PE20180772A1 - Vectores retrovirales que contienen un promotor de ubiquitina c humana de orientacion inversa - Google Patents

Vectores retrovirales que contienen un promotor de ubiquitina c humana de orientacion inversa

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Publication number
PE20180772A1
PE20180772A1 PE2018000004A PE2018000004A PE20180772A1 PE 20180772 A1 PE20180772 A1 PE 20180772A1 PE 2018000004 A PE2018000004 A PE 2018000004A PE 2018000004 A PE2018000004 A PE 2018000004A PE 20180772 A1 PE20180772 A1 PE 20180772A1
Authority
PE
Peru
Prior art keywords
promoter
human ubiquitin
vectors containing
retroviral vectors
reverse oriented
Prior art date
Application number
PE2018000004A
Other languages
English (en)
Inventor
Donald B Kohn
Aaron Ross Cooper
Original Assignee
Univ California
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 Univ California filed Critical Univ California
Publication of PE20180772A1 publication Critical patent/PE20180772A1/es

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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/177—Receptors; Cell surface antigens; Cell surface determinants
    • A61K38/1793—Receptors; Cell surface antigens; Cell surface determinants for cytokines; for lymphokines; for interferons
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    • A61K38/46—Hydrolases (3)
    • A61K38/50—Hydrolases (3) acting on carbon-nitrogen bonds, other than peptide bonds (3.5), e.g. asparaginase
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • 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
    • A61K48/0016—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 wherein the nucleic acid is delivered as a 'naked' nucleic acid, i.e. not combined with an entity such as a cationic lipid
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    • C12N2810/00—Vectors comprising a targeting moiety
    • C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein
    • C12N2810/80—Vectors comprising as targeting moiety peptide derived from defined protein from vertebrates
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    • C12N2810/00—Vectors comprising a targeting moiety
    • C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein
    • C12N2810/80—Vectors comprising as targeting moiety peptide derived from defined protein from vertebrates
    • C12N2810/85—Vectors comprising as targeting moiety peptide derived from defined protein from vertebrates mammalian
    • C12N2810/854—Vectors comprising as targeting moiety peptide derived from defined protein from vertebrates mammalian from hormones
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/80—Vector systems having a special element relevant for transcription from vertebrates
    • C12N2830/85—Vector systems having a special element relevant for transcription from vertebrates mammalian

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Abstract

Se refiere a un vector retroviral recombinante donde el vector comprende un promotor de ubiquitina C (UBC) humana enlazado de forma operativa a un transgen donde el promotor y el transgen se encuentran en una orientacion inversa, de forma tal que la direccion de la transcripcion del transgen del promotor se orienta hacia una repeticion terminal larga (LTR) 5' del vector
PE2018000004A 2015-07-01 2016-06-22 Vectores retrovirales que contienen un promotor de ubiquitina c humana de orientacion inversa PE20180772A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201562187678P 2015-07-01 2015-07-01

Publications (1)

Publication Number Publication Date
PE20180772A1 true PE20180772A1 (es) 2018-05-07

Family

ID=57608918

Family Applications (1)

Application Number Title Priority Date Filing Date
PE2018000004A PE20180772A1 (es) 2015-07-01 2016-06-22 Vectores retrovirales que contienen un promotor de ubiquitina c humana de orientacion inversa

Country Status (12)

Country Link
US (1) US20180185415A1 (es)
EP (1) EP3317416A4 (es)
JP (1) JP2018519827A (es)
KR (1) KR20180015751A (es)
CN (1) CN107949640A (es)
AU (1) AU2016285473A1 (es)
BR (1) BR112017028607A2 (es)
CA (1) CA2990963A1 (es)
CL (1) CL2017003476A1 (es)
MX (1) MX2017017062A (es)
PE (1) PE20180772A1 (es)
WO (1) WO2017003792A1 (es)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
WO2018170239A1 (en) * 2017-03-15 2018-09-20 The Regents Of The University Of California Methods of treating lysosomal disorders
CN109913499A (zh) * 2017-12-13 2019-06-21 上海吉凯基因化学技术有限公司 一种适用于长链非编码rna构建及表达的整合型慢病毒载体系统及其应用
EP3788070A4 (en) * 2018-04-30 2022-03-09 The Children's Hospital Of Philadelphia COMPOSITIONS AND METHODS OF HEMOGLOBIN PRODUCTION
CN108707627A (zh) * 2018-05-31 2018-10-26 深圳市免疫基因治疗研究院 一种mld慢病毒载体、慢病毒及其制备方法和应用
CN108728494A (zh) * 2018-05-31 2018-11-02 深圳市免疫基因治疗研究院 一种x-scid慢病毒载体、慢病毒及其制备方法和应用
CN108728495A (zh) * 2018-05-31 2018-11-02 深圳市免疫基因治疗研究院 一种Sanfilippo A综合症慢病毒载体、慢病毒及其制备方法和应用
US20220136007A1 (en) * 2019-02-14 2022-05-05 The Regents Of The University Of California Optimized lentiviral vector compromising minimal enhancer elements for stem cell gene therapy of hemoglobinopathies
CN112195195A (zh) * 2020-12-07 2021-01-08 和元生物技术（上海）股份有限公司 慢病毒包装载体与包装方法

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1999035280A1 (en) * 1998-01-06 1999-07-15 Bavarian Nordic Research Institute A/S Reconstituting retroviral vector (recon vector) for targeted gene expression
IL161229A0 (en) * 2001-10-02 2004-09-27 Inst Clayton De La Rech Methods and compositions relating to restricted expression lentiviral vectors and their applications.
US20090217399A1 (en) * 2006-03-17 2009-08-27 Massachusetts Institute Of Technology Lentiviral Vectors That Provide Improved Expression And Reduced Variegation After Transgenesis
EP2895611A4 (en) * 2012-09-14 2016-04-27 Univ California LENTIVIRAL VECTORS FOR STEM CELL GENE THERAPY OF SICKEN LEMENIA

Also Published As

Publication number Publication date
CA2990963A1 (en) 2017-01-05
EP3317416A4 (en) 2018-12-26
CL2017003476A1 (es) 2018-08-31
KR20180015751A (ko) 2018-02-13
AU2016285473A1 (en) 2018-01-18
CN107949640A (zh) 2018-04-20
EP3317416A1 (en) 2018-05-09
BR112017028607A2 (pt) 2018-09-04
MX2017017062A (es) 2018-09-05
US20180185415A1 (en) 2018-07-05
JP2018519827A (ja) 2018-07-26
WO2017003792A1 (en) 2017-01-05

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