望体液免疫应答的给药组合
JP6738728B2 (ja) 2013-06-17 2020-08-19 ザ・ブロード・インスティテュート・インコーポレイテッド 肝臓ターゲティングおよび治療のためのＣＲＩＳＰＲ−Ｃａｓ系、ベクターおよび組成物の送達および使用
ES2777217T3 (es) 2013-06-17 2020-08-04 Broad Inst Inc Suministro, modificación y optimización de sistemas de guía en tándem, métodos y composiciones para la manipulación de secuencias
SG11201510327TA (en) 2013-06-17 2016-01-28 Broad Inst Inc Delivery, engineering and optimization of systems, methods and compositions for targeting and modeling diseases and disorders of post mitotic cells
WO2014204725A1 (en) 2013-06-17 2014-12-24 The Broad Institute Inc. Optimized crispr-cas double nickase systems, methods and compositions for sequence manipulation
KR20250012194A (ko) 2013-06-17 2025-01-23 더 브로드 인스티튜트, 인코퍼레이티드 바이러스 구성성분을 사용하여 장애 및 질환을 표적화하기 위한 crispr-cas 시스템 및 조성물의 전달, 용도 및 치료 적용
WO2015077717A1 (en) 2013-11-25 2015-05-28 The Broad Institute Inc. Compositions and methods for diagnosing, evaluating and treating cancer by means of the dna methylation status
WO2015085147A1 (en) 2013-12-05 2015-06-11 The Broad Institute Inc. Polymorphic gene typing and somatic change detection using sequencing data
JP6793547B2 (ja) 2013-12-12 2020-12-02 ザ・ブロード・インスティテュート・インコーポレイテッド 最適化機能ＣＲＩＳＰＲ−Ｃａｓ系による配列操作のための系、方法および組成物
KR20160097338A (ko) 2013-12-12 2016-08-17 더 브로드 인스티튜트, 인코퍼레이티드 뉴클레오티드 반복 장애에서의 ｃｒｉｓｐｒ-ｃａｓ 시스템의 조성물 및 방법 및 용도
WO2015089473A1 (en) 2013-12-12 2015-06-18 The Broad Institute Inc. Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation
CN111206032B (zh) 2013-12-12 2024-07-19 布罗德研究所有限公司 用于基因组编辑的crispr-cas系统和组合物的递送、用途和治疗应用
CN105899658B (zh) 2013-12-12 2020-02-18 布罗德研究所有限公司 针对hbv和病毒性疾病以及障碍的crispr-cas系统和组合物的递送、用途和治疗应用
WO2015089419A2 (en) 2013-12-12 2015-06-18 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
JP7060324B2 (ja) 2013-12-20 2022-04-26 ザ・ブロード・インスティテュート・インコーポレイテッド ネオ抗原ワクチンによる併用療法
EP3180426B1 (en) 2014-08-17 2019-12-25 The Broad Institute, Inc. Genome editing using cas9 nickases
EA201790551A1 (ru) 2014-09-07 2017-07-31 Селекта Байосайенсиз, Инк. Способы и композиции для ослабления иммунных ответов к пропускающему экзон вирусному трансферному вектору
WO2016049024A2 (en) 2014-09-24 2016-03-31 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for modeling competition of multiple cancer mutations in vivo
WO2016049163A2 (en) 2014-09-24 2016-03-31 The Broad Institute Inc. Use and production of chd8+/- transgenic animals with behavioral phenotypes characteristic of autism spectrum disorder
WO2016049251A1 (en) 2014-09-24 2016-03-31 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for modeling mutations in leukocytes
WO2016049258A2 (en) 2014-09-25 2016-03-31 The Broad Institute Inc. Functional screening with optimized functional crispr-cas systems
US20170247762A1 (en) 2014-10-27 2017-08-31 The Board Institute Inc. Compositions, methods and use of synthetic lethal screening
EP3985115A1 (en) 2014-12-12 2022-04-20 The Broad Institute, Inc. Protected guide rnas (pgrnas)
EP3230452B1 (en) 2014-12-12 2025-06-11 The Broad Institute, Inc. Dead guides for crispr transcription factors
WO2016094874A1 (en) 2014-12-12 2016-06-16 The Broad Institute Inc. Escorted and functionalized guides for crispr-cas systems
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)
US10975442B2 (en) 2014-12-19 2021-04-13 Massachusetts Institute Of Technology Molecular biomarkers for cancer immunotherapy
EP3234192B1 (en) 2014-12-19 2021-07-14 The Broad Institute, Inc. Unbiased identification of double-strand breaks and genomic rearrangement by genome-wide insert capture sequencing
EP3234130B1 (en) 2014-12-19 2020-11-25 The Broad Institute, Inc. Methods for profiling the t-cell- receptor repertoire
WO2016106236A1 (en) 2014-12-23 2016-06-30 The Broad Institute Inc. Rna-targeting system
WO2016106244A1 (en) 2014-12-24 2016-06-30 The Broad Institute Inc. Crispr having or associated with destabilization domains
HK1249108A1 (zh) 2015-01-20 2018-10-26 米拉根医疗股份有限公司 Mir-92抑制剂及其用途
US20180271891A1 (en) 2015-03-11 2018-09-27 The Broad Institute Inc. Selective treatment of prmt5 dependent cancer
JP6913032B2 (ja) 2015-05-20 2021-08-04 ザ・ブロード・インスティテュート・インコーポレイテッド 共通ネオ抗原
TW202241500A (zh) 2015-06-09 2022-11-01 美商博德研究所有限公司 用於贅瘤疫苗之調配物及其製備方法
US20180355033A1 (en) 2015-06-10 2018-12-13 Dana-Farber Cancer Institute, Inc. Antibodies, compounds and screens for identifying and treating cachexia or pre-cachexia
EP3929287A3 (en) 2015-06-18 2022-04-13 The Broad Institute, Inc. Crispr enzyme mutations reducing off-target effects
WO2016205745A2 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Cell sorting
CA3012631A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Novel crispr enzymes and systems
AU2016279062A1 (en) 2015-06-18 2019-03-28 Omar O. Abudayyeh Novel CRISPR enzymes and systems
US9790490B2 (en) 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
AU2016308339A1 (en) 2015-08-18 2018-04-12 Baylor College Of Medicine Methods and compositions for altering function and structure of chromatin loops and/or domains
US12241053B2 (en) 2015-10-09 2025-03-04 The Brigham And Women's Hospital, Inc. Modulation of novel immune checkpoint targets
CN116814590A (zh) 2015-10-22 2023-09-29 布罗德研究所有限公司 Vi-b型crispr酶和系统
US11492670B2 (en) 2015-10-27 2022-11-08 The Broad Institute Inc. Compositions and methods for targeting cancer-specific sequence variations
WO2017075465A1 (en) 2015-10-28 2017-05-04 The Broad Institute Inc. Compositions and methods for evaluating and modulating immune responses by detecting and targeting gata3
WO2017075478A2 (en) 2015-10-28 2017-05-04 The Broad Institute Inc. Compositions and methods for evaluating and modulating immune responses by use of immune cell gene signatures
WO2017075451A1 (en) 2015-10-28 2017-05-04 The Broad Institute Inc. Compositions and methods for evaluating and modulating immune responses by detecting and targeting pou2af1
JP6978774B2 (ja) * 2015-11-06 2021-12-08 国立大学法人 熊本大学 心不全の予防又は治療のための医薬組成物
EP3992283A1 (en) 2015-12-11 2022-05-04 The Trustees Of The University Of Pennsylvania Scalable purification method for aavrh10
EP3387138B2 (en) 2015-12-11 2026-05-27 The Trustees Of The University Of Pennsylvania Scalable purification method for aav9
WO2017100674A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Scalable purification method for aav1
US11015174B2 (en) 2015-12-11 2021-05-25 The Trustees Of The University Of Pennsylvania Scalable purification method for AAV8
WO2017106657A1 (en) 2015-12-18 2017-06-22 The Broad Institute Inc. Novel crispr enzymes and systems
AU2017254477A1 (en) 2016-04-18 2018-11-01 Jennifer G. ABELIN Improved HLA epitope prediction
EP3445853A1 (en) 2016-04-19 2019-02-27 The Broad Institute, Inc. Cpf1 complexes with reduced indel activity
KR20260004568A (ko) 2016-04-19 2026-01-08 더 브로드 인스티튜트, 인코퍼레이티드 신규한 crispr 효소 및 시스템
CA3026110A1 (en) 2016-04-19 2017-11-02 The Broad Institute, Inc. Novel crispr enzymes and systems
AU2017283713B2 (en) 2016-06-17 2021-04-08 Massachusetts Institute Of Technology Type VI CRISPR orthologs and systems
WO2018005873A1 (en) 2016-06-29 2018-01-04 The Broad Institute Inc. Crispr-cas systems having destabilization domain
WO2018035387A1 (en) 2016-08-17 2018-02-22 The Broad Institute, Inc. Novel crispr enzymes and systems
US11352647B2 (en) 2016-08-17 2022-06-07 The Broad Institute, Inc. Crispr enzymes and systems
US20190262399A1 (en) 2016-09-07 2019-08-29 The Broad Institute, Inc. Compositions and methods for evaluating and modulating immune responses
US12499971B2 (en) 2016-09-28 2025-12-16 The Broad Institute, Inc. Systematic screening and mapping of regulatory elements in non-coding genomic regions, methods, compositions, and applications thereof
US12447213B2 (en) 2016-10-07 2025-10-21 The Broad Institute, Inc. Modulation of novel immune checkpoint targets
EP3574116A1 (en) 2017-01-24 2019-12-04 The Broad Institute, Inc. Compositions and methods for detecting a mutant variant of a polynucleotide
EP4599848A3 (en) 2017-03-11 2025-12-03 Cartesian Therapeutics, Inc. Methods and compositions related to combined treatment with anti-inflammatories and synthetic nanocarriers comprising an immunosuppressant
CN110959039B (zh) 2017-03-15 2025-03-14 博德研究所 新型cas13b直向同源物crispr酶和系统
AU2018251801B2 (en) 2017-04-12 2024-11-07 Massachusetts Institute Of Technology Novel type VI CRISPR orthologs and systems
WO2018191750A2 (en) 2017-04-14 2018-10-18 The Broad Institute Inc. Novel delivery of large payloads
US11591601B2 (en) 2017-05-05 2023-02-28 The Broad Institute, Inc. Methods for identification and modification of lncRNA associated with target genotypes and phenotypes
WO2018208910A1 (en) 2017-05-09 2018-11-15 The Broad Institute Inc. Gut microbiome function predicts response to anti-integrin biologic therapy in inflammatory bowel diseases
EP3625342B1 (en) 2017-05-18 2022-08-24 The Broad Institute, Inc. Systems, methods, and compositions for targeted nucleic acid editing
CN111328290A (zh) 2017-06-26 2020-06-23 博德研究所 用于靶向核酸编辑的基于crispr/cas-腺嘌呤脱氨酶的组合物、系统和方法
KR102338449B1 (ko) 2017-09-21 2021-12-10 더 브로드 인스티튜트, 인코퍼레이티드 표적화된 핵산 편집을 위한 시스템, 방법, 및 조성물
WO2019071054A1 (en) 2017-10-04 2019-04-11 The Broad Institute, Inc. METHODS AND COMPOSITIONS FOR MODIFYING THE FUNCTION AND STRUCTURE OF BUCKLES AND / OR CHROMATIN DOMAINS
US12227742B2 (en) 2017-10-23 2025-02-18 The Broad Institute, Inc. Nucleic acid modifiers
US11547614B2 (en) 2017-10-31 2023-01-10 The Broad Institute, Inc. Methods and compositions for studying cell evolution
WO2019094983A1 (en) 2017-11-13 2019-05-16 The Broad Institute, Inc. Methods and compositions for treating cancer by targeting the clec2d-klrb1 pathway
WO2019094984A1 (en) 2017-11-13 2019-05-16 The Broad Institute, Inc. Methods for determining spatial and temporal gene expression dynamics during adult neurogenesis in single cells
US20230193242A1 (en) 2017-12-22 2023-06-22 The Broad Institute, Inc. Cas12b systems, methods, and compositions for targeted dna base editing
US10968257B2 (en) 2018-04-03 2021-04-06 The Broad Institute, Inc. Target recognition motifs and uses thereof
KR102853088B1 (ko) 2018-05-11 2025-09-02 빔 테라퓨틱스, 인크. 프로그래밍가능한 염기 편집기 시스템을 이용하여 단일염기다형성을 편집하는 방법
EP3830256A2 (en) 2018-07-31 2021-06-09 The Broad Institute, Inc. Novel crispr enzymes and systems
KR102894715B1 (ko) 2018-08-03 2025-12-03 빔 테라퓨틱스, 인크. 핵산 표적 서열을 변형시키기 위한 다중-이펙터 핵염기 편집기 및 이를 이용하는 방법
CA3106035A1 (en) 2018-08-07 2020-02-13 The Broad Institute, Inc. Cas12b enzymes and systems
WO2020041380A1 (en) 2018-08-20 2020-02-27 The Broad Institute, Inc. Methods and compositions for optochemical control of crispr-cas9
US20210382068A1 (en) 2018-10-02 2021-12-09 Dana-Farber Cancer Institute, Inc. Hla single allele lines
WO2020131586A2 (en) 2018-12-17 2020-06-25 The Broad Institute, Inc. Methods for identifying neoantigens
WO2020131862A1 (en) 2018-12-17 2020-06-25 The Broad Institute, Inc. Crispr-associated transposase systems and methods of use thereof
WO2020127990A1 (en) 2018-12-20 2020-06-25 Biomedrex Ab System for production of crispr-based pharmaceutical compositions
US20220145305A1 (en) 2019-02-12 2022-05-12 Universita' Degli Studi Di Trento CAS12a GUIDE RNA MOLECULES AND USES THEREOF
US20220119787A1 (en) 2019-02-20 2022-04-21 Biomedrex Ab Virus therapy
US12215382B2 (en) 2019-03-01 2025-02-04 The General Hospital Corporation Liver protective MARC variants and uses thereof
US12419915B2 (en) 2019-03-13 2025-09-23 The Broad Institute, Inc. Microglial progenitors for regeneration of functional microglia in the central nervous system and therapeutics uses thereof
JP2022525193A (ja) * 2019-03-15 2022-05-11 ユニヴァーシティ オブ ワシントン Ｐｒｐｆ３１遺伝子発現ノックダウンによる、インビトロで分化したヒト細胞の生存の改善
US12534714B2 (en) 2019-03-18 2026-01-27 The Broad Institute, Inc. Type VII CRISPR proteins and systems
WO2020232271A1 (en) 2019-05-14 2020-11-19 The Broad Institute, Inc. Compositions and methods for targeting multinucleated cells
US20220235340A1 (en) 2019-05-20 2022-07-28 The Broad Institute, Inc. Novel crispr-cas systems and uses thereof
US12553041B2 (en) 2019-06-04 2026-02-17 Cartesian Therapeutics Inc. Formulations and doses of pegylated uricase
CA3147875A1 (en) 2019-07-19 2021-01-28 Flagship Pioneering Innovations Vi, Llc Recombinase compositions and methods of use
US20220298501A1 (en) 2019-08-30 2022-09-22 The Broad Institute, Inc. Crispr-associated mu transposase systems
CA3153563A1 (en) 2019-09-09 2021-03-18 Beam Therapeutics Inc. Novel crispr enzymes, methods, systems and uses thereof
US20220325296A1 (en) 2019-09-12 2022-10-13 The Broad Institute, Inc. Engineered adeno-associated virus capsids
US12297426B2 (en) 2019-10-01 2025-05-13 The Broad Institute, Inc. DNA damage response signature guided rational design of CRISPR-based systems and therapies
US12394502B2 (en) 2019-10-02 2025-08-19 The General Hospital Corporation Method for predicting HLA-binding peptides using protein structural features
JP2022551986A (ja) 2019-10-16 2022-12-14 ザ・ブロード・インスティテュート・インコーポレイテッド 修飾筋肉標的化組成物
CA3162908A1 (en) 2019-12-03 2021-06-10 Beam Therapeutics Inc. Synthetic guide rna, compositions, methods, and uses thereof
WO2021152181A1 (en) 2020-01-30 2021-08-05 Biomedrex Ab Virus therapy
BR112022017715A2 (pt) 2020-03-04 2022-11-16 Flagship Pioneering Innovations Vi Llc Métodos e composições para modular um genoma
EP4149618A4 (en) 2020-05-12 2024-07-03 Duke University COMPOSITIONS AND METHODS FOR CONTROLLING BLOOD PRESSURE
JP7854946B2 (ja) 2020-05-26 2026-05-07 ディオニス セラピューティクス インコーポレイテッド 核酸人工ミニプロテオームライブラリー
US12668814B2 (en) 2020-07-22 2026-06-30 The Broad Institute, Inc. Engineered muscle targeting compositions
US12419948B2 (en) 2020-08-31 2025-09-23 The Broad Institute, Inc. Immunogenic compositions and use thereof
KR20230158531A (ko) 2021-03-23 2023-11-20 빔 테라퓨틱스, 인크. 신규 crispr 효소, 방법, 시스템 및 그 용도
EP4323501A1 (en) 2021-04-16 2024-02-21 Beam Therapeutics Inc. Genetic modification of hepatocytes
AU2022285744A1 (en) 2021-06-02 2023-12-14 Beam Therapeutics Inc. Circular guide rnas for crispr/cas editing systems
US20240325568A1 (en) 2021-07-20 2024-10-03 The Broad Institute, Inc. Engineered targeting compositions for endothelial cells of the central nervous system vasculature and methods of use thereof
KR20240037299A (ko) 2021-07-23 2024-03-21 빔 테라퓨틱스, 인크. Crispr/cas 편집 시스템용 가이드 rna
KR20260037664A (ko) 2021-09-08 2026-03-18 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 게놈을 조절하기 위한 방법 및 조성물
EP4426828A1 (en) 2021-11-01 2024-09-11 Tome Biosciences, Inc. Single construct platform for simultaneous delivery of gene editing machinery and nucleic acid cargo
WO2023076733A1 (en) 2021-11-01 2023-05-04 Dana-Farber Cancer Institute, Inc. Biologically selected nucleic acid artificial mini-proteome libraries
WO2023091988A1 (en) 2021-11-18 2023-05-25 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Expression of the spike s glycoprotein of sars-cov-2 from avian paramyxovirus type 3 (apmv3)
EP4437092A1 (en) 2021-11-26 2024-10-02 Epigenic Therapeutics Inc. Method of modulating pcsk9 and uses thereof
AU2022413670A1 (en) 2021-12-17 2024-07-04 Beam Therapeutics Inc. Crispr enzymes, methzods, systems and uses thereof
US20250057980A1 (en) 2021-12-22 2025-02-20 Tome Biosciences, Inc. Co-Delivery of a Gene Editor Construct and a Donor Template
WO2023196772A1 (en) 2022-04-04 2023-10-12 Beam Therapeutics Inc. Novel rna base editing compositions, systems, methods and uses thereof
WO2023205744A1 (en) 2022-04-20 2023-10-26 Tome Biosciences, Inc. Programmable gene insertion compositions
WO2023215831A1 (en) 2022-05-04 2023-11-09 Tome Biosciences, Inc. Guide rna compositions for programmable gene insertion
EP4526324A1 (en) 2022-05-18 2025-03-26 The Broad Institute, Inc. Engineered viral capsids with increased stability and methods of use thereof
WO2023225670A2 (en) 2022-05-20 2023-11-23 Tome Biosciences, Inc. Ex vivo programmable gene insertion
WO2024015892A1 (en) 2022-07-13 2024-01-18 The Broad Institute, Inc. Hla-ii immunopeptidome methods and systems for antigen discovery
EP4554961A2 (en) 2022-07-14 2025-05-21 The Broad Institute, Inc. Aav capsids that enable cns-wide gene delivery through interactions with the transferrin receptor
WO2024020587A2 (en) 2022-07-22 2024-01-25 Tome Biosciences, Inc. Pleiopluripotent stem cell programmable gene insertion
EP4587560A1 (en) 2022-09-16 2025-07-23 Beam Therapeutics Inc. Large serine recombinases, systems and uses thereof
WO2024138194A1 (en) 2022-12-22 2024-06-27 Tome Biosciences, Inc. Platforms, compositions, and methods for in vivo programmable gene insertion
WO2024163842A2 (en) 2023-02-03 2024-08-08 The Broad Institute, Inc. Delivering genes to the brain endothelium to treat lysosomal storage disorder-derived neuropathology
WO2024168253A1 (en) 2023-02-10 2024-08-15 Possible Medicines Llc Delivery of an rna guided recombination system
WO2024168265A1 (en) 2023-02-10 2024-08-15 Possible Medicines Llc Aav delivery of rna guided recombination system
CN116286820B (zh) * 2023-02-17 2024-11-12 华中科技大学同济医学院附属同济医院 一种shRNA、其重组表达载体、转化体、抗心衰药物及制备方法和制药用途
KR20260021596A (ko) 2023-04-05 2026-02-13 아이오니스 파마수티컬즈, 인코포레이티드 Pln 발현을 감소시키는 화합물 및 방법
WO2024234006A1 (en) 2023-05-11 2024-11-14 Tome Biosciences, Inc. Systems, compositions, and methods for targeting liver sinusodial endothelial cells (lsecs)
US12531162B1 (en) * 2023-05-31 2026-01-20 Northeastern University Multi-dimensional phenotypic space for genotype to phenotype mapping and intelligent design of cancer drug therapies using a deep learning net
EP4750890A2 (en) 2023-07-27 2026-06-03 Life Edit Therapeutics, Inc. Rna-guided nucleases and active fragments and variants thereof and methods of use
WO2025050069A1 (en) 2023-09-01 2025-03-06 Tome Biosciences, Inc. Programmable gene insertion using engineered integration enzymes
WO2025059533A1 (en) 2023-09-13 2025-03-20 The Broad Institute, Inc. Crispr enzymes and systems
CN122422501A (zh) 2023-10-18 2026-07-17 生命编辑治疗股份有限公司 Rna引导的核酸酶及其活性片段和变体以及使用方法
WO2025097055A2 (en) 2023-11-02 2025-05-08 The Broad Institute, Inc. Compositions and methods of use of t cells in immunotherapy
US12562256B2 (en) * 2023-11-07 2026-02-24 New York University Systems, methods and computer-accessible medium for identifying target pairs for CAR-T therapy
WO2025106685A1 (en) 2023-11-15 2025-05-22 Slate Bio, Inc. Interleukin-2 superkine and interleukin-33, compositions and uses thereof
AU2024381770A1 (en) 2023-11-15 2026-05-28 Slate Bio, Inc. Bifunctional il-2 and il-33 proteins for treating autoimmune disease
WO2025106663A1 (en) 2023-11-15 2025-05-22 Slate Bio, Inc. Interleukin-33 variants, compositions and uses thereof
WO2025117544A1 (en) 2023-11-29 2025-06-05 The Broad Institute, Inc. Engineered omega guide molecule and iscb compositions, systems, and methods of use thereof
WO2025155923A1 (en) 2024-01-17 2025-07-24 The Broad Institute, Inc. Aav capsid modifications that enable improved cns-wide gene delivery through interactions with the transferrin receptor
WO2025160155A1 (en) 2024-01-22 2025-07-31 The Broad Institute, Inc. Epigenetic targeting of prion diseases
WO2025174908A1 (en) 2024-02-12 2025-08-21 Life Edit Therapeutics, Inc. Novel rna-guided nucleases and proteins for polymerase editing
WO2025188687A1 (en) 2024-03-04 2025-09-12 Beam Therapeutics Inc. Stable rna compositions with stem loop, and methods thereof
WO2025217163A2 (en) 2024-04-08 2025-10-16 The Broad Institute, Inc. Novel aav capsids binding to human cd59
WO2025217174A1 (en) 2024-04-08 2025-10-16 The Broad Institute, Inc. Aav capsid modifications that enable improved cns-wide gene delivery through interactions with carbonic anhydrase iv
WO2025224107A1 (en) 2024-04-22 2025-10-30 Basecamp Research Ltd Method and compositions for detecting off-target editing
TW202617985A (zh) 2024-06-25 2026-05-01 美商生命編輯治療學公司 新穎反轉錄酶及其用途
CN119242629B (zh) * 2024-08-01 2025-08-01 北京尧景基因技术有限公司 抑制PLN基因表达的siRNA及其缀合物和应用
WO2026107393A2 (en) 2024-11-14 2026-05-21 The Broad Institute, Inc. Aav capsid modifications that reduce immunogenicity

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1463805B1 (en) * 2001-12-17 2014-10-22 The Trustees of The University of Pennsylvania Adeno-associated virus (aav) serotype 9 sequences, vectors containing same, and uses therefor
CN1498964A (zh) * 2002-11-07 2004-05-26 本元正阳基因技术股份有限公司 可诱导RNAi途径的用于基因治疗的系列重组腺相关病毒
US20050019927A1 (en) * 2003-07-13 2005-01-27 Markus Hildinger DECREASING GENE EXPRESSION IN A MAMMALIAN SUBJECT IN VIVO VIA AAV-MEDIATED RNAi EXPRESSION CASSETTE TRANSFER
EP1688356A3 (en) * 2005-02-04 2006-08-16 The Procter and Gamble Company Injection stretch blow-molded container
WO2006089340A2 (en) * 2005-02-23 2006-08-31 V-Kardia Pty Ltd Polynucleotide delivery to cardiac tissue
US20060198825A1 (en) * 2005-03-03 2006-09-07 Kaemmerer William F Reagents, methods and systems to suppress phospholamban expression
GB0518027D0 (en) * 2005-09-05 2005-10-12 Birkeland Innovasjon As Compounds
EP1939289A1 (en) * 2006-12-29 2008-07-02 Charité-Universitätsmedizin Berlin Polynucleotide sequence for the inhibition of phospholamban synthesis

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN108192922A (zh) * 2018-01-03 2018-06-22 李江 受磷蛋白基因反义rna重组aav载体、病毒、构建方法及应用

Also Published As

Publication number Publication date
WO2009088786A1 (en) 2009-07-16
NZ587060A (en) 2012-09-28
EP2225002A1 (en) 2010-09-08
US20110098338A1 (en) 2011-04-28
US8404658B2 (en) 2013-03-26
CA2711179A1 (en) 2009-07-16
JP2011512326A (ja) 2011-04-21
EP2225002A4 (en) 2011-06-22
CN101970051A (zh) 2011-02-09
US20130243730A1 (en) 2013-09-19

Similar Documents

Publication Publication Date Title
US8404658B2 (en) RNA interference for the treatment of heart failure
US11999965B2 (en) Bocaparvovirus small noncoding RNA and uses thereof
JP2022523632A (ja) ＣＲＩＳＰＲ－Ｃａｓによる標的化された核内ＲＮＡ切断及びポリアデニル化
WO2011133901A2 (en) Aav-based treatment of cholesterol-related disorders
CN113874513A (zh) 非病毒dna载体及其用于表达fviii治疗剂的用途
US20250327094A1 (en) Modified aav constructs and uses thereof
EP2699673B1 (en) Recombinant virus products and methods for inhibition of expression of myotilin
US12325856B2 (en) Recombinant virus products and methods for inhibition of expression of myotilin
CN113874508A (zh) 非病毒dna载体及其用于表达苯丙氨酸羟化酶(pah)治疗剂的用途
TW201706412A (zh) Ｆａｂｒｙ氏病基因療法
US20240181085A1 (en) Non-viral dna vectors and uses thereof for expressing pfic therapeutics
US20230138409A1 (en) Non-viral dna vectors and uses thereof for expressing factor ix therapeutics
US20220098614A1 (en) Compositions and Methods for Treating Oculopharyngeal Muscular Dystrophy (OPMD)
WO2025102034A1 (en) Gene therapy for barth syndrome
EP3350319B1 (en) Recombinant virus products and methods for inhibition of expression of myotilin
HK1153969A (en) Rna interference for the treatment of heart failure
US20260028624A1 (en) Aav-mediated delivery of osteoblast/osteoclast-regulating mirnas for osteoporosis therapy
RU2814137C2 (ru) Невирусные днк-векторы и варианты их применения для экспрессии терапевтического средства на основе фенилаланингидроксилазы (pah)
WO2025212926A1 (en) Aav-mediated rhoa knockdown
HK1258752B (en) Recombinant virus products and methods for inhibition of expression of myotilin

Legal Events

Date Code Title Description
MK4 Application lapsed section 142(2)(d) - no continuation fee paid for the application