AR126845A1 - Producción de adn de extremo cerrado con secuencias repetidas de terminal invertida - Google Patents

Producción de adn de extremo cerrado con secuencias repetidas de terminal invertida

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Publication number
AR126845A1
AR126845A1 ARP220102259A ARP220102259A AR126845A1 AR 126845 A1 AR126845 A1 AR 126845A1 AR P220102259 A ARP220102259 A AR P220102259A AR P220102259 A ARP220102259 A AR P220102259A AR 126845 A1 AR126845 A1 AR 126845A1
Authority
AR
Argentina
Prior art keywords
itr
inverted terminal
closed
production
nucleic acid
Prior art date
Application number
ARP220102259A
Other languages
English (en)
Inventor
Ajay Maghodia
Christian Mueller
Tongyao Liu
Original Assignee
Bioverativ 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 Bioverativ Therapeutics Inc filed Critical Bioverativ Therapeutics Inc
Publication of AR126845A1 publication Critical patent/AR126845A1/es

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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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/745—Blood coagulation or fibrinolysis factors
    • C07K14/755—Factors VIII, e.g. factor VIII C (AHF), factor VIII Ag (VWF)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
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    • C12N15/09—Recombinant DNA-technology
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    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N2710/14111—Nucleopolyhedrovirus, e.g. autographa californica nucleopolyhedrovirus
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    • C12N2710/00011—Details
    • C12N2710/14011—Baculoviridae
    • C12N2710/14111—Nucleopolyhedrovirus, e.g. autographa californica nucleopolyhedrovirus
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    • C12N2750/14011—Parvoviridae
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14311—Parvovirus, e.g. minute virus of mice
    • C12N2750/14341—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14343—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14311—Parvovirus, e.g. minute virus of mice
    • C12N2750/14371—Demonstrated in vivo effect
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    • C12N2800/00—Nucleic acids vectors
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
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    • C12N2830/00—Vector systems having a special element relevant for transcription
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    • 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

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  • Health & Medical Sciences (AREA)
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  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
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  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
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  • General Engineering & Computer Science (AREA)
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  • Microbiology (AREA)
  • Hematology (AREA)
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  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Virology (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Diabetes (AREA)
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Abstract

La presente divulgación proporciona moléculas de ácido nucleico que comprenden una primera repetición terminal invertida (ITR), una segunda ITR y un casete genético que codifica una secuencia diana. En algunas realizaciones, la primera ITR y/o la segunda ITR es una ITR de bocavirus humano. También se divulgan métodos para utilizar las moléculas de ácido nucleico en aplicaciones de terapia génica. Reivindicación 1: Una molécula de ácido nucleico que comprende una primera repetición terminal invertida (ITR) y una segunda ITR que flanquean un casete genético que comprende una secuencia de polinucleótidos heteróloga, en donde: la primera ITR comprende una secuencia de polinucleótidos al menos aproximadamente un 75% idéntica a la SEQ ID Nº 1, y la segunda ITR comprende una secuencia de polinucleótidos al menos aproximadamente un 75% idéntica a la SEQ ID Nº 2.
ARP220102259A 2021-08-23 2022-08-22 Producción de adn de extremo cerrado con secuencias repetidas de terminal invertida AR126845A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US202163236215P 2021-08-23 2021-08-23

Publications (1)

Publication Number Publication Date
AR126845A1 true AR126845A1 (es) 2023-11-22

Family

ID=83849025

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP220102259A AR126845A1 (es) 2021-08-23 2022-08-22 Producción de adn de extremo cerrado con secuencias repetidas de terminal invertida

Country Status (10)

Country Link
US (1) US20230091932A1 (es)
KR (1) KR20240049825A (es)
CN (1) CN117881696A (es)
AR (1) AR126845A1 (es)
AU (1) AU2022334714A1 (es)
CA (1) CA3229345A1 (es)
CO (1) CO2024001242A2 (es)
IL (1) IL310963A (es)
TW (1) TW202328174A (es)
WO (1) WO2023028455A1 (es)

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4704362A (en) 1977-11-08 1987-11-03 Genentech, Inc. Recombinant cloning vehicle microbial polypeptide expression
EP0832981A1 (en) 1987-02-17 1998-04-01 Pharming B.V. DNA sequences to target proteins to the mammary gland for efficient secretion
US5633076A (en) 1989-12-01 1997-05-27 Pharming Bv Method of producing a transgenic bovine or transgenic bovine embryo
MX9204374A (es) 1991-07-25 1993-03-01 Idec Pharma Corp Anticuerpo recombinante y metodo para su produccion.
US5736137A (en) 1992-11-13 1998-04-07 Idec Pharmaceuticals Corporation Therapeutic application of chimeric and radiolabeled antibodies to human B lymphocyte restricted differentiation antigen for treatment of B cell lymphoma
ATE236987T1 (de) 1992-11-13 2003-04-15 Idec Pharma Corp Konsensus-kozak-sequenzen zur säugetier- exprimierung
US5827690A (en) 1993-12-20 1998-10-27 Genzyme Transgenics Corporatiion Transgenic production of antibodies in milk
GB9524973D0 (en) 1995-12-06 1996-02-07 Lynxvale Ltd Viral vectors
EP0981637B1 (en) 1997-03-14 2005-05-25 Biogen Idec Inc. Method for integrating genes at specific sites in mammalian cells via homologous recombination and vectors for accomplishing the same
EP2983707B1 (en) * 2013-04-08 2019-06-12 University of Iowa Research Foundation Chimeric adeno-associated virus/ bocavirus parvovirus vector
TWI707951B (zh) 2015-04-08 2020-10-21 美商健臻公司 過大腺相關載體之製造
CA3077380A1 (en) * 2017-09-27 2019-04-04 Sigilon Therapeutics, Inc. Methods, compositions, and implantable elements comprising active cells
EP3736286A1 (en) * 2019-05-09 2020-11-11 Biotest AG Single chain factor viii molecule

Also Published As

Publication number Publication date
WO2023028455A1 (en) 2023-03-02
CA3229345A1 (en) 2023-03-02
IL310963A (en) 2024-04-01
KR20240049825A (ko) 2024-04-17
CN117881696A (zh) 2024-04-12
AU2022334714A1 (en) 2024-04-04
TW202328174A (zh) 2023-07-16
CO2024001242A2 (es) 2024-04-29
US20230091932A1 (en) 2023-03-23

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