HK1203548A1 - Compositions and methods for the delivery of biologically active rnas - Google Patents

Compositions and methods for the delivery of biologically active rnas Download PDF

Info

Publication number
HK1203548A1
HK1203548A1 HK15103893.5A HK15103893A HK1203548A1 HK 1203548 A1 HK1203548 A1 HK 1203548A1 HK 15103893 A HK15103893 A HK 15103893A HK 1203548 A1 HK1203548 A1 HK 1203548A1
Authority
HK
Hong Kong
Prior art keywords
rna
sequence
cell
biologically active
expression vector
Prior art date
Application number
HK15103893.5A
Other languages
English (en)
Chinese (zh)
Inventor
Kevin Polach
Jason Fewell
Khursheed Anwer
Leslie S. WILKINSON
Original Assignee
Clsn实验室股份有限公司
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 Clsn实验室股份有限公司 filed Critical Clsn实验室股份有限公司
Publication of HK1203548A1 publication Critical patent/HK1203548A1/xx

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

Landscapes

  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
HK15103893.5A 2011-12-23 2012-12-24 Compositions and methods for the delivery of biologically active rnas HK1203548A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201161579815P 2011-12-23 2011-12-23
US61/579,815 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
HK1203548A1 true HK1203548A1 (en) 2015-10-30

Family

ID=48654938

Family Applications (1)

Application Number Title Priority Date Filing Date
HK15103893.5A HK1203548A1 (en) 2011-12-23 2012-12-24 Compositions and methods for the delivery of biologically active rnas

Country Status (10)

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

Families Citing this family (9)

* 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
BR112019027692A2 (pt) * 2017-06-28 2020-11-24 University Of South Florida gene ube3a modificado para uma abordagem terapêutica genética para a síndrome de angelman
WO2020144295A1 (en) * 2019-01-10 2020-07-16 Biontech Rna Pharmaceuticals Gmbh Localized administration of rna molecules for therapy
KR102592844B1 (ko) * 2019-02-15 2023-10-20 아트레카, 인크. 종양 조직에 결합하는 항체, 및 그의 진단 및 치료 용도
CN114395576B (zh) * 2020-08-31 2023-11-14 南京工业大学 一种提高梭菌中蛋白表达效率的方法
CA3218711A1 (en) * 2021-05-13 2022-11-17 Roee Amit Methods and compositions for treatment of viral infection
US20250197888A1 (en) * 2022-03-24 2025-06-19 Cell & Brain Co., Ltd. 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
EP1540004A4 (en) * 2002-07-31 2007-10-03 Nucleonics Inc DOUBLE-STRING RNA STRUCTURES AND CONSTRUCTS AND METHOD FOR THE PRODUCTION AND USE THEREOF
US20050008617A1 (en) * 2002-09-28 2005-01-13 Massachusetts Institute Of Technology Compositions and methods for delivery of short interfering RNA and short hairpin RNA
US20090004668A1 (en) * 2007-06-22 2009-01-01 The Board Of Trustees Of The Leland Stanford Junior University Pre-miRNA loop-modulated target regulation
JP2012520085A (ja) * 2009-03-13 2012-09-06 エーゲン、インコーポレイテッド 生物活性ｒｎａの送達のための組成物及び方法
SI2539451T1 (sl) * 2010-02-24 2016-04-29 Arrowhead Research Corporation Sestavki za ciljano dostavo sirna

Also Published As

Publication number Publication date
CN104169419A (zh) 2014-11-26
JP2015507474A (ja) 2015-03-12
US20130164845A1 (en) 2013-06-27
AU2012358181A8 (en) 2015-06-18
CA2860228A1 (en) 2013-06-27
KR20140123054A (ko) 2014-10-21
EP2794879A4 (en) 2015-10-14
WO2013096958A8 (en) 2014-07-24
AU2012358181A1 (en) 2014-07-10
EP2794879A1 (en) 2014-10-29
WO2013096958A1 (en) 2013-06-27
IN2014CN04734A (https=) 2015-09-18

Similar Documents

Publication Publication Date Title
US8569065B2 (en) Compositions and methods for the delivery of biologically active RNAs
JP7820434B2 (ja) アルブミン遺伝子座からの導入遺伝子発現のための組成物及び方法
HK1203548A1 (en) Compositions and methods for the delivery of biologically active rnas
JP2021500863A (ja) ゲノム編集用のポリヌクレオチド、組成物および方法
EP3823995A2 (en) Programmable designer therapeutic fusogenic secreted gectosome vesicles for macromolecule delivery and genome modification
US20200172913A1 (en) Peptides and nanoparticles for intracellular delivery of virus
EP4479542A1 (en) Cell-type specific membrane fusion proteins
US20250073350A1 (en) Compositions and methods for delivering cargo to a target cell
US20250339554A1 (en) Compositions and methods for delivering cargo to a target cell
AU2023299294A1 (en) Engineered t cells
WO2024006988A2 (en) Engineered delivery vesicles and uses thereof
CN118632622A (zh) 突变型肌纤蛋白疾病模型及其用途
US20250171806A1 (en) Engineered viral vectors with enhanced packaging capacity and methods of using the same
US20250332287A1 (en) Identification of tissue-specific extragenic safe harbors for gene therapy approaches
KR102957630B1 (ko) 핵산 구조체 및 사용 방법
JP2024526878A (ja) 調節されたウイルス送達システムおよびその使用
JP2025521154A (ja) Ｃ９ｏｒｆ７２反復伸長疾患のためのｃｒｉｓｐｒ干渉療法
CN121693566A (zh) 使用bGH-SV40L串联PolyA在单向基因插入期间增强转基因表达
KR20210102882A (ko) 핵산 구조체 및 사용 방법