BR112018070249A2 - sistema promotor de u6 modificada para expressão específica de tecido - Google Patents

sistema promotor de u6 modificada para expressão específica de tecido

Info

Publication number
BR112018070249A2
BR112018070249A2 BR112018070249A BR112018070249A BR112018070249A2 BR 112018070249 A2 BR112018070249 A2 BR 112018070249A2 BR 112018070249 A BR112018070249 A BR 112018070249A BR 112018070249 A BR112018070249 A BR 112018070249A BR 112018070249 A2 BR112018070249 A2 BR 112018070249A2
Authority
BR
Brazil
Prior art keywords
tissue specific
promoter system
specific expression
expression modified
mirna
Prior art date
Application number
BR112018070249A
Other languages
English (en)
Portuguese (pt)
Inventor
Quenton Harper Scott
Original Assignee
Research Institute At Nationwide Children's Hospital
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 Research Institute At Nationwide Children's Hospital filed Critical Research Institute At Nationwide Children's Hospital
Publication of BR112018070249A2 publication Critical patent/BR112018070249A2/pt

Links

Classifications

    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01HNEW PLANTS OR NON-TRANSGENIC PROCESSES FOR OBTAINING THEM; PLANT REPRODUCTION BY TISSUE CULTURE TECHNIQUES
    • A01H5/00Angiosperms, i.e. flowering plants, characterised by their plant parts; Angiosperms characterised otherwise than by their botanic taxonomy
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular system
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12QMEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141MicroRNAs, miRNAs
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/32Special delivery means, e.g. tissue-specific
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2330/00Production
    • C12N2330/50Biochemical production, i.e. in a transformed host cell
    • C12N2330/51Specially adapted vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2710/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011Details
    • C12N2710/10011Adenoviridae
    • C12N2710/10041Use of virus, viral particle or viral elements as a vector
    • C12N2710/10043Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12QMEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/70Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving virus or bacteriophage
    • YGENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02ATECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Plant Pathology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Virology (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Analytical Chemistry (AREA)
  • Dermatology (AREA)
  • Physiology (AREA)
  • Botany (AREA)
  • Developmental Biology & Embryology (AREA)
  • Environmental Sciences (AREA)
BR112018070249A 2016-04-02 2017-03-31 sistema promotor de u6 modificada para expressão específica de tecido BR112018070249A2 (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201662317524P 2016-04-02 2016-04-02
PCT/US2017/025614 WO2017173411A1 (en) 2016-04-02 2017-03-31 Modified u6 promoter system for tissue specific expression

Publications (1)

Publication Number Publication Date
BR112018070249A2 true BR112018070249A2 (pt) 2019-01-29

Family

ID=59965277

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112018070249A BR112018070249A2 (pt) 2016-04-02 2017-03-31 sistema promotor de u6 modificada para expressão específica de tecido

Country Status (12)

Country Link
US (3) US11345913B2 (https=)
EP (2) EP4335502A3 (https=)
JP (2) JP7253379B2 (https=)
KR (1) KR102453187B1 (https=)
CN (1) CN109714954A (https=)
AU (1) AU2017240236C1 (https=)
BR (1) BR112018070249A2 (https=)
CA (1) CA3019832C (https=)
IL (1) IL262056B2 (https=)
MA (1) MA44528A (https=)
RU (1) RU2768289C2 (https=)
WO (1) WO2017173411A1 (https=)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2018345772B2 (en) * 2017-10-02 2025-04-17 Research Institute At Nationwide Children's Hospital MiRNA detargeting system for tissue specific interference
CN108823209B (zh) * 2018-07-18 2021-04-09 华中农业大学 骨骼肌特异性启动子及应用
SG11202100928QA (en) 2018-08-02 2021-02-25 Dyne Therapeutics Inc Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
US12018087B2 (en) 2018-08-02 2024-06-25 Dyne Therapeutics, Inc. Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject
JP2021534755A (ja) 2018-08-22 2021-12-16 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル 筋強直性ジストロフィープロテインキナーゼの発現を抑制および/またはdmpk遺伝子の3’非翻訳領域におけるトリヌクレオチドリピート伸長に干渉するための組換えウイルス産物および方法
EP3906308A1 (en) * 2018-12-31 2021-11-10 Research Institute at Nationwide Children's Hospital Dux4 rna silencing using rna targeting crispr-cas13b
KR20230023612A (ko) * 2020-04-02 2023-02-17 마이레큘, 인크. 조작된 올리고뉴클레오티드를 사용한 표적화된 억제
CA3189065A1 (en) 2020-09-11 2022-03-17 Arrowhead Pharmaceuticals, Inc. Rnai agents for inhibiting expression of dux4, compositions thereof, and methods of use
US12275941B2 (en) * 2021-04-15 2025-04-15 Research Institute At Nationwide Children's Hospital Products and methods for inhibition of expression of dynamin-1 variants
EP4504952A4 (en) * 2022-04-07 2026-04-08 Kate Therapeutics Inc COMPOSITIONS AND METHODS FOR MUSCULAR DISORDERS
WO2026011009A1 (en) 2024-07-02 2026-01-08 Kate Therapeutics, Inc. Compositions and methods for muscle disorders
WO2026030242A1 (en) 2024-07-29 2026-02-05 Kate Therapeutics, Inc. Adeno-associated virus compositions for the treatment of facioscapulohumeral muscular dystrophy

Family Cites Families (33)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
WO1995013365A1 (en) 1993-11-09 1995-05-18 Targeted Genetics Corporation Generation of high titers of recombinant aav vectors
PT728214E (pt) 1993-11-09 2004-11-30 Ohio Med College Linhas celulares estaveis capazes de expressar o gene de replicacao do virus adeno-associado
US5658785A (en) 1994-06-06 1997-08-19 Children's Hospital, Inc. Adeno-associated virus materials and methods
US5856152A (en) 1994-10-28 1999-01-05 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV vector and methods of use therefor
CA2207927A1 (en) 1994-12-06 1996-06-13 Targeted Genetics Corporation Packaging cell lines for generation of high titers of recombinant aav vectors
FR2737730B1 (fr) 1995-08-10 1997-09-05 Pasteur Merieux Serums Vacc Procede de purification de virus par chromatographie
WO1997008298A1 (en) 1995-08-30 1997-03-06 Genzyme Corporation Chromatographic purification of adenovirus and aav
US6632670B1 (en) 1995-09-08 2003-10-14 Genzyme Corporation AAV vectors for gene therapy
US5910434A (en) 1995-12-15 1999-06-08 Systemix, Inc. Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant
JP2001500497A (ja) 1996-09-06 2001-01-16 トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 組換えアデノ随伴ウイルスに指図される遺伝子治療の方法
DK1009808T3 (da) 1997-09-05 2013-01-21 Genzyme Corp Fremgangsmåder til generering af hjælper-frie præparater af rekombinante aav-vektorer med høj titer
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6258595B1 (en) 1999-03-18 2001-07-10 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
US7056502B2 (en) 2000-04-28 2006-06-06 The Trustees Of The University Of Pennsylvania Recombinant aav vectors with AAV5 capsids and AAV5 vectors pseudotyped in heterologous capsids
WO2002053703A2 (en) 2001-01-05 2002-07-11 Children's Hospital, Inc. Aav2 vectors and methods
PT1453547T (pt) * 2001-12-17 2016-12-28 Univ Pennsylvania Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas
US8030473B2 (en) * 2005-01-07 2011-10-04 State Of Oregon Acting By And Through The State Board Of Higher Education On Behalf Of Oregon State University Method to trigger RNA interference
DK2164967T3 (en) * 2007-05-31 2015-10-19 Univ Iowa Res Found Reduction of off-target rna interferenstoksicitet
WO2009082488A2 (en) 2007-12-24 2009-07-02 Bergenbio As Methods for creating and identifying functional rna interference elements
US9217155B2 (en) * 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
EP3444346B1 (en) * 2010-04-23 2022-07-27 University of Massachusetts Aav-based treatment of cholesterol-related disorders
ES2596977T3 (es) 2010-09-02 2017-01-13 Université de Mons Agentes útiles en el tratamiento de la distrofia muscular facioescapulohumeral
WO2012143401A1 (en) * 2011-04-18 2012-10-26 INSERM (Institut National de la Santé et de la Recherche Médicale) Tissue-specific regulation of transgene expression
EP2736539B1 (en) * 2011-07-25 2017-08-23 Nationwide Children's Hospital, Inc. Recombinant virus products and methods for inhibition of expression of dux4
WO2013120038A2 (en) * 2012-02-10 2013-08-15 Regents Of The University Of Minnesota Morpholino targeting dux4 for treating fshd
DE102012007232B4 (de) 2012-04-07 2014-03-13 Susanne Weller Verfahren zur Herstellung von rotierenden elektrischen Maschinen
CA2917348A1 (en) 2013-07-11 2015-01-15 Moderna Therapeutics, Inc. Compositions comprising synthetic polynucleotides encoding crispr related proteins and synthetic sgrnas and methods of use
JP2015092462A (ja) 2013-09-30 2015-05-14 Tdk株式会社 正極及びそれを用いたリチウムイオン二次電池
CA2942515C (en) 2014-03-18 2025-12-09 University Of Massachusetts Raav-based compositions and methods for treating amyotrophic lateral sclerosis
WO2015141521A1 (ja) 2014-03-21 2015-09-24 株式会社日立国際電気 基板処理装置、半導体装置の製造方法及び記録媒体
EP3171895A1 (en) 2014-07-23 2017-05-31 Modernatx, Inc. Modified polynucleotides for the production of intrabodies
JP6197169B2 (ja) 2014-09-29 2017-09-20 東芝メモリ株式会社 半導体装置の製造方法

Also Published As

Publication number Publication date
RU2768289C2 (ru) 2022-03-23
MA44528A (fr) 2019-02-06
NZ746893A (en) 2025-05-30
EP3435755A4 (en) 2019-11-27
IL262056A (en) 2018-11-29
US20230077409A1 (en) 2023-03-16
KR102453187B1 (ko) 2022-10-07
JP7253379B2 (ja) 2023-04-06
EP3435755A1 (en) 2019-02-06
AU2017240236A1 (en) 2018-10-18
EP4335502A2 (en) 2024-03-13
EP4335502A3 (en) 2024-07-03
IL262056B1 (en) 2023-06-01
AU2017240236B2 (en) 2023-07-27
RU2018137677A (ru) 2020-05-12
CA3019832A1 (en) 2017-10-05
US20250019697A1 (en) 2025-01-16
AU2017240236C1 (en) 2024-03-07
CN109714954A (zh) 2019-05-03
JP2019515663A (ja) 2019-06-13
JP2022046792A (ja) 2022-03-23
US11939579B2 (en) 2024-03-26
CA3019832C (en) 2023-05-09
US20190136235A1 (en) 2019-05-09
US11345913B2 (en) 2022-05-31
EP3435755B1 (en) 2023-11-01
KR20180130550A (ko) 2018-12-07
IL262056B2 (en) 2023-10-01
WO2017173411A1 (en) 2017-10-05
RU2018137677A3 (https=) 2020-07-23

Similar Documents

Publication Publication Date Title
BR112018070249A2 (pt) sistema promotor de u6 modificada para expressão específica de tecido
MX2017008500A (es) Supresión del gen de la huntingtina inducida por la arni.
AR121312A2 (es) AGENTES DE ARNi, COMPOSICIONES Y MÉTODOS DE USO DE LOS MISMOS PARA TRATAR ENFERMEDADES ASOCIADAS CON TRANSTIRETINA (TTR)
EP4253544A3 (en) Modified messenger rna comprising functional rna elements
BR112017028318A2 (pt) compostos antibacterianos
EP4520841A3 (en) Enzyme- and amplification-free sequencing
BR112016003591A8 (pt) promotor u6 de polimerase iii de soja e métodos de uso
MX2018000981A (es) Composiciones de acido ribonucleico interferente (arni) de transtiretina (ttr) y metodos para su uso para el tratamiento o prevencion de enfermedades asociadas con transtiretina (ttr).
MX385048B (es) Composiciones y metodos para inhibir la expresion del gen alas1.
BR112016007226A2 (pt) composições e métodos para inibição da expressão do gene alas1
MX2015016798A (es) Regulacion transcripcional guiada por acido ribonucleico.
TR201818825T4 (tr) Kardi̇yak hi̇pertrofi̇ni̇n tedavi̇si̇ne ve tanisina yöneli̇k lncrnalar
BR112021023488A2 (pt) Oligonucleotídeos de gapmer modificados e métodos de uso
BR112015021210A2 (pt) métodos e composições para o direcionamento de rna polimerases e biogênese de rna não codificante para locais específicos
BR112016002524A2 (pt) alvos moleculares para a prevenção e/ou tratamento de fibrose, cicatrizes hipertróficas e quelóides
MA39990A (fr) Induction de régénération cardiaque par micro-arn
MX381947B (es) Mirna natural para controlar la expresion de gen y uso del mismo.
BR112021006520A2 (pt) ácido ribonucleico de fita dupla que inibe a expressão de c5 complementar
MX2021013879A (es) Variantes de lipasa y polinucleotidos que las codifican.
BR112017012389A2 (pt) supressão de rnai parental de gene kruppel para controlar pragas de coleóptero
PL403939A1 (pl) Aptamery DNA rozpoznające metkę histydynową oraz ich zastosowanie
MX380693B (es) Produccion de manool.
UA114603C2 (uk) Конструкція для сайленсингу гена p0 та її застосування
BR112017013970A2 (pt) promotores específicos da semente de brassica napus identificados por análise de microarranjo
AR090582A1 (es) METODOS Y COMPOSICIONES PARA TRATAR CANCER USANDO ARNhc SRC 3 BIFUNCIONAL

Legal Events

Date Code Title Description
B350 Update of information on the portal [chapter 15.35 patent gazette]
B06W Patent application suspended after preliminary examination (for patents with searches from other patent authorities) chapter 6.23 patent gazette]
B11B Dismissal acc. art. 36, par 1 of ipl - no reply within 90 days to fullfil the necessary requirements