[go: up one dir, main page]

MX2017011010A - Moduladores de rodopsina p23h con especificidad de alelo. - Google Patents

Moduladores de rodopsina p23h con especificidad de alelo.

Info

Publication number
MX2017011010A
MX2017011010A MX2017011010A MX2017011010A MX2017011010A MX 2017011010 A MX2017011010 A MX 2017011010A MX 2017011010 A MX2017011010 A MX 2017011010A MX 2017011010 A MX2017011010 A MX 2017011010A MX 2017011010 A MX2017011010 A MX 2017011010A
Authority
MX
Mexico
Prior art keywords
rhodopsin
allele
adrp
subject
present
Prior art date
Application number
MX2017011010A
Other languages
English (en)
Other versions
MX391217B (es
Inventor
M Freier Susan
P Seth Punit
L Mccaleb Michael
Singh Priyam
F Murray Susan
Original Assignee
Ionis Pharmaceuticals 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 Ionis Pharmaceuticals Inc filed Critical Ionis Pharmaceuticals Inc
Publication of MX2017011010A publication Critical patent/MX2017011010A/es
Publication of MX391217B publication Critical patent/MX391217B/es

Links

Classifications

    • 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
    • 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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/47—Quinolines; Isoquinolines
    • A61K31/475—Quinolines; Isoquinolines having an indole ring, e.g. yohimbine, reserpine, strychnine, vinblastine
    • 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
    • A61K31/7028—Compounds having saccharide radicals attached to non-saccharide compounds by glycosidic linkages
    • A61K31/7034—Compounds having saccharide radicals attached to non-saccharide compounds by glycosidic linkages attached to a carbocyclic compound, e.g. phloridzin
    • A61K31/704—Compounds having saccharide radicals attached to non-saccharide compounds by glycosidic linkages attached to a carbocyclic compound, e.g. phloridzin attached to a condensed carbocyclic ring system, e.g. sennosides, thiocolchicosides, escin, daunorubicin
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • 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
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70571—Receptors; Cell surface antigens; Cell surface determinants for neuromediators, e.g. serotonin receptor, dopamine receptor
    • 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
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/341—Gapmers, i.e. of the type ===---===
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/345—Spatial arrangement of the modifications having at least two different backbone modifications
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • 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
    • C12N2320/34—Allele or polymorphism specific uses
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING 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
    • C12Q2600/00—Oligonucleotides characterized by their use
    • C12Q2600/178—Oligonucleotides characterized by their use miRNA, siRNA or ncRNA

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Biochemistry (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Ophthalmology & Optometry (AREA)
  • Biophysics (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Cell Biology (AREA)
  • Toxicology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Neurology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Plural Heterocyclic Compounds (AREA)

Abstract

Las modalidades de la presente invención proporcionan métodos, compuestos y composiciones para tratar, prevenir, mitigar o enlentecer el avance de la retinitis pigmentaria (RP), tal como la retinitis pigmentaria autosómica dominante (AdRP, por sus siglas en inglés) mediante la administración de un inhibidor específico de la rodopsina P23H a un sujeto. Las modalidades que se proporcionan en la presente invención se dirigen a composiciones y compuestos útiles para tratar, prevenir, mitigar o enlentecer el avance de la retinitis pigmentaria (RP), tal como la retinitis pigmentaria autosómica dominante (AdRP) . En determinadas modalidades, los inhibidores de la rodopsina P23H que se proporcionan en la presente invención son compuestos antisentido con especificidad de alelo dirigidos a un alelo mutante P23H que son capaces de inhibir de manera selectiva la expresión de la proteína mutante de la rodopsina P23H en mayor medida que la proteína de tipo salvaje. En determinadas modalidades, la administración de compuestos antisentido con especificidad de alelo en un sujeto con AdRP produce la inhibición selectiva de la rodopsina P23H y permite que la proteína normal producida a partir del alelo de tipo salvaje mantenga la supervivencia y el funcionamiento de los bastones en el sujeto.
MX2017011010A 2015-02-26 2016-02-26 Moduladores de rodopsina p23h con especificidad de alelo. MX391217B (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562121286P 2015-02-26 2015-02-26
PCT/US2016/019725 WO2016138353A1 (en) 2015-02-26 2016-02-26 Allele specific modulators of p23h rhodopsin

Publications (2)

Publication Number Publication Date
MX2017011010A true MX2017011010A (es) 2017-10-20
MX391217B MX391217B (es) 2025-03-21

Family

ID=56789889

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2017011010A MX391217B (es) 2015-02-26 2016-02-26 Moduladores de rodopsina p23h con especificidad de alelo.

Country Status (11)

Country Link
US (3) US10426789B2 (es)
EP (1) EP3261643B1 (es)
AU (1) AU2016222546B2 (es)
BR (1) BR112017017178A2 (es)
CA (1) CA2977965C (es)
ES (1) ES2848377T3 (es)
HK (1) HK1248132A1 (es)
IL (1) IL253503B (es)
MX (1) MX391217B (es)
NZ (1) NZ733844A (es)
WO (1) WO2016138353A1 (es)

Families Citing this family (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2977965C (en) * 2015-02-26 2021-12-21 Ionis Pharmaceuticals, Inc. Allele specific modulators of p23h rhodopsin
ES2923877T3 (es) 2016-03-01 2022-10-03 Univ Florida Vectores VAA para el tratamiento de la retinitis pigmentosa dominante
US11584932B2 (en) 2016-11-01 2023-02-21 The Research Foundation For The State University Of New York 5-halouracil-modified microRNAs and their use in the treatment of cancer
AU2017368050A1 (en) 2016-11-29 2019-06-20 Puretech Lyt, Inc. Exosomes for delivery of therapeutic agents
AR112779A1 (es) 2017-08-08 2019-12-11 Wave Life Sciences Ltd Composiciones de nucleótidos y métodos relacionados
AU2018372763A1 (en) 2017-11-21 2020-05-28 Bayer Healthcare Llc Materials and methods for treatment of autosomal dominant Retinitis Pigmentosa
CA3090901A1 (en) 2018-02-12 2019-08-15 Ionis Pharmaceuticals, Inc. Modified compounds and uses thereof
PE20201349A1 (es) * 2018-04-11 2020-11-30 Ionis Pharmaceuticals Inc Moduladores de la expresion de ezh2
CN112204145B (zh) 2018-06-01 2024-03-26 佛罗里达大学研究基金会有限公司 用于治疗显性视网膜色素变性的组合物和方法
CA3124493A1 (en) 2019-01-28 2020-08-06 Proqr Therapeutics Ii B.V. Rna-editing oligonucleotides for the treatment of usher syndrome
EP3941483A4 (en) * 2019-03-21 2023-10-18 Arnay Sciences, Llc ANTISEN OLIGONUCLEOTIDES FOR ALLEL SPECIFICITY
WO2020219983A2 (en) * 2019-04-25 2020-10-29 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
EP4081638A1 (en) 2019-12-23 2022-11-02 ProQR Therapeutics II B.V. Antisense oligonucleotides for nucleotide deamination in the treatment of stargardt disease
US20230151366A1 (en) * 2020-04-17 2023-05-18 Ionis Pharmaceuticals, Inc. COMPOSITIONS AND METHODS OF INHIBITING SEVERE ACUTE RESPIRATORY SYNDROME CORONAVIRUS 2 (SARS-CoV-2)
WO2021262919A2 (en) 2020-06-26 2021-12-30 The Research Foundation For The State University Of New York 5-halouracil-modified micrornas and their use in the treatment of cancer
US20240141340A1 (en) 2021-03-05 2024-05-02 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for use in the treatment of corneal dystrophies
EP4422645A4 (en) * 2021-10-27 2025-12-03 Wave Life Sciences Ltd OLIGONUCLEOTID COMPOSITIONS AND THEIR METHODS OF USE
US20250154504A1 (en) 2022-02-14 2025-05-15 Proqr Therapeutics Ii B.V. Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia

Family Cites Families (73)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5118800A (en) 1983-12-20 1992-06-02 California Institute Of Technology Oligonucleotides possessing a primary amino group in the terminal nucleotide
FR2567892B1 (fr) 1984-07-19 1989-02-17 Centre Nat Rech Scient Nouveaux oligonucleotides, leur procede de preparation et leurs applications comme mediateurs dans le developpement des effets des interferons
US5506337A (en) 1985-03-15 1996-04-09 Antivirals Inc. Morpholino-subunit combinatorial library and method
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US5185444A (en) 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
US5166315A (en) 1989-12-20 1992-11-24 Anti-Gene Development Group Sequence-specific binding polymers for duplex nucleic acids
US5591722A (en) 1989-09-15 1997-01-07 Southern Research Institute 2'-deoxy-4'-thioribonucleosides and their antiviral activity
DK0942000T3 (da) 1989-10-24 2004-11-01 Isis Pharmaceuticals Inc 2'-modificerede oligonukleotider
US5670633A (en) 1990-01-11 1997-09-23 Isis Pharmaceuticals, Inc. Sugar modified oligonucleotides that detect and modulate gene expression
US5646265A (en) 1990-01-11 1997-07-08 Isis Pharmceuticals, Inc. Process for the preparation of 2'-O-alkyl purine phosphoramidites
US5262529A (en) 1990-01-24 1993-11-16 President And Fellows Of Harvard College Diagnosis of hereditary retinal degenerative diseases
US5225546A (en) 1990-01-24 1993-07-06 President And Fellows Of Harvard College Diagnosis of hereditary retinal degenerative disease
US5498521A (en) 1990-01-24 1996-03-12 President And Fellows Of Harvard College Diagnosis of hereditary retinal degenerative diseases
GB9009980D0 (en) 1990-05-03 1990-06-27 Amersham Int Plc Phosphoramidite derivatives,their preparation and the use thereof in the incorporation of reporter groups on synthetic oligonucleotides
EP0745689A3 (en) 1990-05-11 1996-12-11 Microprobe Corporation A dipstick for a nucleic acid hybridization assay
US6582908B2 (en) 1990-12-06 2003-06-24 Affymetrix, Inc. Oligonucleotides
DE59208572D1 (de) 1991-10-17 1997-07-10 Ciba Geigy Ag Bicyclische Nukleoside, Oligonukleotide, Verfahren zu deren Herstellung und Zwischenprodukte
US5359044A (en) 1991-12-13 1994-10-25 Isis Pharmaceuticals Cyclobutyl oligonucleotide surrogates
FR2687679B1 (fr) 1992-02-05 1994-10-28 Centre Nat Rech Scient Oligothionucleotides.
EP0577558A2 (de) 1992-07-01 1994-01-05 Ciba-Geigy Ag Carbocyclische Nukleoside mit bicyclischen Ringen, Oligonukleotide daraus, Verfahren zu deren Herstellung, deren Verwendung und Zwischenproduckte
JPH08504559A (ja) 1992-12-14 1996-05-14 ハネウエル・インコーポレーテッド 個別に制御される冗長巻線を有するモータシステム
JPH08508492A (ja) 1993-03-30 1996-09-10 スターリング ウィンスロップ インコーポレイティド 非環式ヌクレオシド類似体及びそれらを含むオリゴヌクレオチド配列
US5801154A (en) 1993-10-18 1998-09-01 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of multidrug resistance-associated protein
US5446137B1 (en) 1993-12-09 1998-10-06 Behringwerke Ag Oligonucleotides containing 4'-substituted nucleotides
US5519134A (en) 1994-01-11 1996-05-21 Isis Pharmaceuticals, Inc. Pyrrolidine-containing monomers and oligomers
US5627053A (en) 1994-03-29 1997-05-06 Ribozyme Pharmaceuticals, Inc. 2'deoxy-2'-alkylnucleotide containing nucleic acid
US5597909A (en) 1994-08-25 1997-01-28 Chiron Corporation Polynucleotide reagents containing modified deoxyribose moieties, and associated methods of synthesis and use
GB9604449D0 (en) 1996-03-01 1996-05-01 Farrar Gwyneth J Genetic strategy ii
GB9606961D0 (en) 1996-04-02 1996-06-05 Farrar Gwyneth J Genetic strategy III
US8551970B2 (en) 1996-04-02 2013-10-08 Optigen Patents Limited Genetic suppression and replacement
JP3756313B2 (ja) 1997-03-07 2006-03-15 武 今西 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体
US6770748B2 (en) 1997-03-07 2004-08-03 Takeshi Imanishi Bicyclonucleoside and oligonucleotide analogue
CA2287508A1 (en) 1997-04-21 1998-10-29 The University Of Florida Materials and methods for ribozyme treatment of retinal diseases
US20050096282A1 (en) 1997-04-21 2005-05-05 Lewin Alfred S. Adeno-associated virus-delivered ribozyme compositions and methods for the treatment of retinal diseases
US6794499B2 (en) 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
EP2341058A3 (en) 1997-09-12 2011-11-23 Exiqon A/S Oligonucleotide Analogues
US20030228597A1 (en) 1998-04-13 2003-12-11 Cowsert Lex M. Identification of genetic targets for modulation by oligonucleotides and generation of oligonucleotides for gene modulation
US6043352A (en) 1998-08-07 2000-03-28 Isis Pharmaceuticals, Inc. 2'-O-Dimethylaminoethyloxyethyl-modified oligonucleotides
EP1171586B1 (en) 1999-04-21 2013-06-12 Alnylam Pharmaceuticals, Inc. Methods and compositions for inhibiting the function of polynucleotide sequences
ATE356824T1 (de) 1999-05-04 2007-04-15 Santaris Pharma As L-ribo-lna analoge
US6525191B1 (en) 1999-05-11 2003-02-25 Kanda S. Ramasamy Conformationally constrained L-nucleosides
ES2261270T3 (es) 1999-12-30 2006-11-16 K.U. LEUVEN RESEARCH & DEVELOPMENT Acidos nucleicos que contienen ciclohexeno.
US20050019915A1 (en) * 2001-06-21 2005-01-27 Bennett C. Frank Antisense modulation of superoxide dismutase 1, soluble expression
CA2452458A1 (en) 2001-07-03 2003-01-16 Isis Pharmaceuticals, Inc. Nuclease resistant chimeric oligonucleotides
US20040142325A1 (en) 2001-09-14 2004-07-22 Liat Mintz Methods and systems for annotating biomolecular sequences
EP2957568B1 (en) 2002-11-05 2016-12-21 Ionis Pharmaceuticals, Inc. Compositions comprising alternating 2'-modified nucleosides for use in gene modulation
CA2504694C (en) 2002-11-05 2013-10-01 Isis Pharmaceuticals, Inc. Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
EP1560931B1 (en) 2002-11-14 2011-07-27 Dharmacon, Inc. Functional and hyperfunctional sirna
US6673661B1 (en) 2002-12-20 2004-01-06 Taiwan Semiconductor Manufacturing Co., Ltd. Self-aligned method for forming dual gate thin film transistor (TFT) device
WO2004106356A1 (en) 2003-05-27 2004-12-09 Syddansk Universitet Functionalized nucleotide derivatives
EP1661905B9 (en) 2003-08-28 2012-12-19 IMANISHI, Takeshi Novel artificial nucleic acids of n-o bond crosslinkage type
JP5379347B2 (ja) 2003-09-18 2013-12-25 アイシス ファーマシューティカルズ， インコーポレーテッド ４’−チオヌクレオシドおよびオリゴマー化合物
US7687616B1 (en) 2004-05-14 2010-03-30 Rosetta Genomics Ltd Small molecules modulating activity of micro RNA oligonucleotides and micro RNA targets and uses thereof
JP2008501694A (ja) 2004-06-03 2008-01-24 アイシス ファーマシューティカルズ、インク． 遺伝子調節の使用のために個別に修飾された鎖を有する二本鎖組成物
EP1812569A2 (en) 2004-11-08 2007-08-01 K.U. Leuven Research and Development Modified nucleosides for rna interference
DK2314594T3 (da) 2006-01-27 2014-10-27 Isis Pharmaceuticals Inc 6-modificerede bicykliske nukleinsyreanaloger
AU2007249349B2 (en) 2006-05-11 2012-03-08 Isis Pharmaceuticals, Inc. 5'-Modified bicyclic nucleic acid analogs
US20100190837A1 (en) 2007-02-15 2010-07-29 Isis Pharmaceuticals, Inc. 5'-Substituted-2-F' Modified Nucleosides and Oligomeric Compounds Prepared Therefrom
EP2170917B1 (en) 2007-05-30 2012-06-27 Isis Pharmaceuticals, Inc. N-substituted-aminomethylene bridged bicyclic nucleic acid analogs
ES2386492T3 (es) 2007-06-08 2012-08-21 Isis Pharmaceuticals, Inc. Análogos de ácidos nucleicos bicíclicos carbocíclicos
ATE538127T1 (de) 2007-07-05 2012-01-15 Isis Pharmaceuticals Inc 6-disubstituierte bicyclische nukleinsäureanaloga
EP2227545A2 (en) 2007-11-09 2010-09-15 Isis Pharmaceuticals, Inc. Modulation of factor 7 expression
EP2361256B1 (en) 2008-09-24 2013-04-10 Isis Pharmaceuticals, Inc. Cyclohexenyl nucleic acid analogs
US8450473B2 (en) 2009-04-30 2013-05-28 The Research Foundation Of State University Of New York Compositions and methods for therapy of macular degeneration
US20130028889A1 (en) 2010-02-04 2013-01-31 Ico Therapeutics Inc. Dosing regimens for treating and preventing ocular disorders using c-raf antisense
JP6005628B2 (ja) 2010-04-28 2016-10-12 アイオーニス ファーマシューティカルズ， インコーポレーテッドＩｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． 修飾ヌクレオシド、その類似体、およびこれらから調製されるオリゴマー化合物
US20120204282A1 (en) 2011-02-04 2012-08-09 Sangamo Biosciences, Inc. Methods and compositions for treating occular disorders
EP2702170B1 (en) 2011-04-28 2015-12-16 Life Technologies Corporation Methods and compositions for multiplex pcr
EP3922722B1 (en) 2011-08-11 2023-06-28 Ionis Pharmaceuticals, Inc. Selective antisense compounds and uses thereof
US20140378533A1 (en) 2012-02-08 2014-12-25 Isis Pharmaceuticals, Inc. Modulation of rna by repeat targeting
JP2016522674A (ja) 2012-05-16 2016-08-04 ラナ セラピューティクス インコーポレイテッド 遺伝子発現を調節するための組成物及び方法
AR092982A1 (es) 2012-10-11 2015-05-13 Isis Pharmaceuticals Inc Modulacion de la expresion de receptores androgenicos
CA2977965C (en) * 2015-02-26 2021-12-21 Ionis Pharmaceuticals, Inc. Allele specific modulators of p23h rhodopsin

Also Published As

Publication number Publication date
BR112017017178A2 (pt) 2018-04-03
WO2016138353A1 (en) 2016-09-01
HK1248132A1 (zh) 2018-10-12
IL253503B (en) 2021-08-31
CA2977965A1 (en) 2016-09-01
US10426789B2 (en) 2019-10-01
US20180169131A1 (en) 2018-06-21
IL253503A0 (en) 2017-09-28
AU2016222546A1 (en) 2017-08-03
MX391217B (es) 2025-03-21
US11744846B2 (en) 2023-09-05
EP3261643A4 (en) 2018-11-07
US20210346421A1 (en) 2021-11-11
EP3261643A1 (en) 2018-01-03
AU2016222546B2 (en) 2020-01-23
CA2977965C (en) 2021-12-21
ES2848377T3 (es) 2021-08-09
NZ733844A (en) 2022-07-01
EP3261643B1 (en) 2020-11-25
US20200069722A1 (en) 2020-03-05
US11013758B2 (en) 2021-05-25

Similar Documents

Publication Publication Date Title
MX2017011010A (es) Moduladores de rodopsina p23h con especificidad de alelo.
UY37464A (es) Métodos para tratar infecciones por el virus filoviridae
DOP2019000266A (es) Inhibidores de pd-1/pd-l1
CL2018003213A1 (es) Inhibidores enzimáticos.
UY36748A (es) Compuestos de aminopiridina sustituida con heteroarilo como inhibidores de quinasa y moduladores de irak-4, y composiciones farmacéuticas que los contienen
UY36747A (es) Compuestos de aminopiridina sustituida con heteroarilo como inhibidores de quinasa y modulantes de irak-4 y composiciones farmacéuticas que los contienen
MX2019002750A (es) Derivados de pirazolopiridina como moduladores de cinasa 1 progenitora hematopoyetica (hpk1) y usos de los mismos para tratamiento de cancer.
UY35617A (es) Inhibidores de la fosfatidilinositol 3-quinasa
CO2017007671A2 (es) Composiciones para modular la expresión de c9orf72
UY36749A (es) Compuestos de aminopiridina sustituida con heteroarilo como inhibidores de quinasa y moduladores de irak-4, y composiciones farmacéuticas que los contienen
CU24389B1 (es) Compuestos de heterociclilo bicíclico como inhibidores de irak4
MX2015012867A (es) Compuestos aromaticos multisustituidos como inhibidores de serina proteasa.
MX2017000450A (es) Compuestos terapeuticos inhibidores.
CO2017003838A2 (es) Compuestos de aminopurina sustituida, composiciones del mismo, y métodos de tratamiento con los mismos
MX2017004618A (es) Inhibidores de la biosintesis de sulfato de heparano para el tratamiento de enfermedades.
CO2017000011A2 (es) Derivados de quinolizinona como inhibidores de pi3k
MX2017012068A (es) Nuevos agentes citotóxicos que preferentemente se dirigen al factor inhibidor de leucemia (lif) para el tratamiento de malignidades y como nuevos agentes anticonceptivos.
DOP2014000046A (es) COMPOSICIONES Y MÉTODOS PARA TRATAR EL CÁNCER USANDO EL INHIBIDOR DE PI3Kbeta Y EL INHIBIDOR DE LA VÍA DE MAPK, INCLUIDOS LOS INHIBIDORES DE MEK Y RAF
ECSP16083017A (es) Derivados de diheterociclo enlazado a cicloalquilo
CO2021010485A2 (es) Derivados de pirazolopiridina como inhibidores serina/treonina cinasa que contiene el dominio per-arnt-sim (pask)
CO2019008876A2 (es) Liposomas hiperestabilizados aumentan el ataque direccionado a células mitóticas
AR109850A2 (es) Métodos para tratar infecciones por el virus filoviridae
AR104542A1 (es) COMPOSICIONES DE ARNi CONTRA FACTOR XII (FACTOR DE HAGEMAN) (F12), CALICREÍNA B, PLASMÁTICA (FACTOR DE FLETCHER) 1 (KLKB1), Y QUININÓGENO 1 (KNG1) Y MÉTODOS DE USO DE LOS MISMOS