CN111132699A - 修饰的封闭端dna(cedna) - Google Patents

修饰的封闭端dna(cedna) Download PDF

Info

Publication number
CN111132699A
CN111132699A CN201880058155.7A CN201880058155A CN111132699A CN 111132699 A CN111132699 A CN 111132699A CN 201880058155 A CN201880058155 A CN 201880058155A CN 111132699 A CN111132699 A CN 111132699A
Authority
CN
China
Prior art keywords
itr
vector
cedna
itrs
seq
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.)
Pending
Application number
CN201880058155.7A
Other languages
English (en)
Chinese (zh)
Inventor
R·M·科廷
O·阿尔坎
A·琼斯
D·A·科尔
A·K·玛拉基安
M·J·西蒙斯
T·L·怀特
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.)
Generational Biology Co
Original Assignee
Generational Biology Co
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 Generational Biology Co filed Critical Generational Biology Co
Publication of CN111132699A publication Critical patent/CN111132699A/zh
Pending legal-status Critical Current

Links

Images

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/64General methods for preparing the vector, for introducing it into the cell or for selecting the vector-containing host
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/66General methods for inserting a gene into a vector to form a recombinant vector using cleavage and ligation; Use of non-functional linkers or adaptors, e.g. linkers containing the sequence for a restriction endonuclease
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/50Physical structure
    • C12N2310/53Physical structure partially self-complementary or closed
    • C12N2310/531Stem-loop; Hairpin
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2710/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011Details
    • C12N2710/14011Baculoviridae
    • C12N2710/14111Nucleopolyhedrovirus, e.g. autographa californica nucleopolyhedrovirus
    • C12N2710/14141Use of virus, viral particle or viral elements as a vector
    • C12N2710/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14121Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00Nucleic acids vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/48Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2840/00Vectors comprising a special translation-regulating system
    • C12N2840/60Vectors comprising a special translation-regulating system from viruses

Landscapes

  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Cell Biology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
CN201880058155.7A 2017-09-08 2018-09-07 修饰的封闭端dna(cedna) Pending CN111132699A (zh)

Applications Claiming Priority (13)

Application Number Priority Date Filing Date Title
US201762556335P 2017-09-08 2017-09-08
US201762556319P 2017-09-08 2017-09-08
US201762556281P 2017-09-08 2017-09-08
US201762556329P 2017-09-08 2017-09-08
US201762556324P 2017-09-08 2017-09-08
US201762556331P 2017-09-08 2017-09-08
US62/556,335 2017-09-08
US62/556,319 2017-09-08
US62/556,324 2017-09-08
US62/556,331 2017-09-08
US62/556,329 2017-09-08
US62/556,281 2017-09-08
PCT/US2018/049996 WO2019051255A1 (en) 2017-09-08 2018-09-07 CLOSED-ENDED DNA (CEDNA) MODIFIED

Publications (1)

Publication Number Publication Date
CN111132699A true CN111132699A (zh) 2020-05-08

Family

ID=65635235

Family Applications (1)

Application Number Title Priority Date Filing Date
CN201880058155.7A Pending CN111132699A (zh) 2017-09-08 2018-09-07 修饰的封闭端dna(cedna)

Country Status (15)

Country Link
US (1) US20200283794A1 (enExample)
EP (1) EP3678710A4 (enExample)
JP (3) JP2020532981A (enExample)
KR (1) KR20200051011A (enExample)
CN (1) CN111132699A (enExample)
AU (1) AU2018327348A1 (enExample)
BR (1) BR112020004151A2 (enExample)
CA (1) CA3075168A1 (enExample)
IL (1) IL272797A (enExample)
MA (1) MA50100A (enExample)
MX (1) MX2020002500A (enExample)
PH (1) PH12020500465A1 (enExample)
SG (1) SG11202000698SA (enExample)
UA (1) UA129010C2 (enExample)
WO (1) WO2019051255A1 (enExample)

Families Citing this family (44)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10704021B2 (en) 2012-03-15 2020-07-07 Flodesign Sonics, Inc. Acoustic perfusion devices
CN105939767B (zh) 2014-01-08 2018-04-06 弗洛设计声能学公司 具有双声电泳腔的声电泳装置
US11377651B2 (en) 2016-10-19 2022-07-05 Flodesign Sonics, Inc. Cell therapy processes utilizing acoustophoresis
US11708572B2 (en) 2015-04-29 2023-07-25 Flodesign Sonics, Inc. Acoustic cell separation techniques and processes
MY203184A (en) 2017-08-09 2024-06-13 Bioverativ Therapeutics Inc Nucleic acid molecules and uses thereof
EP3720952A4 (en) * 2017-12-06 2021-09-01 Generation Bio Co. GENE EDITING USING CLOSED-END MODIFIED DNA (ADNCE)
EP3725092A4 (en) 2017-12-14 2021-09-22 FloDesign Sonics, Inc. DRIVE AND CONTROL UNIT FOR ACOUSTIC CONVERTER
CA3091250A1 (en) * 2018-02-14 2019-08-22 Generation Bio Co. Non-viral dna vectors and uses thereof for antibody and fusion protein production
MX2021001599A (es) 2018-08-09 2021-07-02 Bioverativ Therapeutics Inc Moleculas de acido nucleico y sus usos para la terapia genica no viral.
CN113316640A (zh) * 2018-11-09 2021-08-27 世代生物公司 包含对称的被修饰的反向末端重复序列的被修饰的封闭端dna(cedna)
GB201905651D0 (en) 2019-04-24 2019-06-05 Lightbio Ltd Nucleic acid constructs and methods for their manufacture
EP3999646A4 (en) * 2019-07-17 2023-08-30 Generation Bio Co. COMPOSITIONS AND PRODUCTION OF NICKEL-CLOSED CLOSED-END DNA VECTOR
AU2020315444A1 (en) * 2019-07-17 2021-12-23 Generation Bio Co. Synthetic production of single-stranded adeno associated viral DNA vectors
BR112022002794A2 (pt) * 2019-08-15 2022-08-09 Childrens Hospital Philadelphia Terapia combinada de transgene e mirna derivado de íntron para tratamento de sca1
CA3150452A1 (en) * 2019-09-06 2021-03-11 Generation Bio Co. Lipid nanoparticle compositions comprising closed-ended dna and cleavable lipids and methods of use thereof
CN115066255A (zh) 2019-09-18 2022-09-16 星际治疗有限公司 合成的dna载体和使用方法
WO2021072031A1 (en) * 2019-10-11 2021-04-15 Insideoutbio, Inc. Methods and compositions for the manufacture and use of circular dna encoded therapeutics for genetic disorders and other diseases
WO2021169167A1 (en) * 2020-02-29 2021-09-02 Nanjing GenScript Biotech Co., Ltd. Method for treating coronavirus infections
DE102020111571A1 (de) 2020-03-11 2021-09-16 Immatics US, Inc. Wpre-mutantenkonstrukte, zusammensetzungen und zugehörige verfahren
CN115667530A (zh) * 2020-03-24 2023-01-31 世代生物公司 非病毒dna载体和其用于表达因子ix治疗剂的用途
US20230134550A1 (en) * 2020-03-24 2023-05-04 Generation Bio Co. Non-viral dna vectors and uses thereof for expressing gaucher therapeutics
CA3175957A1 (en) * 2020-05-18 2021-11-25 Generation Bio Co. Novel lipids and nanoparticle compositions thereof
BR112023001648A2 (pt) 2020-07-27 2023-04-04 Anjarium Biosciences Ag Moléculas de dna de fita dupla, veículo de entrega e método para preparar uma molécula de dna com extremidade em grampo
TW202227466A (zh) 2020-08-23 2022-07-16 美商百歐維拉提夫治療公司 用於改進封閉端DNA(ceDNA)的產生之經修飾的桿狀病毒系統
US20220243201A1 (en) * 2020-08-23 2022-08-04 Bioverativ Therapeutics Inc. Engineered itr sequences and methods of use
US20230383311A1 (en) * 2020-09-16 2023-11-30 Generation Bio Co. Non-viral dna vectors and uses thereof for expressing fviii therapeutics
KR20230066615A (ko) * 2020-09-16 2023-05-16 제너레이션 바이오 컴퍼니 폐쇄형 dna 벡터 및 페닐알라닌 히드록실라아제(pah) 발현을 위한 이의 용도
GB202014751D0 (en) * 2020-09-18 2020-11-04 Lightbio Ltd Targeting vector
CN117295530A (zh) * 2021-03-19 2023-12-26 世代生物公司 非病毒dna载体及其用于表达pfic治疗剂的用途
US20240209393A1 (en) * 2021-04-26 2024-06-27 University Of Florida Research Foundation, Incorporated Synthetic aav vectors for repeated delivery of therapeutic genes
MX2023012643A (es) * 2021-04-27 2024-01-05 Generation Bio Co Vectores de adn no virales que expresan anticuerpos terapéuticos y usos de estos.
WO2022232286A1 (en) * 2021-04-27 2022-11-03 Generation Bio Co. Non-viral dna vectors expressing anti-coronavirus antibodies and uses thereof
US20240277833A1 (en) * 2021-05-07 2024-08-22 Generation Bio Co. Non-viral dna vectors for vaccine delivery
EP4333906A4 (en) * 2021-05-07 2025-04-02 Generation Bio Co. Lyophilized non-viral dna vector compositions and uses thereof
EP4419677A4 (en) * 2021-10-18 2025-09-24 Flagship Pioneering Innovations Vii Llc DNA COMPOSITIONS AND RELATED METHODS
KR20240161965A (ko) 2022-03-14 2024-11-13 제너레이션 바이오 컴퍼니 이종 프라임 부스트 백신 조성물 및 사용 방법
CN117802161A (zh) * 2022-06-30 2024-04-02 苏州吉恒基因科技有限公司 精准重组腺相关病毒载体及其用途
WO2024040222A1 (en) * 2022-08-19 2024-02-22 Generation Bio Co. Cleavable closed-ended dna (cedna) and methods of use thereof
WO2024182707A1 (en) 2023-03-02 2024-09-06 Krystal Biotech, Inc. Interleukin-2 and interleukin-12 for cancer therapy
US11767541B1 (en) * 2023-03-27 2023-09-26 Genecraft, Inc. Adeno-associated virus complex with improved expression of RUNX3 gene and uses for preventing or treating KRAS mutated lung cancer
US11891617B1 (en) * 2023-03-27 2024-02-06 Genecraft Inc. Modified complex platform of adeno-associated virus with improved rate of expression of loaded genes and reduced genotoxicity
US11993783B1 (en) * 2023-03-27 2024-05-28 Genecraft Inc. Nucleic acid molecule comprising asymmetrically modified ITR for improving expression rate of inserted gene, and use thereof
WO2024220969A2 (en) * 2023-04-21 2024-10-24 Arizona Board Of Regents On Behalf Of Arizona State University Novel transmembrane sensors and method of characterization for lysis-free detection of intracellular targets
WO2025114932A1 (en) * 2023-11-30 2025-06-05 Sanofi Purification of closed-ended dna molecules

Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050019824A1 (en) * 1994-03-08 2005-01-27 Human Genome Sciences, Inc. Fibroblast Growth Factor-10
US20050026838A1 (en) * 1995-06-05 2005-02-03 Human Genome Sciences, Inc. Fibroblast Growth Factor-13
US20060166363A1 (en) * 2004-01-27 2006-07-27 Sergei Zolotukhin Modified baculovirus expression system for production of pseudotyped rAAV vector
US20070253936A1 (en) * 2006-01-31 2007-11-01 Kay Mark A Self-complementary parvoviral vectors, and methods for making and using the same
CN103764831A (zh) * 2011-03-11 2014-04-30 肌肉学研究协会 不含衣壳的aav载体、组合物以及制备载体和基因递送的方法
CN104087613A (zh) * 2014-06-30 2014-10-08 中国科学院苏州生物医学工程技术研究所 基于aav-itr的基因表达微载体及其构建方法和应用
WO2017123918A1 (en) * 2016-01-15 2017-07-20 American Gene Technologies International Inc. Methods and compositons for the activation of gamma-delta t-cells

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE10066104A1 (de) * 2000-09-08 2003-01-09 Medigene Ag Wirtszellen zur Verpackung von rekombinantem Adeno-assoziiertem Virus (rAAV), Verfahren zu ihrer Herstellung und deren Verwendung
US9943574B2 (en) * 2012-06-27 2018-04-17 Arthrogen B.V. Combination for treating an inflammatory disorder
MX2018010633A (es) * 2016-03-03 2019-06-13 Univ Massachusetts Acido desoxirribonucleico (adn) lineal duplex de extremos cerrados para transferencia genica no viral.

Patent Citations (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050019824A1 (en) * 1994-03-08 2005-01-27 Human Genome Sciences, Inc. Fibroblast Growth Factor-10
US20050026838A1 (en) * 1995-06-05 2005-02-03 Human Genome Sciences, Inc. Fibroblast Growth Factor-13
US20060166363A1 (en) * 2004-01-27 2006-07-27 Sergei Zolotukhin Modified baculovirus expression system for production of pseudotyped rAAV vector
US20070253936A1 (en) * 2006-01-31 2007-11-01 Kay Mark A Self-complementary parvoviral vectors, and methods for making and using the same
CN103764831A (zh) * 2011-03-11 2014-04-30 肌肉学研究协会 不含衣壳的aav载体、组合物以及制备载体和基因递送的方法
CN104087613A (zh) * 2014-06-30 2014-10-08 中国科学院苏州生物医学工程技术研究所 基于aav-itr的基因表达微载体及其构建方法和应用
WO2017123918A1 (en) * 2016-01-15 2017-07-20 American Gene Technologies International Inc. Methods and compositons for the activation of gamma-delta t-cells

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
LINA LI等: "Production and characterization of novel recombinant adeno-associated virus replicative-form genomes: a eukaryotic source of DNA for gene transfer" *
MICHAËL HOCQUEMILLER等: "Adeno-Associated Virus-Based Gene Therapy for CNS Diseases" *
WU Z等: "Adeno-associated virus serotypes: vector toolkit for human gene therapy" *

Also Published As

Publication number Publication date
UA129010C2 (uk) 2024-12-25
JP2020532981A (ja) 2020-11-19
EP3678710A4 (en) 2021-06-09
EP3678710A1 (en) 2020-07-15
AU2018327348A1 (en) 2020-02-20
RU2020109904A (ru) 2021-10-08
BR112020004151A2 (pt) 2020-09-08
KR20200051011A (ko) 2020-05-12
US20200283794A1 (en) 2020-09-10
NZ761178A (en) 2024-05-31
MX2020002500A (es) 2020-09-17
JP2025010253A (ja) 2025-01-20
WO2019051255A1 (en) 2019-03-14
CA3075168A1 (en) 2019-03-14
MA50100A (fr) 2020-07-15
PH12020500465A1 (en) 2021-01-25
JP2022190081A (ja) 2022-12-22
SG11202000698SA (en) 2020-03-30
IL272797A (en) 2020-04-30

Similar Documents

Publication Publication Date Title
CN111132699A (zh) 修饰的封闭端dna(cedna)
US12442015B2 (en) Closed-ended DNA vectors obtainable from cell-free synthesis and process for obtaining ceDNA vectors
JP7590963B2 (ja) 対称的な修飾型逆位末端反復配列を含む修飾型閉端dna(cedna)
CN113454232B (zh) 封闭端dna(cedna)产生中对rep蛋白活性的调节
CN111886343A (zh) 使用闭合端dna(cedna)载体控制转基因的表达
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
AU2020315444A1 (en) Synthetic production of single-stranded adeno associated viral DNA vectors
CN113874513A (zh) 非病毒dna载体及其用于表达fviii治疗剂的用途
CN115667531A (zh) 非病毒DNA载体和其用于表达戈谢(Gaucher)治疗剂的用途
CN118489008A (zh) 封闭端dna载体的可扩展且高纯度无细胞合成
RU2816963C2 (ru) МОДИФИЦИРОВАННАЯ ДНК С ЗАМКНУТЫМИ КОНЦАМИ (зкДНК), СОДЕРЖАЩАЯ СИММЕТРИЧНЫЕ МОДИФИЦИРОВАННЫЕ ИНВЕРТИРОВАННЫЕ КОНЦЕВЫЕ ПОВТОРЫ

Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination