ATE449170T1 - CHIMÄRE SNRNA MOLEKÜLE MIT ANTISENSE-SEQUENZEN GEGEN DIE ßSPLICE JUNCTIONSß DES DYSTROPHIN-GENS UND IHRE THERAPEUTISCHEN VERWENDUNGEN - Google Patents

CHIMÄRE SNRNA MOLEKÜLE MIT ANTISENSE-SEQUENZEN GEGEN DIE ßSPLICE JUNCTIONSß DES DYSTROPHIN-GENS UND IHRE THERAPEUTISCHEN VERWENDUNGEN

Info

Publication number
ATE449170T1
ATE449170T1 AT03749984T AT03749984T ATE449170T1 AT E449170 T1 ATE449170 T1 AT E449170T1 AT 03749984 T AT03749984 T AT 03749984T AT 03749984 T AT03749984 T AT 03749984T AT E449170 T1 ATE449170 T1 AT E449170T1
Authority
AT
Austria
Prior art keywords
gene
ßsplice
junctionsß
therapeutic uses
antisense sequences
Prior art date
Application number
AT03749984T
Other languages
English (en)
Inventor
I Bozzoni
Angelis F G De
Original Assignee
Uni Degli Studi Di Roma Sla Sa
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 Uni Degli Studi Di Roma Sla Sa filed Critical Uni Degli Studi Di Roma Sla Sa
Application granted granted Critical
Publication of ATE449170T1 publication Critical patent/ATE449170T1/de

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
    • C12N15/113—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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C12N2310/111—Antisense spanning the whole gene, or a large part of it

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
AT03749984T 2002-05-08 2003-05-06 CHIMÄRE SNRNA MOLEKÜLE MIT ANTISENSE-SEQUENZEN GEGEN DIE ßSPLICE JUNCTIONSß DES DYSTROPHIN-GENS UND IHRE THERAPEUTISCHEN VERWENDUNGEN ATE449170T1 (de)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
IT2002RM000253A ITRM20020253A1 (it) 2002-05-08 2002-05-08 Molecole chimeriche di snrna con sequenze antisenso per le giunzioni di splicing del gene della distrofina e applicazioni terapeutiche.
PCT/IT2003/000273 WO2003095647A2 (en) 2002-05-08 2003-05-06 CHIMERIC snRNA MOLECULES CARRYING ANTISENSE SEQUENCES AGAINST THE SPLICE JUNCTIONS OF THE DYSTROPHIN GENE AND THEIR THERAPEUTIC APPLICATIONS

Publications (1)

Publication Number Publication Date
ATE449170T1 true ATE449170T1 (de) 2009-12-15

Family

ID=11456291

Family Applications (1)

Application Number Title Priority Date Filing Date
AT03749984T ATE449170T1 (de) 2002-05-08 2003-05-06 CHIMÄRE SNRNA MOLEKÜLE MIT ANTISENSE-SEQUENZEN GEGEN DIE ßSPLICE JUNCTIONSß DES DYSTROPHIN-GENS UND IHRE THERAPEUTISCHEN VERWENDUNGEN

Country Status (11)

Country Link
EP (2) EP2151497B1 (de)
AT (1) ATE449170T1 (de)
AU (1) AU2003231928A1 (de)
CY (1) CY1109746T1 (de)
DE (1) DE60330136D1 (de)
DK (1) DK1501931T3 (de)
ES (1) ES2336909T3 (de)
IT (1) ITRM20020253A1 (de)
PT (1) PT1501931E (de)
SI (1) SI1501931T1 (de)
WO (1) WO2003095647A2 (de)

Families Citing this family (63)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1999049029A1 (en) 1998-03-20 1999-09-30 Benitec Australia Ltd Control of gene expression
AUPP249298A0 (en) 1998-03-20 1998-04-23 Ag-Gene Australia Limited Synthetic genes and genetic constructs comprising same I
US6423885B1 (en) 1999-08-13 2002-07-23 Commonwealth Scientific And Industrial Research Organization (Csiro) Methods for obtaining modified phenotypes in plant cells
AUPR621501A0 (en) 2001-07-06 2001-08-02 Commonwealth Scientific And Industrial Research Organisation Delivery of ds rna
WO2005026356A1 (en) * 2003-09-12 2005-03-24 Commonwealth Scientific And Industrial Research Organisation Modified gene-silencing nucleic acid molecules and uses thereof
FR2874384B1 (fr) 2004-08-17 2010-07-30 Genethon Vecteur viral adeno-associe pour realiser du saut d'exons dans un gene codant une proteine a domaines dispensables
WO2007123391A1 (en) 2006-04-20 2007-11-01 Academisch Ziekenhuis Leiden Therapeutic intervention in a genetic disease in an individual by modifying expression of an aberrantly expressed gene.
EP1857548A1 (de) 2006-05-19 2007-11-21 Academisch Ziekenhuis Leiden Mittel und Verfahren zur Induktion von Exon-skipping
AU2007282224B2 (en) 2006-08-11 2013-08-29 Vico Therapeutics B.V. Methods and means for treating DNA repeat instability associated genetic disorders
EP2152873A2 (de) * 2007-03-16 2010-02-17 Biorigen S.r.l Technologie zur regulierung der genexpression und nichtkodierende rna für diagnose und therapie
US8343941B2 (en) 2007-03-30 2013-01-01 Rutgers, The State University Of New Jersey Compositions and methods for gene silencing
US8907075B2 (en) 2007-03-30 2014-12-09 Samuel Ian Gunderson Compositions and methods for gene silencing
EP2142672B1 (de) * 2007-03-30 2012-09-05 Rutgers, The State University of New Jersey Zusammensetzungen und Verfahren zum Gen-Silencing
US9441221B2 (en) 2007-03-30 2016-09-13 Rutgers, The State University Of New Jersey Compositions and methods for gene silencing
WO2009008727A2 (en) 2007-07-12 2009-01-15 Prosensa Technologies B.V. Molecules for targeting compounds to various selected organs or tissues
CA2693742A1 (en) 2007-07-12 2009-01-15 Prosensa Technologies B.V. Molecules for targeting compounds to various selected organs, tissues or tumor cells
USRE48468E1 (en) 2007-10-26 2021-03-16 Biomarin Technologies B.V. Means and methods for counteracting muscle disorders
AU2008317566B2 (en) 2007-10-26 2014-05-01 Academisch Ziekenhuis Leiden Means and methods for counteracting muscle disorders
NZ587178A (en) 2008-02-08 2011-11-25 Prosensa Holding Bv Methods and means for treating dna repeat instability associated genetic disorders
EP2119783A1 (de) 2008-05-14 2009-11-18 Prosensa Technologies B.V. Verfahren für effizientes Exon (44)-Skipping bei Duchenne-Muskeldystrophie und entsprechende Mittel
EP2396343B1 (de) 2009-02-11 2017-05-17 The University of North Carolina At Chapel Hill Modifizierte virusvektoren sowie verfahren zu ihrer herstellung und verwendung
CA2759899A1 (en) 2009-04-24 2010-10-28 Prosensa Technologies B.V. Oligonucleotide comprising an inosine for treating dmd
EP2258863A1 (de) * 2009-05-25 2010-12-08 Universita'Degli Studi di Roma "La Sapienza" miRNA biomarkers für die Diagnose und Weiterentwicklung der Duchenne Dystrophie, für die Überwachung therapeutischer Interventionen und als Therapeutik
WO2011078672A1 (en) 2009-12-24 2011-06-30 Prosensa Technologies B.V. Molecule for treating an inflammatory disorder
ES2683695T3 (es) 2010-01-12 2018-09-27 The University Of North Carolina At Chapel Hill Repeticiones terminales invertidas restrictivas para vectores virales
TWI541024B (zh) 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
WO2012109570A1 (en) 2011-02-10 2012-08-16 The University Of North Carolina At Chapel Hill Viral vectors with modified transduction profiles and methods of making and using the same
WO2012138846A2 (en) * 2011-04-05 2012-10-11 The Regents Of The University Of California Method and compositions comprising small rna agonist and antagonists to modulate inflammation
CN112251436A (zh) 2012-01-27 2021-01-22 比奥马林技术公司 治疗杜兴型肌营养不良症和贝克型肌营养不良症的具有改善特性的rna调节性寡核苷酸
US9636370B2 (en) 2012-09-28 2017-05-02 The University Of North Carolina At Chapel Hill AAV vectors targeted to oligodendrocytes
US9447433B2 (en) 2013-03-15 2016-09-20 The University Of North Carolina At Chapel Hill Synthetic adeno-associated virus inverted terminal repeats
JP6396988B2 (ja) 2013-03-15 2018-09-26 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル 二重グリカン結合性ａａｖベクターのための方法および組成物
WO2016081811A1 (en) 2014-11-21 2016-05-26 The University Of North Carolina At Chapel Hill Aav vectors targeted to the central nervous system
AU2016206624B2 (en) 2015-01-14 2020-08-06 The University Of North Carolina At Chapel Hill Methods and compositions for targeted gene transfer
MX2017010698A (es) 2015-02-20 2018-04-30 Univ Texas Metodos y composiciones para chlamydia atenuada como vacuna y vector.
BR112018003665A2 (en) 2015-09-28 2018-09-25 The University Of North Carolina At Chapel Hill methods and compositions for antibody evasion viral vectors
EP4659816A3 (de) 2015-12-14 2025-12-31 The University of North Carolina at Chapel Hill Modifizierte kapsidproteine zur verbesserten abgabe von parvovirusvektoren
CN110770346B (zh) 2017-03-15 2024-01-12 北卡罗来纳-查佩尔山大学 多倍体腺相关病毒载体及其制备和使用方法
IL273245B2 (en) 2017-09-15 2025-03-01 Commw Scient Ind Res Org RNA molecules
BR112020016699A2 (pt) 2018-02-28 2020-12-15 The University Of North Carolina At Chapel Hill Métodos e composições para vetores de vírus de evasão de anticorpos
CA3094465A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Virus vectors for targeting ophthalmic tissues
CN112543766A (zh) 2018-04-03 2021-03-23 斯特里迪比奥公司 抗体逃避性病毒载体
US20210363191A1 (en) 2018-04-03 2021-11-25 Stridebio, Inc. Antibody-evading virus vectors
CN112566923B (zh) 2018-06-12 2024-10-29 北卡罗来纳大学教堂山分校 合成嗜肝性腺相关病毒衣壳及其用途
EP3810782A2 (de) 2018-06-22 2021-04-28 Asklepios Biopharmaceutical, Inc. Vektoren zur genfreisetzung, die in zellen persistieren
BR112021018776A2 (pt) 2019-03-21 2021-11-30 Stridebio Inc Vetores de vírus adenoassociado recombinante
EP3959227A1 (de) 2019-04-26 2022-03-02 The University of North Carolina at Chapel Hill Verfahren und zusammensetzungen für dualen glycanbindenden aav2,5-vektor
KR20220083714A (ko) 2019-10-17 2022-06-20 스트라이드바이오 인코포레이티드 니만-피크 질환 c형의 치료를 위한 아데노-연관 바이러스 벡터
EP4100523A4 (de) 2020-02-04 2024-03-06 Duke University Zusammensetzungen und verfahren zur zirkulären rna-expression
US12209251B2 (en) 2020-05-05 2025-01-28 The University Of North Carolina At Chapel Hill Modified adeno-associated virus 5 capsids and uses thereof
EP4200408A1 (de) 2020-08-19 2023-06-28 Sarepta Therapeutics, Inc. Adeno-assoziierte virusvektoren zur behandlung des rett-syndroms
ES3001131T3 (es) 2020-10-28 2025-03-04 Univ North Carolina Chapel Hill Métodos y composiciones para la unión dual de glicanos al vector de AAV2.5
WO2022093769A1 (en) 2020-10-28 2022-05-05 The University Of North Carolina At Chapel Hill Methods and compositions for dual glycan binding aav2.5 vector
TW202242124A (zh) 2021-01-14 2022-11-01 美商史崔德生物公司 靶向ｔ細胞之ａａｖ載體
US20240398993A1 (en) 2021-01-15 2024-12-05 Glenna FOIGHT Small molecule-regulated gene expression system
JP2024515626A (ja) 2021-04-16 2024-04-10 アスクレピオス バイオファーマシューティカル， インコーポレイテッド 血液脳関門を横断し低減された液性応答を惹起する合理的ポリプロイドａａｖビリオン
WO2023150687A1 (en) 2022-02-04 2023-08-10 Ginkgo Bioworks, Inc. Recombinant adeno-associated virus vectors, and methods of use thereof
JP2025507958A (ja) 2022-03-04 2025-03-21 ロックアネイビオ， インコーポレイテッド 操作された核内低分子ＲＮＡ（ｓｎＲＮＡ）を含む組成物および方法
AR128918A1 (es) * 2022-03-30 2024-06-26 Biomarin Pharm Inc Oligonucleótidos que omiten el exón 51 de distrofina y sus aplicaciones
CN120129747A (zh) * 2022-10-11 2025-06-10 广州瑞风生物科技有限公司 snRNA核酸分子及其应用
CN115806984B (zh) * 2022-10-18 2023-10-10 昆明理工大学 环状rna及载体和载体的应用
WO2024124019A2 (en) 2022-12-07 2024-06-13 Ginkgo Bioworks, Inc. Aav vectors targeting hematopoietic stem cells
WO2025079025A1 (en) 2023-10-13 2025-04-17 Takeda Pharmaceutical Company Limited Ides-rapamycin conjugate in aav gene therapy

Family Cites Families (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1191097A1 (de) * 2000-09-21 2002-03-27 Leids Universitair Medisch Centrum Induktion von "exon-skipping" in eukaryotischen Zellen

Also Published As

Publication number Publication date
PT1501931E (pt) 2010-02-23
SI1501931T1 (sl) 2010-03-31
EP2151497B1 (de) 2014-01-08
AU2003231928A8 (en) 2003-11-11
EP2151497A2 (de) 2010-02-10
CY1109746T1 (el) 2014-09-10
WO2003095647A2 (en) 2003-11-20
EP1501931B1 (de) 2009-11-18
WO2003095647A3 (en) 2004-02-19
DK1501931T3 (da) 2010-03-29
ITRM20020253A1 (it) 2003-11-10
DE60330136D1 (de) 2009-12-31
ES2336909T3 (es) 2010-04-19
EP1501931A2 (de) 2005-02-02
AU2003231928A1 (en) 2003-11-11
ITRM20020253A0 (it) 2002-05-08
EP2151497A3 (de) 2010-03-10

Similar Documents

Publication Publication Date Title
ATE449170T1 (de) CHIMÄRE SNRNA MOLEKÜLE MIT ANTISENSE-SEQUENZEN GEGEN DIE ßSPLICE JUNCTIONSß DES DYSTROPHIN-GENS UND IHRE THERAPEUTISCHEN VERWENDUNGEN
EP1569695A4 (de) Antisense-modulationder apolipoprotein-b-expression
EP2253706A3 (de) Antisense-Modulation von Kinesin-1-Expression
EP1325019A4 (de) Antisense-modulation der clusterinexpression
ITRM20030149A1 (it) Oligonucleotidi (odn) antisenso per smad7 e loro usi in campo medico
EP1576108A4 (de) Nitrilasen, diese codierende nukleinsäuren sowie verfahren zur herstellung und verwendung davon
EP1417351A4 (de) Antisense-modulation der expression von apolipoprotein(a)
EP1250157A4 (de) Antisens-modulation der expression der induzierbaren stickstoffmonoxid-synthase
WO2003097662A8 (en) Antisense modulation of apolipoprotein b expression
WO2008143668A3 (en) Indoleamine-2, 3-dioxygenase-2
WO2003054154A3 (en) Antisense modulation of mucin 1, transmembrane expression
WO2003014306A3 (en) Antisense modulation of cholesteryl ester transfer protein expression
EP1648910A4 (de) Antisense-modulation der expression von stearoyl coa desaturase
WO2001029175A3 (en) Antisense modulation of fra-1 expression
EA200400812A1 (ru) Секретируемый белок
PL1631296T3 (pl) Pochodne amidu kwasu 2,3,4,5,-tetrahydrobenzo[f][1,4]oksazepino-5-karboksylowego jako inhibitory gamma-sekretazy do leczenia choroby Alzheimera
WO2003099224A3 (en) Antisense moodulation of kinesin-like 1 expression
EP1390388A4 (de) Antisense-modulation der expression des interferon-gamma-rezeptors 1
WO2003040161A3 (en) Antisense modulation of activating transcription factor 3 expression
WO2004001061A3 (en) Antisense modulation of heme oxygenase 1 expression
EP1441740A4 (de) Antisense-modulation der matrix-metalloproteinase-1-expression
IL169256A0 (en) Metalloprotease proteins
EP1373291A4 (de) Antisense-modulierung der recql4 expression
AU2003255504A8 (en) Use of 6 amino-quinoline-5, 8 quinones and nucleic acids associated with senescence for the treatment of tumors
HUP0100660A1 (hu) Negatív szabályozóelemek alkalmazása transzgének neurospecifikus kifejezésére

Legal Events

Date Code Title Description
UEP Publication of translation of european patent specification

Ref document number: 1501931

Country of ref document: EP