MX2022003291A - Vectores de adn sinteticos y metodos de uso. - Google Patents

Vectores de adn sinteticos y metodos de uso.

Info

Publication number
MX2022003291A
MX2022003291A MX2022003291A MX2022003291A MX2022003291A MX 2022003291 A MX2022003291 A MX 2022003291A MX 2022003291 A MX2022003291 A MX 2022003291A MX 2022003291 A MX2022003291 A MX 2022003291A MX 2022003291 A MX2022003291 A MX 2022003291A
Authority
MX
Mexico
Prior art keywords
dna vectors
methods
dna
synthetic dna
subject
Prior art date
Application number
MX2022003291A
Other languages
English (en)
Inventor
Bruce C Schnepp
Philip R Johnson
Original Assignee
Intergalactic 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 Intergalactic Therapeutics Inc filed Critical Intergalactic Therapeutics Inc
Publication of MX2022003291A publication Critical patent/MX2022003291A/es

Links

Classifications

    • 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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • 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
    • C12N15/64—General methods for preparing the vector, for introducing it into the cell or for selecting the vector-containing host
    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific
    • 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
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Cell Biology (AREA)
  • Virology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Diabetes (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Hematology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

Se proporciona en la presente vectores de ADN aislados que comprenden un gen heterólogo, en donde el vector de ADN está desprovisto de ADN plasmídico bacteriano y/o firmas bacterianas, que pueden anular la persistencia in vivo. La invención también presenta composiciones farmacéuticas (composiciones farmacéuticas no inmunogénicas) incluido los vectores de ADN de la invención, que se pueden usar para inducción de la expresión episomática a largo plazo de un gen heterólogo en un sujeto. La invención implica métodos para tratar un sujeto al administrar los vectores de ADN de la invención, incluidos métodos para tratar trastornos asociados con un defecto en un gen objetivo.
MX2022003291A 2019-09-18 2020-09-18 Vectores de adn sinteticos y metodos de uso. MX2022003291A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962902084P 2019-09-18 2019-09-18
PCT/US2020/051507 WO2021055760A1 (en) 2019-09-18 2020-09-18 Synthetic dna vectors and methods of use

Publications (1)

Publication Number Publication Date
MX2022003291A true MX2022003291A (es) 2022-09-09

Family

ID=74884687

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2022003291A MX2022003291A (es) 2019-09-18 2020-09-18 Vectores de adn sinteticos y metodos de uso.

Country Status (12)

Country Link
US (6) US11766490B2 (es)
EP (1) EP4031168A4 (es)
JP (1) JP2022549138A (es)
KR (1) KR20220128607A (es)
CN (1) CN115066255A (es)
AU (1) AU2020351204A1 (es)
CA (1) CA3151464A1 (es)
GB (1) GB2606844A (es)
IL (1) IL291437A (es)
MX (1) MX2022003291A (es)
TW (1) TW202124722A (es)
WO (1) WO2021055760A1 (es)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2021516953A (ja) * 2018-03-15 2021-07-15 インターガラクティック セラピューティクス インコーポレイテッド 合成ｄｎａベクターおよびその使用
CN116479022A (zh) * 2018-12-12 2023-07-25 中南大学 一种重组car19-il24基因、慢病毒载体、car19-il24-t细胞及应用
JP2022549138A (ja) 2019-09-18 2022-11-24 インターガラクティック セラピューティクス インコーポレイテッド 合成ｄｎａベクターおよびその使用法
US11926842B2 (en) * 2021-02-02 2024-03-12 University Of Massachusetts Use of inverted terminal repeats (ITRS) from adeno-associated virus serotypes 8 and RH.39 in gene therapy vectors
CN118679248A (zh) * 2021-09-27 2024-09-20 阿尔德弗龙有限责任公司 环状dna载体的合成产生
WO2023064934A1 (en) * 2021-10-15 2023-04-20 Intergalactic Therapeutics, Inc. Respiratory delivery of therapeutic agents
KR20240128889A (ko) * 2021-12-20 2024-08-27 알데브론 엘엘씨 조작된 박테리아에서 유전자 치료 벡터의 생산
WO2024115669A1 (en) * 2022-11-30 2024-06-06 Evaxion Biotech A/S Process for production of synthetic circular dna

Family Cites Families (85)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US2488641A (en) * 1947-06-17 1949-11-22 Herschel V Seawright Revolving display rack for showcases
US6027722A (en) 1990-10-25 2000-02-22 Nature Technology Corporation Vectors for gene transfer
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
US6825012B2 (en) * 1995-02-23 2004-11-30 Gencell S.A. DNA molecules, preparation and use in gene therapy
FR2731014B1 (fr) 1995-02-23 1997-03-28 Rhone Poulenc Rorer Sa Molecules d'adn, preparation et utilisation en therapie genique
US5994104A (en) 1996-11-08 1999-11-30 Royal Free Hospital School Of Medicine Interleukin-12 fusion protein
US7030098B2 (en) 1999-03-12 2006-04-18 The Board Of Trustees Of The Leland Stanford Junior University DNA vaccination for treatment of autoimmune disease
RU2001135838A (ru) 1999-05-28 2004-01-20 Камбрекс Байо Сайенс Инк. (Us) Способы очистки ДНК и очищенная ДНК
EP1317560A4 (en) 2000-09-08 2005-10-05 Gen Hospital Corp SELF-ORDERING DNA VECTORS (18.03.02)
US20070196838A1 (en) 2000-12-08 2007-08-23 Invitrogen Corporation Methods and compositions for synthesis of nucleic acid molecules using multiple recognition sites
US20030082559A1 (en) 2001-01-19 2003-05-01 David Beach Methods and reagents for amplification and manipulation of vector and target nucleic acid sequences
FR2821855B1 (fr) * 2001-03-09 2004-04-02 Cayla Genes synthetiques et plasmides bacteriens depourvus de cpg
EP1572943B1 (en) 2002-08-29 2015-04-22 The Board of Trustees of The Leland S. Stanford Junior University Circular nucleic acid vectors, and methods for making and using the same
US7595303B1 (en) 2002-09-05 2009-09-29 University Of South Florida Genetic adjuvants for immunotherapy
US20050069991A1 (en) * 2003-03-25 2005-03-31 Hyman Edward D. Method for plasmid preparation by conversion of open circular plasmid to supercoiled plasmid
AT412400B (de) 2003-05-08 2005-02-25 Mayrhofer Peter Mag Dr Minicircle-herstellung
US8802643B1 (en) 2003-05-30 2014-08-12 University Of South Florida Method for the treatment of malignancies
EP1486567A1 (en) 2003-06-11 2004-12-15 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Improved adeno-associated virus (AAV) vector for gene therapy
EP1781800B1 (en) 2004-08-19 2013-06-19 Nature Technology Corp. Process for plasmid dna fermentation
AU2005314431B2 (en) 2004-12-11 2011-02-17 Cytogenix, Inc. Cell free biosynthesis of high-quality nucleic acid and uses thereof
US7622252B2 (en) 2005-06-10 2009-11-24 Baylor College Of Medicine Generation of minicircle DNA with physiological supercoiling
WO2007018744A2 (en) 2005-08-03 2007-02-15 Cytogenix, Inc. Cell-free biosynthesis of nucleic acid
US20080318283A1 (en) 2006-02-01 2008-12-25 Clague P. Hodgson Fermentation Process for Continuous Plasmid Dna Production
US9017966B2 (en) 2007-05-23 2015-04-28 Nature Technology Corporation E. coli plasmid DNA production
AU2008262478B9 (en) 2007-05-29 2014-06-19 Aldevron, L.L.C. Vectors and methods for genetic immunization
EP2262899A1 (en) 2008-04-02 2010-12-22 Fondazione Telethon Method of treating genetic disorders
US8945885B2 (en) 2008-07-03 2015-02-03 The Board Of Trustees Of The Leland Stanford Junior University Minicircle DNA vector preparations and methods of making and using the same
US8921072B2 (en) 2008-09-02 2014-12-30 General Electric Compnay Methods to generate DNA mini-circles
US9045759B2 (en) 2009-01-21 2015-06-02 James Arthur Williams DNA plasmids with improved copy number
GB0901593D0 (en) 2009-01-30 2009-03-11 Touchlight Genetics Ltd Production of closed linear DNA
US8729044B2 (en) 2009-10-16 2014-05-20 Baylor College Of Medicine Supercoiled minivectors for gene therapy applications
US9506082B2 (en) 2010-04-12 2016-11-29 Nature Technology Corporation Eukaryotic expression vectors resistant to transgene silencing
GB201013153D0 (en) 2010-08-04 2010-09-22 Touchlight Genetics Ltd Primer for production of closed linear DNA
EP2439276A1 (de) * 2010-10-05 2012-04-11 Rentschler Biotechnologie GmbH Verfahren zur semi-synthetischen Herstellung hochreiner "MiniCircle" DNA-Vektoren aus Plasmiden
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
EA026926B1 (ru) 2011-12-12 2017-05-31 Дзе Трастиз Оф Дзе Юниверсити Оф Пенсильвания Композиции, содержащие улучшенные генетические конструкции il-12, вакцины, иммунотерапевтические составы и способы их применения
US9347073B2 (en) 2012-02-10 2016-05-24 The Board Of Trustees Of The Leland Stanford Junior University Mini-intronic plasmid vectors
US20140350087A9 (en) 2012-03-22 2014-11-27 Halozyme, Inc. Oncovector Nucleic Acid Molecules and Methods of Use
EP2854866A4 (en) 2012-05-30 2015-12-23 Baylor College Medicine SUPERVOLVED MINIVECTORS AS A TOOL FOR THE REPAIR, MODIFICATION AND REPLACEMENT OF DNA
US20150322439A1 (en) 2012-11-19 2015-11-12 Nature Technology Corporation Replicative minicircle vectors with improved expression
KR20150093834A (ko) 2012-12-13 2015-08-18 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 Ｄｎａ 항체 작제물 및 그 이용 방법
US9701981B2 (en) 2013-03-15 2017-07-11 The Board Of Trustees Of The Leland Stanford Junior University Nucleosome-excluding sequences (NES) as a DNA spacer in vectors results in prolonged transgene expression in eukaryotic cells
GB201307075D0 (en) 2013-04-19 2013-05-29 Mayrhofer Peter Plasmid for minicircle production
AU2014262867B2 (en) 2013-05-10 2019-12-05 Sangamo Therapeutics, Inc. Delivery methods and compositions for nuclease-mediated genome engineering
CN104342453A (zh) 2013-08-06 2015-02-11 深圳先进技术研究院 含基因工程抗体基因表达盒的微环dna重组母质粒、含该表达盒的微环dna及应用
WO2016094639A1 (en) 2014-12-10 2016-06-16 Wisconsin Alumni Research Foundation Mini-intronic plasmid dna vaccines in combination with lag3 blockade
JP2018502604A (ja) * 2015-01-27 2018-02-01 エフ．ホフマン−ラ ロシュ アーゲーＦ． Ｈｏｆｆｍａｎｎ−Ｌａ Ｒｏｃｈｅ Ａｋｔｉｅｎｇｅｓｅｌｌｓｃｈａｆｔ 組換えＨＢＶ ｃｃｃＤＮＡ、その作製方法およびその使用
WO2016126987A1 (en) 2015-02-04 2016-08-11 Twist Bioscience Corporation Compositions and methods for synthetic gene assembly
PL3286311T3 (pl) 2015-03-26 2021-09-13 Oncosec Medical Incorporated Sposób leczenia nowotworów złośliwych
JP6929230B2 (ja) 2015-05-14 2021-09-01 セント ジュード チルドレンズ リサーチ ホスピタル インコーポレイテッド スペーサーを含む核酸分子およびその使用の方法
JP7142571B2 (ja) * 2015-09-22 2022-09-27 ユリウス－マクシミリアン－ウニヴェルシテート・ヴュルツブルク リンパ球における高レベル且つ安定な遺伝子移入のための方法
AU2016355343B2 (en) 2015-11-19 2023-12-14 Lloyd G. Mitchell Compositions and methods for correction of heritable ocular disease
KR20190031424A (ko) 2015-12-18 2019-03-26 온코섹 메디컬 인코포레이티드 이종 단백질 발현을 위한 플라스미드 작제물 및 사용 방법
EP3205356A1 (en) 2016-02-10 2017-08-16 Onkoloski Institut Ljubljana Plasmid expression vector encoding human interleukin 12 under transcriptional control of p21 promoter and without antibiotic resistance for cancer gene therapy and other uses thereof
AU2017228413A1 (en) 2016-03-03 2018-08-23 University Of Massachusetts Closed-ended linear duplex DNA for non-viral gene transfer
US10077459B2 (en) 2016-05-04 2018-09-18 General Electric Company Cell-free protein expression using rolling circle amplification product
WO2018111914A1 (en) 2016-12-14 2018-06-21 Synthetic Genomics, Inc. Methods for assembling dna molecules
AU2018228154A1 (en) 2017-02-28 2019-09-26 OriCiro Genomics, Inc. Method for replicating or amplifying circular DNA
US20180305701A1 (en) * 2017-03-14 2018-10-25 Baylor College Of Medicine Targeting minivectors to specific tissue using shape
JP7257377B2 (ja) 2017-03-21 2023-04-13 ミヒャラキス，スティリアノス Ｃｎｇｂ１連鎖性網膜色素変性症の治療のための遺伝子療法
AU2018248304C1 (en) 2017-04-05 2023-02-16 University Of Massachusetts Minigene therapy
CN108728465A (zh) 2017-04-14 2018-11-02 深圳新诺微环生物科技有限公司 一种表达靶细胞-效应细胞桥接器的微环dna载体及其制备方法和应用
WO2018229696A1 (en) 2017-06-13 2018-12-20 Oncosec Medical Incorporated Multigene construct for immune-modulatory protein expression and methods of use
EP3638776A1 (en) * 2017-06-14 2020-04-22 Technische Universität Dresden Methods and means for genetic alteration of genomes utilizing designer dna recombining enzymes
KR20200051011A (ko) * 2017-09-08 2020-05-12 제너레이션 바이오 컴퍼니 변형된 폐쇄-종결된 dna(cedna)
ES2821655T5 (es) 2017-09-19 2024-08-06 Deutsches Krebsforsch Vectores de ADN no integrantes para la modificación genética de células
WO2019057774A1 (en) 2017-09-19 2019-03-28 Deutsches Krebsforschungszentrum NON-INTEGRATING DNA VECTORS FOR THE GENETIC MODIFICATION OF CELLS
US20200048716A1 (en) 2017-11-03 2020-02-13 Twister Biotech, Inc Using minivectors to treat ovarian cancer
JP2021516953A (ja) 2018-03-15 2021-07-15 インターガラクティック セラピューティクス インコーポレイテッド 合成ｄｎａベクターおよびその使用
WO2019183248A1 (en) 2018-03-21 2019-09-26 Nature Technology Corporation Viral and non-viral nanoplasmid vectors with improved production
US20210346306A1 (en) 2018-05-23 2021-11-11 Modernatx, Inc. Delivery of dna
EP3863677A4 (en) 2018-10-10 2022-12-28 The Johns Hopkins University POLY (Beta-AMINO ESTER) NANOPARTICLES FOR NON-VIRAL DELIVERY OF PLASMID DNA FOR GENE EDITING AND RETINA GENE THERAPY
WO2020101828A2 (en) 2018-10-12 2020-05-22 Children's Hospital Medical Center Modular expression systems for gene expression and methods of using same
US20210383891A1 (en) 2018-10-19 2021-12-09 New England Biolabs, Inc. Improved Ordered Assembly of Multiple DNA Fragments
CN109402168A (zh) 2018-10-30 2019-03-01 深圳新诺微环生物科技有限公司 微环dna表达连接her2阳性细胞与效应细胞的桥接分子及其应用
CA3120564A1 (en) 2018-11-27 2020-06-04 Oncosec Medical Incorporated Plasmid constructs for treating cancer and methods of use
CN111378677A (zh) 2018-12-27 2020-07-07 南京中科游子生物技术研究院有限公司 一种dna组装的方法及其应用
WO2020150293A1 (en) 2019-01-15 2020-07-23 University Of Maryland, Baltimore County A method for assembling circular and linear dna molecules in an ordered manner
US11959069B2 (en) 2019-02-28 2024-04-16 Proteonic Biotechnology Ip B.V. Self-immolative plasmid backbone
EP3955973A4 (en) 2019-04-19 2023-01-18 University of Massachusetts GENE THERAPIES FOR STARGARDT'S DISEASE (ABCA4)
US20220202959A1 (en) 2019-04-19 2022-06-30 University Of Massachusetts Gene therapies for usher syndrome (ush2a)
US20220175967A1 (en) 2019-04-19 2022-06-09 University Of Massachusetts Gene therapies for usher syndrome (ush1b)
CN110684798A (zh) 2019-09-03 2020-01-14 深圳新诺微环生物科技有限公司 肌肉靶向的微环dna基因治疗
CN110564751A (zh) 2019-09-03 2019-12-13 深圳新诺微环生物科技有限公司 微环dna疫苗设计及应用
JP2022549138A (ja) 2019-09-18 2022-11-24 インターガラクティック セラピューティクス インコーポレイテッド 合成ｄｎａベクターおよびその使用法

Also Published As

Publication number Publication date
EP4031168A1 (en) 2022-07-27
US20210330817A1 (en) 2021-10-28
US11684680B2 (en) 2023-06-27
GB202204355D0 (en) 2022-05-11
CA3151464A1 (en) 2021-03-25
CN115066255A (zh) 2022-09-16
EP4031168A4 (en) 2023-10-11
KR20220128607A (ko) 2022-09-21
TW202124722A (zh) 2021-07-01
US11324839B2 (en) 2022-05-10
US20220168451A1 (en) 2022-06-02
US20210322579A1 (en) 2021-10-21
AU2020351204A1 (en) 2022-04-21
US20230089543A1 (en) 2023-03-23
US11602569B2 (en) 2023-03-14
GB2606844A (en) 2022-11-23
JP2022549138A (ja) 2022-11-24
IL291437A (en) 2022-05-01
US11766490B2 (en) 2023-09-26
US20230270888A1 (en) 2023-08-31
WO2021055760A1 (en) 2021-03-25
US20210322576A1 (en) 2021-10-21

Similar Documents

Publication Publication Date Title
MX2022003291A (es) Vectores de adn sinteticos y metodos de uso.
MX2020009579A (es) Vectores de adn sintetico y metodos de uso.
PH12020500309A1 (en) Rnai agents and compositions for inhibiting expression of angiopoietin-like 3 (angptl3), and methods of use
MX2020004647A (es) Métodos de rescate de codones de detención por medio de reasignación genética con arn de transferencia editado anti-codones (ace-trna).
SA517390590B1 (ar) Ix عامل معدل، وتركيبات وطرق واستخدامات لنقل الجين إلى الخلايا، والأعضاء والأنسجة
PH12021550843A1 (en) Nucleic acid constructs and methods of use
MX2017014908A (es) Proteinas de union triespecificas y metodos de uso.
WO2016109546A3 (en) Methods and compositions for prognosis and treatment of cancers
EP4310500A3 (en) Methods and compositons for the activation of gamma-delta t-cells
EP2574170A4 (en) IMMUNOSTIMULATORY AND VACCINE COMPOSITIONS
WO2015149944A3 (en) Polymeric carrier cargo complex for use as an immunostimulating agent or as an adjuvant
WO2019094791A3 (en) Targeted crispr delivery platforms
MX2021006730A (es) Citomegalovirus humano compuesto por antigenos exogenos.
MX2020009736A (es) Composiciones que comprenden cepas bacterianas.
MX2019006426A (es) Reguladores de tau, y composiciones y metodos para su administracion.
WO2019226939A8 (en) Immune checkpoint inhibitor co-expression vectors
MX2022006602A (es) Vacunas de herpesvirus de pavo recombinante y uso de las mismas.
MX2018016051A (es) Composiciones y métodos para diagnosticar tipos de cáncer de pulmón usando perfiles de expresión génica.
MX2021006253A (es) Terapias génicas para enfermedad neurodegenerativa.
MX2021009696A (es) Vectores de genoterapia para el tratamiento de la enfermedad de danon.
IL276424A (en) A special promoter for oligodendrocytes, a special mirna for the plp1 gene, a vector including the above-mentioned promoter and/or mirna, and a pharmaceutical composition including the above-mentioned vector
EP4079750A3 (en) Adenoviruses and methods for using adenoviruses
MX2021002041A (es) Terapia génica para el tratamiento de galactosemia.
WO2018183692A8 (en) VECTORS AND COMPOSITIONS FOR THE TREATMENT OF HEMOGLOBINOPATHIES
WO2020079489A3 (en) Treatment of neuropathy with igf-1-encoding dna constructs and hgf-encoding dna constructs