IN2014CN04734A - - Google Patents

Info

Publication number
IN2014CN04734A
IN2014CN04734A IN4734CHN2014A IN2014CN04734A IN 2014CN04734 A IN2014CN04734 A IN 2014CN04734A IN 4734CHN2014 A IN4734CHN2014 A IN 4734CHN2014A IN 2014CN04734 A IN2014CN04734 A IN 2014CN04734A
Authority
IN
India
Prior art keywords
cells
vectors
compositions
biologically active
provides
Prior art date
Application number
Inventor
Kevin Polach
Jason Fewell
Khursheed Anwer
Lestlie S Wilkinson
Original Assignee
Egen 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 Egen Inc filed Critical Egen Inc
Publication of IN2014CN04734A publication Critical patent/IN2014CN04734A/en

Links

Classifications

    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/02—Fusion polypeptide containing a localisation/targetting motif containing a signal sequence
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/10—Fusion polypeptide containing a localisation/targetting motif containing a tag for extracellular membrane crossing, e.g. TAT or VP22
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/85—Fusion polypeptide containing an RNA binding domain
    • 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
    • C12N2330/00—Production
    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
    • C12N2330/51—Specially adapted 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE

Abstract

Novel compounds compositions and methods for the delivery of biologically active RNA molecules to cells. Specifically the invention provides novel nucleic acid molecules polypeptides and RNA protein complexes useful for the delivery of biologically active RNAs to cells and polynucleotides encoding the same. The invention also provides vectors for expressing said polynucleotides. In addition the invention provides cells and compositions comprising the novel compounds and vectors which can be used as transfection reagents. The invention further provides methods for producing said compounds vectors cells and compositions. Additionally vectors and methods for delivering biologically active RNA molecules to cells and/or tissues are provided. The novel compounds vectors cells and compositions are useful for example in delivering biologically active RNA molecules to cells to modulate target gene expression in the diagnosis prevention amelioration and/or treatment of diseases discorders or conditions in a subject or organism.
IN4734CHN2014 2011-12-23 2012-12-24 IN2014CN04734A (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201161579815P 2011-12-23 2011-12-23
PCT/US2012/071576 WO2013096958A1 (en) 2011-12-23 2012-12-24 Compositions and methods for the delivery of biologically active rnas

Publications (1)

Publication Number Publication Date
IN2014CN04734A true IN2014CN04734A (en) 2015-09-18

Family

ID=48654938

Family Applications (1)

Application Number Title Priority Date Filing Date
IN4734CHN2014 IN2014CN04734A (en) 2011-12-23 2012-12-24

Country Status (10)

Country Link
US (1) US20130164845A1 (en)
EP (1) EP2794879A4 (en)
JP (1) JP2015507474A (en)
KR (1) KR20140123054A (en)
CN (1) CN104169419A (en)
AU (1) AU2012358181A1 (en)
CA (1) CA2860228A1 (en)
HK (1) HK1203548A1 (en)
IN (1) IN2014CN04734A (en)
WO (1) WO2013096958A1 (en)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11639371B2 (en) * 2016-02-17 2023-05-02 The Chinese University Of Hong Kong Peptidylic inhibitors of nucleolin (NCL) targeting CAG-repeat RNA toxicity and methods for reducing polyQ-mediated toxicity in polyQ diseases
US11352625B2 (en) 2017-01-12 2022-06-07 Duke University Compositions and methods for disrupting the molecular mechanisms associated with mitochondrial dysfunction and neurodegenerative disease
AU2018291137A1 (en) * 2017-06-28 2020-01-23 University Of South Florida Modified UBE3A gene for a gene therapy approach for Angelman syndrome
MX2021009727A (en) * 2019-02-15 2021-11-12 Atreca Inc Antibodies that bind tumor tissue, and their diagnostic and therapeutic uses.
CN114395576B (en) * 2020-08-31 2023-11-14 南京工业大学 Method for improving protein expression efficiency in clostridium
WO2022241480A1 (en) * 2021-05-13 2022-11-17 Technion Research & Development Foundation Limited Methods and compositions for treatment of viral infection
WO2023182870A1 (en) * 2022-03-24 2023-09-28 (주)셀레브레인 Novel recombinant vector and use thereof
EP4303305A1 (en) * 2022-07-07 2024-01-10 Kutzner, Christoph Fusion polypeptides for cell penetration and cell targeting

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5585263A (en) * 1994-05-20 1996-12-17 University Of Alabama At Birmingham Research Foundation Purified retroviral constitutive transport enhancer and its use to facilitate mRNA transport, and to produce recombinant, attenuated HIV
WO2004011624A2 (en) * 2002-07-31 2004-02-05 Nucleonics, Inc. Double stranded rna structures and constructs, and methods for generating and using the same
EP1658304A4 (en) * 2002-09-28 2009-01-14 Massachusetts Inst Technology Influenza therapeutic
WO2009002462A1 (en) * 2007-06-22 2008-12-31 The Board Of Trustees Of The Leland Stanford Junior University Pre-mirna loop-modulated target regulation
KR20110128345A (en) * 2009-03-13 2011-11-29 에젠, 인코포레이티드 Compositions and methods for the delivery of biologically active rnas
JP6137834B2 (en) * 2010-02-24 2017-05-31 アローヘッド ファーマシューティカルズ インコーポレイテッド Composition for targeted delivery of siRNA

Also Published As

Publication number Publication date
CN104169419A (en) 2014-11-26
EP2794879A1 (en) 2014-10-29
JP2015507474A (en) 2015-03-12
KR20140123054A (en) 2014-10-21
EP2794879A4 (en) 2015-10-14
AU2012358181A1 (en) 2014-07-10
US20130164845A1 (en) 2013-06-27
WO2013096958A1 (en) 2013-06-27
CA2860228A1 (en) 2013-06-27
HK1203548A1 (en) 2015-10-30
AU2012358181A8 (en) 2015-06-18
WO2013096958A8 (en) 2014-07-24

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