CA2961555A1 - Adeno-associated virus vector variants for high efficiency genome editing and methods thereof - Google Patents

Adeno-associated virus vector variants for high efficiency genome editing and methods thereof Download PDF

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Publication number
CA2961555A1
CA2961555A1 CA2961555A CA2961555A CA2961555A1 CA 2961555 A1 CA2961555 A1 CA 2961555A1 CA 2961555 A CA2961555 A CA 2961555A CA 2961555 A CA2961555 A CA 2961555A CA 2961555 A1 CA2961555 A1 CA 2961555A1
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Prior art keywords
aav
cell
capsid
clade
nucleotide sequence
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CA2961555A
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English (en)
French (fr)
Inventor
Saswati Chatterjee
Laura Smith
Kamehameha Wong
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City of Hope
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City of Hope
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • 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
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A—HUMAN NECESSITIES
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    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
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    • 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
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    • 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
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    • 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
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
    • C12N15/8645—Adeno-associated virus
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    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
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    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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
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    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/16011—Herpesviridae
    • C12N2710/16611—Simplexvirus, e.g. human herpesvirus 1, 2
    • C12N2710/16622—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
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    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • C12N2750/14144—Chimeric viral vector comprising heterologous viral elements for production of another viral vector
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    • 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
    • C12N2750/14145—Special targeting system for viral vectors
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14151—Methods of production or purification of viral material
    • C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14171—Demonstrated in vivo effect
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/24—Vectors characterised by the absence of particular element, e.g. selectable marker, viral origin of replication
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/70—Vectors containing special elements for cloning, e.g. topoisomerase, adaptor sites
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    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/44—Vectors comprising a special translation-regulating system being a specific part of the splice mechanism, e.g. donor, acceptor

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  • Animal Behavior & Ethology (AREA)
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  • Public Health (AREA)
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  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Epidemiology (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Oncology (AREA)
  • Communicable Diseases (AREA)
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CA2961555A 2014-09-24 2015-09-23 Adeno-associated virus vector variants for high efficiency genome editing and methods thereof Pending CA2961555A1 (en)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201462054899P 2014-09-24 2014-09-24
US62/054,899 2014-09-24
US201462063587P 2014-10-14 2014-10-14
US62/063,587 2014-10-14
US201562209862P 2015-08-25 2015-08-25
US62/209,862 2015-08-25
PCT/US2015/051785 WO2016049230A1 (en) 2014-09-24 2015-09-23 Adeno-associated virus vector variants for high efficiency genome editing and methods thereof

Publications (1)

Publication Number Publication Date
CA2961555A1 true CA2961555A1 (en) 2016-03-31

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CA2961555A Pending CA2961555A1 (en) 2014-09-24 2015-09-23 Adeno-associated virus vector variants for high efficiency genome editing and methods thereof

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US (6) US9890396B2 (enExample)
EP (2) EP3198018B1 (enExample)
JP (3) JP6683691B2 (enExample)
KR (2) KR102526711B1 (enExample)
CN (2) CN113667696A (enExample)
AU (2) AU2015320694B2 (enExample)
BR (1) BR112017005892A2 (enExample)
CA (1) CA2961555A1 (enExample)
DK (1) DK3198018T3 (enExample)
ES (1) ES2856090T3 (enExample)
MX (2) MX388392B (enExample)
PL (1) PL3198018T3 (enExample)
PT (1) PT3198018T (enExample)
SG (3) SG11201702073XA (enExample)
WO (1) WO2016049230A1 (enExample)

Families Citing this family (206)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN102199626B (zh) 2003-09-30 2015-06-24 宾夕法尼亚大学托管会 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用
ES2400235T3 (es) 2006-04-28 2013-04-08 The Trustees Of The University Of Pennsylvania Método de producción escalable de AAV
US9839696B2 (en) 2010-04-30 2017-12-12 City Of Hope Recombinant adeno-associated vectors for targeted treatment
CA2853829C (en) 2011-07-22 2023-09-26 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
JP6591956B2 (ja) 2013-03-15 2019-10-16 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア Ｍｐｓ１を治療するための組成物および方法
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
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US9228207B2 (en) 2013-09-06 2016-01-05 President And Fellows Of Harvard College Switchable gRNAs comprising aptamers
US20150166982A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting pi3k point mutations
EP3177718B1 (en) 2014-07-30 2022-03-16 President and Fellows of Harvard College Cas9 proteins including ligand-dependent inteins
EP3307310A2 (en) 2015-05-13 2018-04-18 The Trustees Of The University Of Pennsylvania Aav-mediated expression of anti-influenza antibodies and methods of use thereof
US10966414B2 (en) 2015-05-26 2021-04-06 California Institute Of Technology Population control using engineered translocations
CN114606267A (zh) 2015-09-28 2022-06-10 北卡罗来纳-查佩尔山大学 逃避抗体的病毒载体的方法和组合物
SG11201803173VA (en) 2015-10-23 2018-05-30 Harvard College Nucleobase editors and uses thereof
EP3368054A4 (en) 2015-10-28 2019-07-03 Voyager Therapeutics, Inc. REGULATORY EXPRESSION USING THE ADENO-ASSOCIATED VIRUS (AAV)
US10406244B2 (en) 2015-12-02 2019-09-10 The Board Of Trustees Of The Leland Stanford Junior University AAV vectors with expanded packaging capacity
US10385320B2 (en) 2015-12-02 2019-08-20 The Board Of Trustees Of The Leland Stanford Junior University Recombinant adeno-associated virus capsids with enhanced human skeletal muscle tropism
WO2017100674A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Scalable purification method for aav1
US11098286B2 (en) 2015-12-11 2021-08-24 The Trustees Of The University Of Pennsylvania Scalable purification method for AAV9
EP3387118B1 (en) 2015-12-11 2022-04-06 The Trustees Of The University Of Pennsylvania Scalable purification method for aavrh10
WO2017100676A1 (en) 2015-12-11 2017-06-15 The Trustees Of The University Of Pennsylvania Scalable purification method for aav8
MA43968A (fr) 2016-02-03 2018-12-12 Univ Pennsylvania Thérapie génique pour traiter la mucopolysaccharidose de type i
AU2017219865B2 (en) 2016-02-16 2023-04-13 The Board Of Trustees Of The Leland Stanford Junior University Novel recombinant adeno-associated virus capsids resistant to pre-existing human neutralizing antibodies
MA44874A (fr) 2016-04-15 2019-03-13 Univ Pennsylvania Thérapie génique pour traiter la mucopolysaccharidose de type ii
WO2017192750A1 (en) * 2016-05-04 2017-11-09 Oregon Health & Science University Recombinant adeno-associated viral vectors
IL302748B1 (en) 2016-05-18 2025-09-01 Voyager Therapeutics Inc Modulatory polynucleotides
KR20230161535A (ko) * 2016-07-26 2023-11-27 바이오마린 파머수티컬 인크. 신규 아데노-관련 바이러스 캡시드 단백질
JP7231935B2 (ja) 2016-08-03 2023-03-08 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 アデノシン核酸塩基編集因子およびそれらの使用
CA3033327A1 (en) 2016-08-09 2018-02-15 President And Fellows Of Harvard College Programmable cas9-recombinase fusion proteins and uses thereof
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
WO2018071868A1 (en) 2016-10-14 2018-04-19 President And Fellows Of Harvard College Aav delivery of nucleobase editors
EP3535396A1 (en) 2016-11-01 2019-09-11 Novartis AG Methods and compositions for enhancing gene editing
US10745677B2 (en) 2016-12-23 2020-08-18 President And Fellows Of Harvard College Editing of CCR5 receptor gene to protect against HIV infection
KR20190135000A (ko) 2017-02-28 2019-12-05 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 Aav 벡터를 기반으로 하는 인플루엔자 백신
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
CN118956781A (zh) * 2017-02-28 2024-11-15 宾夕法尼亚州大学信托人 腺相关病毒(aav)进化枝f载体及其用途
US12390514B2 (en) 2017-03-09 2025-08-19 President And Fellows Of Harvard College Cancer vaccine
EP3592853A1 (en) 2017-03-09 2020-01-15 President and Fellows of Harvard College Suppression of pain by gene editing
JP2020510439A (ja) 2017-03-10 2020-04-09 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 シトシンからグアニンへの塩基編集因子
KR20240116572A (ko) 2017-03-23 2024-07-29 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵산 프로그램가능한 dna 결합 단백질을 포함하는 핵염기 편집제
WO2018204722A1 (en) * 2017-05-05 2018-11-08 Califorinia Institute Of Technology Dna sequence modification-based gene drive
IL270422B2 (en) 2017-05-11 2025-04-01 Univ Pennsylvania Gene therapy for neuronal ceroid lipofuscinosis
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
CN107034233B (zh) * 2017-05-18 2021-06-08 中国科学院重庆绿色智能技术研究院 一种内源性启动子驱动外源基因表达的方法
SG11201911623WA (en) 2017-06-15 2020-01-30 Toolgen Inc Platform for expressing protein of interest in liver
WO2019010335A1 (en) 2017-07-06 2019-01-10 The Trustees Of The University Of Pennsylvania AAV9 MEDIATED GENE THERAPY FOR TREATING MUCOPOLYSACCHARIDOSIS TYPE I
EP3658573A1 (en) 2017-07-28 2020-06-03 President and Fellows of Harvard College Methods and compositions for evolving base editors using phage-assisted continuous evolution (pace)
CN111448308A (zh) 2017-08-03 2020-07-24 沃雅戈治疗公司 递送aav的组合物和方法
WO2019139645A2 (en) 2017-08-30 2019-07-18 President And Fellows Of Harvard College High efficiency base editors comprising gam
IL273427B2 (en) 2017-09-22 2025-04-01 Univ Pennsylvania Gene therapy for the treatment of mucocutaneous polycythemia vera type II
AU2018352592C1 (en) 2017-10-16 2025-09-25 Beam Therapeutics, Inc. Uses of adenosine base editors
BR112020007765A2 (pt) * 2017-10-18 2020-10-20 City Of Hope composições de vírus adeno-associados para restaurar a função do gene hbb e métodos de uso das mesmas
CN107937441B (zh) * 2017-11-14 2021-02-26 中国科学院苏州生物医学工程技术研究所 AAVS1位点定点整合外源基因的人iPS细胞的构建方法
CA3083416A1 (en) 2017-11-30 2019-06-06 The Trustees Of The University Of Pennsylvania Gene therapy for mucopolysaccharidosis iii a
KR20200104864A (ko) 2017-11-30 2020-09-04 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 뮤코다당류증 iiib형에 대한 유전자 요법
EP3724214A4 (en) 2017-12-15 2021-09-01 The Broad Institute Inc. SYSTEMS AND METHODS FOR PREDICTING REPAIR RESULTS IN GENETIC ENGINEERING
WO2019152843A1 (en) * 2018-02-01 2019-08-08 Homology Medicines, Inc. Adeno-associated virus compositions for restoring pah gene function and methods of use thereof
CN111886021A (zh) * 2018-02-01 2020-11-03 同源药物公司 用于pah基因转移的腺伴随病毒组合物及其使用方法
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
US11306329B2 (en) 2018-02-19 2022-04-19 City Of Hope Adeno-associated virus compositions for restoring F8 gene function and methods of use thereof
KR102848518B1 (ko) * 2018-02-27 2025-08-22 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 캡시드 탈아미드화가 감소된 신규 아데노-연관 바이러스(aav) 벡터 및 이의 용도
BR112020020266A2 (pt) 2018-04-03 2021-01-19 Stridebio, Inc. Vetores de vírus com evasão de anticorpos
EP3774852A1 (en) 2018-04-03 2021-02-17 Stridebio, Inc. Antibody-evading virus vectors
EA202092362A1 (ru) 2018-04-03 2021-01-11 Страйдбайо, Инк. Вирусные векторы для нацеливания на ткани глаза
BR112020020223A2 (pt) 2018-04-05 2021-01-19 Genethon Sorotipo de vírus adeno-associado híbrido recombinante entre aav9 e aavrh74 com tropismo pelo fígado reduzido
JP7575273B2 (ja) 2018-04-16 2024-10-29 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア デュシェンヌ型筋ジストロフィーを治療するための組成物及び方法
EP3788165A1 (en) 2018-04-29 2021-03-10 REGENXBIO Inc. Systems and methods of spectrophotometry for the determination of genome content, capsid content and full/empty ratios of adeno-associated virus particles
CA3098565A1 (en) 2018-04-29 2019-11-07 Claire G. ZHANG Scalable clarification process for recombinant aav production
AU2019265560A1 (en) 2018-05-09 2020-11-26 Biomarin Pharmaceutical Inc. Methods of treating phenylketonuria
TW202005978A (zh) 2018-05-14 2020-02-01 美商拜奧馬林製藥公司 新穎肝靶向腺相關病毒載體
US20210230632A1 (en) 2018-05-15 2021-07-29 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
CA3099306A1 (en) 2018-05-15 2019-11-21 Voyager Therapeutics, Inc. Compositions and methods for the treatment of parkinson's disease
US12157760B2 (en) 2018-05-23 2024-12-03 The Broad Institute, Inc. Base editors and uses thereof
WO2019241535A2 (en) 2018-06-14 2019-12-19 Regenxbio Inc. Anion exchange chromatography for recombinant aav production
JP7565218B2 (ja) * 2018-07-02 2024-10-10 ボイジャー セラピューティクス インコーポレイテッド 筋萎縮性側索硬化症および脊髄に関連する障害の治療
CN112567027A (zh) 2018-08-10 2021-03-26 再生生物股份有限公司 用于重组aav生产的可扩展方法
SG11202101576UA (en) 2018-08-18 2021-03-30 Harvard College In situ gene editing
EP3856762A1 (en) 2018-09-28 2021-08-04 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
CA3113975A1 (en) 2018-10-01 2020-04-09 Ultragenyx Pharmaceutical Inc. Gene therapy for treating propionic acidemia
CN113438954A (zh) 2018-10-01 2021-09-24 宾夕法尼亚州大学信托人 可用于治疗gm1神经节苷脂病的组合物
US20210371470A1 (en) 2018-10-12 2021-12-02 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
US20210324483A1 (en) 2018-10-15 2021-10-21 Regenxbio Inc. Method for measuring the infectivity of replication defective viral vectors and viruses
US12281338B2 (en) 2018-10-29 2025-04-22 The Broad Institute, Inc. Nucleobase editors comprising GeoCas9 and uses thereof
US11965172B2 (en) 2018-11-05 2024-04-23 California Institute Of Technology DNA sequence modification-based gene drive
SG11202106855YA (en) 2019-01-04 2021-07-29 Ultragenyx Pharmaceutical Inc Gene therapy constructs for treating wilson disease
WO2020150556A1 (en) 2019-01-18 2020-07-23 Voyager Therapeutics, Inc. Methods and systems for producing aav particles
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
CA3130055A1 (en) 2019-02-26 2020-09-03 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of krabbe disease
GB2601617B (en) 2019-03-19 2024-02-21 Broad Inst Inc Methods and compositions for editing nucleotide sequences
BR112021018776A2 (pt) 2019-03-21 2021-11-30 Stridebio Inc Vetores de vírus adenoassociado recombinante
TW202102526A (zh) 2019-04-04 2021-01-16 美商銳進科斯生物股份有限公司 重組腺相關病毒及其用途
HUE064411T2 (hu) 2019-04-11 2024-03-28 Regenxbio Inc Méretkizárásos kromatográfiás módszerek rekombináns adeno-asszociált víruskészítmények jellemzésére
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
MX2021012564A (es) 2019-04-19 2022-01-18 Regenxbio Inc Formulaciones y métodos de vectores de virus adenoasociados.
CN114144197A (zh) 2019-04-24 2022-03-04 再生生物股份有限公司 完全人类翻译后修饰的抗体治疗剂
AU2020270421A1 (en) 2019-05-03 2021-11-18 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of metachromatic leukodystrophy
US20240124889A1 (en) 2019-05-07 2024-04-18 Voyager Therapeutics, Inc. Compositions and methods for the vectored augmentation of protein destruction, expression and/or regulation
WO2021005223A1 (en) 2019-07-10 2021-01-14 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods for the treatment of epilepsy
US20230042103A1 (en) 2019-07-26 2023-02-09 Regenxbio Inc. Engineered nucleic acid regulatory element and methods of uses thereof
US20220333133A1 (en) 2019-09-03 2022-10-20 Voyager Therapeutics, Inc. Vectorized editing of nucleic acids to correct overt mutations
EP4038190A1 (en) 2019-10-03 2022-08-10 Artisan Development Labs, Inc. Crispr systems with engineered dual guide nucleic acids
JP2022550435A (ja) 2019-10-04 2022-12-01 ウルトラジェニックス ファーマシューティカル インコーポレイテッド 組換えａａｖの改善された治療的使用のための方法
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
CN114787180A (zh) 2019-10-17 2022-07-22 斯特里迪比奥公司 用于治疗c型尼曼-匹克病的腺相关病毒载体
WO2021099394A1 (en) 2019-11-19 2021-05-27 INSERM (Institut National de la Santé et de la Recherche Médicale) Antisense oligonucleotides and their use for the treatment of cancer
JP2023503637A (ja) 2019-11-28 2023-01-31 リジェネックスバイオ インコーポレイテッド マイクロジストロフィン遺伝子治療コンストラクト及びその使用
CN113122657B (zh) * 2019-12-31 2022-07-29 上海吉凯基因医学科技股份有限公司 一种多重aav病毒滴度的检测方法
TW202140791A (zh) 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 治療苯酮尿症之方法
CA3165057A1 (en) 2020-02-02 2021-08-05 James M. Wilson Compositions useful for treating gm1 gangliosidosis
MX2022009982A (es) 2020-02-14 2022-09-12 Ultragenyx Pharmaceutical Inc Terapia genica para el tratamiento de transtorno por deficiencia de cdkl5.
US20230140435A1 (en) * 2020-03-16 2023-05-04 The Trustees Of The University Of Pennsylvania Composition and methods for improving heart function and treating heart failure
EP4127189A1 (en) 2020-03-31 2023-02-08 Ultragenyx Pharmaceutical Inc. Gene therapy for treating propionic acidemia
US20230131352A1 (en) 2020-04-01 2023-04-27 Voyager Therapeutics, Inc. Redirection of tropism of aav capsids
KR20230020394A (ko) 2020-04-15 2023-02-10 보이저 테라퓨틱스, 인크. Tau 결합 화합물
DE112021002672T5 (de) 2020-05-08 2023-04-13 President And Fellows Of Harvard College Vefahren und zusammensetzungen zum gleichzeitigen editieren beider stränge einer doppelsträngigen nukleotid-zielsequenz
WO2021231443A1 (en) 2020-05-12 2021-11-18 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of krabbe disease
BR112022022212A2 (pt) 2020-05-12 2022-12-13 Univ Pennsylvania Composições para redução específica de drg de expressão de transgene
PE20230767A1 (es) 2020-05-13 2023-05-09 Voyager Therapeutics Inc Redireccion del tropismo de las capsides de aav
TW202208632A (zh) 2020-05-27 2022-03-01 美商同源醫藥公司 用於恢復ｐａｈ基因功能的腺相關病毒組成物及其使用方法
WO2021247995A2 (en) 2020-06-04 2021-12-09 Voyager Therapeutics, Inc. Compositions and methods of treating neuropathic pain
CA3177613A1 (en) 2020-07-10 2022-04-13 Inserm (Institut National De La Sante Et De La Recherche Medicale) Methods and compositions for treating epilepsy
WO2022026410A2 (en) 2020-07-27 2022-02-03 Voyager Therapeutics, Inc Compositions and methods for the treatment of niemann-pick type c1 disease
TW202221125A (zh) 2020-07-27 2022-06-01 美商航海家醫療公司 用於治療與葡萄糖神經醯胺酶β缺陷相關之神經病症的組合物及方法
US12104163B2 (en) 2020-08-19 2024-10-01 Sarepta Therapeutics, Inc. Adeno-associated virus vectors for treatment of Rett syndrome
WO2022060916A1 (en) 2020-09-15 2022-03-24 Regenxbio Inc. Vectorized antibodies for anti-viral therapy
EP4213890A1 (en) 2020-09-15 2023-07-26 RegenxBio Inc. Vectorized lanadelumab and administration thereof
WO2022061002A1 (en) 2020-09-18 2022-03-24 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Novel adeno-associated viral (aav) vectors to treat hereditary methylmalonic acidemia (mma) caused by methylmalonyl-coa mutase (mmut) deficiency
JP2023545722A (ja) 2020-10-07 2023-10-31 リジェネックスバイオ インコーポレイテッド 遺伝子治療剤の眼送達のためのアデノ随伴ウイルス
WO2022076750A2 (en) 2020-10-07 2022-04-14 Regenxbio Inc. Recombinant adeno-associated viruses for cns or muscle delivery
JP2023544803A (ja) 2020-10-07 2023-10-25 レジェンクスバイオ インコーポレーテッド Ｃｌｎ２疾患の眼症状に対する遺伝子療法
JP2023545433A (ja) 2020-10-09 2023-10-30 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア ファブリー病の治療のための組成物及び方法
WO2022094157A1 (en) 2020-10-28 2022-05-05 Regenxbio Inc. Vectorized anti-cgrp and anti-cgrpr antibodies and administration thereof
WO2022094078A1 (en) 2020-10-28 2022-05-05 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of rett syndrome
AR123948A1 (es) 2020-10-28 2023-01-25 Regenxbio Inc ANTICUERPOS ANTI-TNF-a VECTORIZADOS PARA INDICACIONES OCULARES
IL302282A (en) 2020-10-29 2023-06-01 Regenxbio Inc Vectorized tnf-alpha antagonists for ocular indications
WO2022094255A2 (en) 2020-10-29 2022-05-05 Regenxbio Inc. Vectorized factor xii antibodies and administration thereof
US20240018493A1 (en) * 2020-11-10 2024-01-18 The Board Of Trustees Of The Leland Stanford Junior University Knock-in of large dna for long-term high genomic expression
MX2023006444A (es) 2020-12-01 2023-08-10 Univ Pennsylvania Composiciones novedosas con motivos dirigidos a tejido específico y composiciones que los contienen.
IL303614A (en) 2020-12-16 2023-08-01 Regenxbio Inc Method of producing a recombinant adeno-associated virus particle
WO2022147087A1 (en) 2020-12-29 2022-07-07 Regenxbio Inc. Tau-specific antibody gene therapy compositions, methods and uses thereof
WO2022159662A1 (en) 2021-01-21 2022-07-28 Regenxbio Inc. Improved production of recombinant polypeptides and viruses
JP2024505257A (ja) 2021-02-01 2024-02-05 レジェンクスバイオ インコーポレーテッド 神経セロイドリポフスチン症の遺伝子治療
AU2022227650A1 (en) 2021-02-25 2023-10-12 Celyntra Therapeutics Sa Compositions and methods for targeting, editing, or modifying genes
CN115197967B (zh) * 2021-04-08 2023-09-15 广州派真生物技术有限公司 制备重组腺相关病毒的辅助质粒及其应用
WO2022226263A1 (en) 2021-04-23 2022-10-27 The Trustees Of The University Of Pennsylvania Novel compositions with brain-specific targeting motifs and compositions containing same
WO2022232117A1 (en) * 2021-04-26 2022-11-03 University Of Massachusetts Direct raav-mediated in vivo gene editing of hematopoietic stem cells
JP2024517143A (ja) 2021-04-26 2024-04-19 リジェネックスバイオ インコーポレイテッド ジストロフィン異常症の治療のためのマイクロジストロフィン遺伝子療法投与
WO2022235614A2 (en) 2021-05-04 2022-11-10 Regenxbio Inc. Novel aav vectors and methods and uses thereof
EP4337267A1 (en) 2021-05-11 2024-03-20 RegenxBio Inc. Treatment of duchenne muscular dystrophy and combinations thereof
US20250179481A1 (en) 2021-06-01 2025-06-05 Celyntra Therapeutics Sa Compositions and methods for targeting, editing, or modifying genes
CA3223311A1 (en) 2021-06-18 2022-12-22 Andrea BARGHETTI Compositions and methods for targeting, editing or modifying human genes
WO2022272297A1 (en) 2021-06-25 2022-12-29 Oxford Biomedica Solutions Llc Adeno-associated virus packaging systems
EP4392016A1 (en) 2021-08-24 2024-07-03 Oxford Biomedica (US) LLC Adeno-associated virus formulations
CN118202060A (zh) 2021-10-05 2024-06-14 再生生物股份有限公司 用于重组aav生产的组合物和方法
WO2023060113A1 (en) 2021-10-05 2023-04-13 Regenxbio Inc. Compositions and methods for recombinant aav production
WO2023060269A1 (en) 2021-10-07 2023-04-13 Regenxbio Inc. Recombinant adeno-associated viruses for targeted delivery
WO2023060272A2 (en) 2021-10-07 2023-04-13 Regenxbio Inc. Recombinant adeno-associated viruses for cns tropic delivery
EP4423285A1 (en) 2021-10-28 2024-09-04 RegenxBio Inc. Engineered nucleic acid regulatory elements and methods and uses thereof
US20230183658A1 (en) 2021-10-29 2023-06-15 Oxford Biomedica Solutions Llc Methods and compositions for the purification of adeno-associated virus
US20250295807A1 (en) 2021-11-15 2025-09-25 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
WO2023093905A1 (zh) 2021-11-29 2023-06-01 上海瑞宏迪医药有限公司 Aadc、gdnf多核苷酸及其用于治疗帕金森病
WO2023102517A1 (en) 2021-12-02 2023-06-08 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of fabry disease
US20230279433A1 (en) 2021-12-15 2023-09-07 Oxford Biomedica Solutions Llc Methods and compositions for the production of adeno-associated virus
WO2023133584A1 (en) 2022-01-10 2023-07-13 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of metachromatic leukodystrophy
AU2023211652A1 (en) 2022-01-25 2024-08-08 The Trustees Of The University Of Pennsylvania Aav capsids for improved heart transduction and detargeting of liver
WO2023155918A1 (zh) 2022-02-21 2023-08-24 上海瑞宏迪医药有限公司 Vegf结合分子及其医药用途
WO2023167882A1 (en) 2022-03-01 2023-09-07 Artisan Development Labs, Inc. Composition and methods for transgene insertion
WO2023178053A1 (en) 2022-03-13 2023-09-21 Regenxbio Inc. Modified muscle-specific promoters
EP4499154A1 (en) 2022-03-25 2025-02-05 REGENXBIO Inc. Dominant-negative tumor necrosis factor alpha adeno-associated virus gene therapy
WO2023201308A1 (en) 2022-04-14 2023-10-19 Regenxbio Inc. Gene therapy for treating an ocular disease
WO2023201277A1 (en) 2022-04-14 2023-10-19 Regenxbio Inc. Recombinant adeno-associated viruses for cns tropic delivery
US20250288697A1 (en) 2022-05-03 2025-09-18 Regenxbio Inc. Vectorized anti-tnf-alpha inhibitors for ocular indications
WO2023215806A2 (en) 2022-05-03 2023-11-09 Regenxbio Inc. Vectorized anti-complement antibodies and complement agents and administration thereof
US20250308637A1 (en) 2022-05-20 2025-10-02 Celyntra Therapeutics Sa Systems and methods for assessing risk of genome editing events
WO2023239627A2 (en) 2022-06-08 2023-12-14 Regenxbio Inc. Methods for recombinant aav production
WO2024017990A1 (en) 2022-07-21 2024-01-25 Institut National de la Santé et de la Recherche Médicale Methods and compositions for treating chronic pain disorders
JP2025527658A (ja) 2022-08-24 2025-08-22 リジェネックスバイオ インコーポレイテッド 組換えアデノ随伴ウイルス及びその使用
WO2024062138A1 (en) 2022-09-23 2024-03-28 Mnemo Therapeutics Immune cells comprising a modified suv39h1 gene
CN115948408A (zh) * 2022-09-23 2023-04-11 上海信致医药科技有限公司 改进的人凝血因子viii基因表达盒及其应用
WO2024081746A2 (en) 2022-10-11 2024-04-18 Regenxbio Inc. Engineered nucleic acid regulatory elements and methods and uses thereof
CN115896135B (zh) * 2022-11-02 2024-03-01 苏州诺洁贝生物技术有限公司 优化的pah基因和表达盒及其用途
WO2024130070A2 (en) 2022-12-17 2024-06-20 The Trustees Of The University Of Pennsylvania Recombinant aav capsids with cardiac- and skeletal muscle- specific targeting motifs and uses thereof
WO2024130067A2 (en) 2022-12-17 2024-06-20 The Trustees Of The University Of Pennsylvania Recombinant aav mutant vectors with cardiac and skeletal muscle-specific targeting motifs and compositions containing same
CN120752339A (zh) 2023-01-06 2025-10-03 国家医疗保健研究所 静脉内施用用于治疗疼痛的反义寡核苷酸
WO2024163012A1 (en) 2023-02-02 2024-08-08 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
CN116726198B (zh) * 2023-03-13 2024-02-13 陕西师范大学 一种抗肿瘤药物及其在脑胶质瘤治疗中的应用
WO2024192281A2 (en) 2023-03-15 2024-09-19 Regenxbio Inc. Exon skipping gene therapy constructs, vectors and uses thereof
WO2024211780A1 (en) 2023-04-07 2024-10-10 Regenxbio Inc. Compositions and methods for recombinant aav production
WO2024216244A2 (en) 2023-04-13 2024-10-17 Regenxbio Inc. Targeting aav capsids, methods of manufacturing and using same
WO2024233529A2 (en) 2023-05-07 2024-11-14 Regenxbio Inc. Compositions and methods for recombinant aav production
WO2024238867A1 (en) 2023-05-16 2024-11-21 Regenxbio Inc. Vectorized anti-complement antibodies and administration thereof
WO2024238859A1 (en) 2023-05-16 2024-11-21 Regenxbio Inc. Vectorized c5 inhibitor agents and administration thereof
WO2024238853A1 (en) 2023-05-16 2024-11-21 Regenxbio Inc. Adeno-associated viruses for ocular delivery of gene therapy
AR133098A1 (es) 2023-06-29 2025-08-27 Univ Pennsylvania Aav mutante con motivos dirigidos al sistema nervioso central y composiciones que lo contienen
WO2025008406A1 (en) 2023-07-04 2025-01-09 Institut National de la Santé et de la Recherche Médicale Antisense oligonucleotides and their use for the treatment of cancer
CN119320802A (zh) * 2023-07-17 2025-01-17 苏州吉恒基因科技有限公司 单链单极DNA重组腺相关病毒的cis包装元件、spAAV载体和应用
WO2025090962A1 (en) 2023-10-25 2025-05-01 Regenxbio Inc. Compositions and methods for recombinant aav production
WO2025106661A1 (en) 2023-11-14 2025-05-22 The Trustees Of The University Of Pennsylvania Compositions with cardiac and skeletal musclespecific targeting motifs and uses thereof
WO2025108407A2 (en) 2023-11-23 2025-05-30 Neuexcell Therapeutics (Suzhou) Co., Ltd. Gene therapy compositions and methods for treating glioma
WO2025113676A1 (en) 2023-11-29 2025-06-05 Neuexcell Therapeutics (Suzhou) Co., Ltd. Compositions and methods for treating stroke in primates
WO2025217230A1 (en) 2024-04-08 2025-10-16 Regenxbio Inc. Vectorized anti-complement antibodies and complement agents and administration thereof
WO2025217214A2 (en) 2024-04-08 2025-10-16 Regenxbio Inc. Recombinant adeno-associated viruses and uses thereof
WO2025237990A1 (en) 2024-05-14 2025-11-20 Institut National de la Santé et de la Recherche Médicale Antisense oligonucleotides and their use for the treatment of pulmonary fibrosis
CN121006370A (zh) * 2024-05-23 2025-11-25 深圳市深研生物科技有限公司 提高腺相关病毒生产效率的方法

Family Cites Families (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4866042A (en) 1987-11-18 1989-09-12 Neuwelt Edward A Method for the delivery of genetic material across the blood brain barrier
WO1991018088A1 (en) 1990-05-23 1991-11-28 The United States Of America, Represented By The Secretary, United States Department Of Commerce Adeno-associated virus (aav)-based eucaryotic vectors
US5252479A (en) 1991-11-08 1993-10-12 Research Corporation Technologies, Inc. Safe vector for gene therapy
US6207457B1 (en) 1995-09-08 2001-03-27 Avigen, Inc. Targeted nucleotide sequence delivery and integration system
CA2289277C (en) 1997-04-24 2013-02-12 University Of Washington Targeted gene modification by parvoviral vectors
US6534261B1 (en) 1999-01-12 2003-03-18 Sangamo Biosciences, Inc. Regulation of endogenous gene expression in cells using zinc finger proteins
EP2266396A3 (en) 2001-09-24 2011-06-15 Sangamo BioSciences, Inc. Modulation of stem cells using zinc finger proteins
WO2003089612A2 (en) * 2002-04-17 2003-10-30 University Of Florida Research Foundation, Inc. IMPROVED rAAV VECTORS
ATE405295T1 (de) * 2002-05-01 2008-09-15 Univ Florida Verbesserte raav-expressionssysteme für die genetische modifikation spezifischer capsidproteine
GB0322402D0 (en) * 2003-09-24 2003-10-22 Oxxon Pharmaccines Ltd HIV pharmaccines
CN102199626B (zh) 2003-09-30 2015-06-24 宾夕法尼亚大学托管会 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用
US20100310583A1 (en) 2007-01-31 2010-12-09 Immune Disease Institute Let-7 microrna and mimetics thereof as therapeutics for cancer
US20120094386A1 (en) 2009-03-11 2012-04-19 Sapphire Energy, Inc. Engineering salt tolerance in photosynthetic microorganisms
WO2010127097A1 (en) * 2009-04-30 2010-11-04 The Trustees Of The University Of Pennsylvania Compositions for targeting conducting airway cells comprising adeno-associated virus constructs
US8927514B2 (en) 2010-04-30 2015-01-06 City Of Hope Recombinant adeno-associated vectors for targeted treatment
US8628966B2 (en) * 2010-04-30 2014-01-14 City Of Hope CD34-derived recombinant adeno-associated vectors for stem cell transduction and systemic therapeutic gene transfer
ES2562421T3 (es) * 2010-10-12 2016-03-04 The Children's Hospital Of Philadelphia Métodos y composiciones para tratar la hemofilia B
EP2500434A1 (en) * 2011-03-12 2012-09-19 Association Institut de Myologie Capsid-free AAV vectors, compositions, and methods for vector production and gene delivery
WO2014186585A2 (en) 2013-05-15 2014-11-20 Sangamo Biosciences, Inc. Methods and compositions for treatment of a genetic condition
WO2015057976A1 (en) 2013-10-17 2015-04-23 Sangamo Biosciences, Inc. Delivery methods and compositions for nuclease-mediated genome engineering in hematopoietic stem cells
CA2939847C (en) 2014-03-21 2023-09-05 The Board Of Trustees Of The Leland Stanford Junior University Genome editing without nucleases

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