MX2017010369A - Arni variante. - Google Patents

Arni variante.

Info

Publication number
MX2017010369A
MX2017010369A MX2017010369A MX2017010369A MX2017010369A MX 2017010369 A MX2017010369 A MX 2017010369A MX 2017010369 A MX2017010369 A MX 2017010369A MX 2017010369 A MX2017010369 A MX 2017010369A MX 2017010369 A MX2017010369 A MX 2017010369A
Authority
MX
Mexico
Prior art keywords
rnai
guide sequence
recombinant
disease
further provided
Prior art date
Application number
MX2017010369A
Other languages
English (en)
Inventor
Pablo Sardi Sergio
O´Riordan Catherine
M Stanek Lisa
Palermo Adam
Richards Brenda
Song Antonius
Original Assignee
Genzyme Corp
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 Genzyme Corp filed Critical Genzyme Corp
Publication of MX2017010369A publication Critical patent/MX2017010369A/es

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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/08—Antiepileptics; Anticonvulsants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • 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
    • C12N15/86—Viral 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • 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
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
    • 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
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/533—Physical structure partially self-complementary or closed having a mismatch or nick in at least one of the strands
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10022—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10041—Use of virus, viral particle or viral elements as a vector
    • 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
    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10041—Use of virus, viral particle or viral elements as a vector
    • C12N2710/10043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041—Use of virus, viral particle or viral elements as a vector

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Epidemiology (AREA)
  • Psychology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Pain & Pain Management (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Urology & Nephrology (AREA)
  • Vascular Medicine (AREA)
  • Cardiology (AREA)

Abstract

En la presente descripción se proporcionan moléculas de ARNi que incluyen una primera cadena que contiene una secuencia guía y una segunda cadena que comprende una secuencia no guía en donde la secuencia no guía contiene una protuberancia opuesta a la región semilla de las secuencias guía; p. ej., opuesta a la secuencia de escisión. En algunos aspectos, la invención proporciona ARNi para el tratamiento de la enfermedad de Huntington. Adicionalmente se proporcionan en la presente descripción casetes de expresión, vectores (p. ej., vectores de rAAV, adenovirales recombinantes, lentivirales recombinantes, y de HSV recombinantes), células, partículas virales y composiciones farmacéuticas que contienen el ARNi. Asimismo se proporcionan adicionalmente en la presente descripción métodos y kits relacionados con el uso del ARNi, por ejemplo, para el tratamiento de la enfermedad de Huntington.
MX2017010369A 2015-02-10 2016-02-09 Arni variante. MX2017010369A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562114578P 2015-02-10 2015-02-10
PCT/US2016/017207 WO2016130589A2 (en) 2015-02-10 2016-02-09 VARIANT RNAi

Publications (1)

Publication Number Publication Date
MX2017010369A true MX2017010369A (es) 2017-12-14

Family

ID=56614905

Family Applications (2)

Application Number Title Priority Date Filing Date
MX2017010369A MX2017010369A (es) 2015-02-10 2016-02-09 Arni variante.
MX2022013005A MX2022013005A (es) 2015-02-10 2017-08-10 Arni variante.

Family Applications After (1)

Application Number Title Priority Date Filing Date
MX2022013005A MX2022013005A (es) 2015-02-10 2017-08-10 Arni variante.

Country Status (26)

Country Link
US (4) US10450563B2 (es)
EP (1) EP3256588A2 (es)
JP (2) JP2018506304A (es)
KR (1) KR102670852B1 (es)
CN (1) CN107438671B (es)
AR (1) AR103646A1 (es)
AU (2) AU2016219396B2 (es)
BR (1) BR112017017028A2 (es)
CA (1) CA2976075A1 (es)
CL (1) CL2017002027A1 (es)
CO (1) CO2017009083A2 (es)
CR (1) CR20170406A (es)
EA (1) EA201791805A1 (es)
EC (1) ECSP17059343A (es)
HK (1) HK1247641A1 (es)
IL (1) IL253893B (es)
MA (1) MA40819B1 (es)
MX (2) MX2017010369A (es)
MY (2) MY181458A (es)
PE (1) PE20171382A1 (es)
PH (1) PH12017501432A1 (es)
SG (3) SG11201706444TA (es)
TN (1) TN2017000354A1 (es)
TW (1) TWI781079B (es)
UY (1) UY36554A (es)
WO (1) WO2016130589A2 (es)

Families Citing this family (27)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2744987C (en) 2008-12-02 2018-01-16 Chiralgen, Ltd. Method for the synthesis of phosphorus atom modified nucleic acids
BR112012000828A8 (pt) 2009-07-06 2017-10-10 Ontorii Inc Novas pró-drogas de ácido nucleico e métodos de uso das mesmas
WO2012039448A1 (ja) 2010-09-24 2012-03-29 株式会社キラルジェン 不斉補助基
US9605019B2 (en) 2011-07-19 2017-03-28 Wave Life Sciences Ltd. Methods for the synthesis of functionalized nucleic acids
KR20220139425A (ko) 2012-07-13 2022-10-14 웨이브 라이프 사이언시스 리미티드 키랄 제어
KR101835401B1 (ko) 2012-07-13 2018-03-08 신 니뽄 바이오메디칼 라보라토리즈, 엘티디. 키랄 핵산 어쥬번트
AU2013288048A1 (en) 2012-07-13 2015-01-22 Wave Life Sciences Ltd. Asymmetric auxiliary group
US10322173B2 (en) 2014-01-15 2019-06-18 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having anti-allergic activity, and anti-allergic agent
WO2015108048A1 (ja) 2014-01-15 2015-07-23 株式会社新日本科学 抗腫瘍作用を有するキラル核酸アジュバンド及び抗腫瘍剤
EP3095461A4 (en) 2014-01-15 2017-08-23 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having immunity induction activity, and immunity induction activator
PT3094728T (pt) 2014-01-16 2022-05-19 Wave Life Sciences Ltd Desenho quiral
MX2017006216A (es) 2014-11-14 2018-08-29 Voyager Therapeutics Inc Composiciones y métodos para tratar la esclerosis lateral amiotrófica (ela).
US10450563B2 (en) 2015-02-10 2019-10-22 Genzyme Corporation Variant RNAi
MA43072A (fr) 2015-07-22 2018-05-30 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
WO2017068791A1 (ja) * 2015-10-23 2017-04-27 レナセラピューティクス株式会社 少なくとも１つのバルジ構造を有する核酸複合体
MA45270A (fr) 2016-05-04 2017-11-09 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
IL302748A (en) 2016-05-18 2023-07-01 Voyager Therapeutics Inc modulatory polynucleotides
CN109831916B (zh) 2016-05-18 2023-07-21 沃雅戈治疗公司 治疗亨廷顿氏舞蹈病的组合物和方法
US11752181B2 (en) 2017-05-05 2023-09-12 Voyager Therapeutics, Inc. Compositions and methods of treating Huntington's disease
WO2019060726A1 (en) * 2017-09-22 2019-03-28 Genzyme Corporation VARIANT OF ARNI
CA3084985A1 (en) * 2017-12-06 2019-06-13 Ovid Therapeutics Inc. Use of mir101 or mir128 in the treatment of seizure disorders
CA3099306A1 (en) 2018-05-15 2019-11-21 Voyager Therapeutics, Inc. Compositions and methods for the treatment of parkinson's disease
US20210355454A1 (en) 2018-07-24 2021-11-18 Voyager Therapeutics, Inc. Systems and methods for producing gene therapy formulations
WO2020028816A1 (en) * 2018-08-03 2020-02-06 Genzyme Corporation Variant rnai against alpha-synuclein
EP3908658A1 (en) * 2019-01-09 2021-11-17 Universidade de Coimbra Double stranded rna and uses thereof
EP3983077A4 (en) * 2019-06-17 2023-12-20 Alnylam Pharmaceuticals, Inc. DELIVERY OF OLIGONUCLEOTIDES TO THE STRIATUM
JP2023522701A (ja) * 2020-04-20 2023-05-31 ユニバーシティー オブ マサチューセッツ Ｍｓｈ３調節のためのオリゴヌクレオチド

Family Cites Families (29)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2001500015A (ja) 1996-09-06 2001-01-09 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 Ｔ７ポリメラーゼを利用する組換えアデノ随伴ウイルスの誘導可能な製造方法
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6995006B2 (en) 1997-09-05 2006-02-07 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
ES2308989T3 (es) 1999-08-09 2008-12-16 Targeted Genetics Corporation Aumento de la expresion de una secuencia nucleotidica heterologa a partir de vectores viricos recombinantes que contienen una secuencia que forman pares de bases intracatenarios.
CA2410828C (en) 2000-06-01 2012-01-24 University Of North Carolina At Chapel Hill Duplexed parvovirus vectors
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
ATE520707T1 (de) 2001-11-13 2011-09-15 Univ Pennsylvania Verfahren für den nachweis und/oder die identifikation von sequenzen des adeno- assoziierten virus (aav) sowie isolation von dadurch identifizierten, neuen sequenzen
US20080274989A1 (en) * 2002-08-05 2008-11-06 University Of Iowa Research Foundation Rna Interference Suppression of Neurodegenerative Diseases and Methods of Use Thereof
CA2881743A1 (en) 2002-09-25 2004-04-08 University Of Massachusetts In vivo gene silencing by chemically modified and stable sirna
US7589189B2 (en) * 2003-05-14 2009-09-15 Japan Science And Technology Agency Inhibition of the expression of huntingtin gene
WO2006119432A2 (en) 2005-04-29 2006-11-09 The Government Of The U.S.A., As Rep. By The Sec., Dept. Of Health & Human Services Isolation, cloning and characterization of new adeno-associated virus (aav) serotypes
US7902352B2 (en) * 2005-05-06 2011-03-08 Medtronic, Inc. Isolated nucleic acid duplex for reducing huntington gene expression
WO2007120542A2 (en) 2006-03-30 2007-10-25 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid library and aav capsid proteins
CA2688514A1 (en) 2007-04-26 2008-11-06 University Of Iowa Research Foundation Rna interference suppression of neurodegenerative diseases and methods of use thereof
PT2164967E (pt) 2007-05-31 2015-10-27 Univ Iowa Res Found Redução da toxicidade não dirigida do arn de interferência
US20110077283A1 (en) 2008-02-04 2011-03-31 David Frederik Fischer Molecular targets and compounds, and methods to identify the same, useful in the treatment of neurodegenerative diseases
EP2250183A4 (en) * 2008-02-14 2012-07-25 Michael Paul Marie Gantier SMALL IMMUNOSTIMULATORY RNAI MOLECULES
FR2929292A1 (fr) 2008-03-28 2009-10-02 Exonhit Therapeutics S A Sa Procede et methodes de diagnostic de la maladie d'alzheimer
WO2011034811A1 (en) * 2009-09-17 2011-03-24 Sigma-Aldrich Co. Short rna mimetics
CA3066596A1 (en) 2010-04-23 2011-10-27 University Of Massachusetts Cns targeting aav vectors and methods of use thereof
US8663624B2 (en) 2010-10-06 2014-03-04 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
PL2675902T3 (pl) 2011-02-17 2019-08-30 The Trustees Of The University Of Pennsylvania Kompozycje i sposoby do zmieniania specyficzności względem tkanki i poprawy transferu genu z udziałem aav9
CN108285491B (zh) 2012-02-29 2021-08-10 桑格摩生物科学股份有限公司 治疗亨廷顿氏病的方法和组合物
US9550989B2 (en) * 2013-10-03 2017-01-24 Washington University Rational design of microRNA-siRNA chimeras for multi-functional target suppression
FI3628334T3 (fi) 2014-03-21 2023-09-15 Genzyme Corp Geenihoito verkkokalvon pigmenttirappeuma
MX2016014220A (es) 2014-05-02 2017-02-06 Genzyme Corp Vectores de aav para la terapia genica de la retina y el snc.
CA2971920C (en) 2014-12-24 2024-05-07 Uniqure Ip B.V. Rnai induced huntingtin gene suppression
US10450563B2 (en) 2015-02-10 2019-10-22 Genzyme Corporation Variant RNAi
WO2019060726A1 (en) 2017-09-22 2019-03-28 Genzyme Corporation VARIANT OF ARNI

Also Published As

Publication number Publication date
MY181458A (en) 2020-12-22
US11781137B2 (en) 2023-10-10
US20200109401A1 (en) 2020-04-09
PH12017501432A1 (en) 2018-01-15
AU2022204274A1 (en) 2022-07-07
SG10201907396XA (en) 2019-09-27
SG10201912942YA (en) 2020-02-27
US20210047641A1 (en) 2021-02-18
US20180023082A1 (en) 2018-01-25
AU2016219396B2 (en) 2022-03-17
IL253893B (en) 2021-08-31
WO2016130589A2 (en) 2016-08-18
TWI781079B (zh) 2022-10-21
JP2021118740A (ja) 2021-08-12
AU2016219396A1 (en) 2017-09-28
MY194175A (en) 2022-11-17
EA201791805A1 (ru) 2018-05-31
KR20170110149A (ko) 2017-10-10
BR112017017028A2 (pt) 2018-04-10
JP2018506304A (ja) 2018-03-08
US10450563B2 (en) 2019-10-22
AR103646A1 (es) 2017-05-24
IL253893A0 (en) 2017-10-31
CN107438671B (zh) 2021-05-25
UY36554A (es) 2016-09-30
MA40819B1 (fr) 2020-04-30
HK1247641A1 (zh) 2018-09-28
CA2976075A1 (en) 2016-08-18
KR102670852B1 (ko) 2024-05-29
MX2022013005A (es) 2022-11-09
TW201704470A (zh) 2017-02-01
US10760079B2 (en) 2020-09-01
US20240182896A1 (en) 2024-06-06
ECSP17059343A (es) 2019-03-29
PE20171382A1 (es) 2017-09-15
MA40819A1 (fr) 2018-07-31
CL2017002027A1 (es) 2018-04-20
SG11201706444TA (en) 2017-09-28
CR20170406A (es) 2017-11-14
TN2017000354A1 (en) 2019-01-16
CO2017009083A2 (es) 2018-01-31
WO2016130589A3 (en) 2016-09-15
EP3256588A2 (en) 2017-12-20
CN107438671A (zh) 2017-12-05

Similar Documents

Publication Publication Date Title
MX2022013005A (es) Arni variante.
MX2018014152A (es) Composiciones y métodos para tratar la enfermedad de huntington.
PH12020550117A1 (en) Variant rnai
BR112017004349A2 (pt) terapia com gene de globina para o tratamento de hemoglobinopatias
SA519401379B1 (ar) علاج فيروس مرتبط بغدة لمرض هنتنجتون
BR112017005892A2 (pt) variantes de vetor de vírus adeno-associado para edição de genoma de alta eficácia e métodos da mesma
PH12019550076A1 (en) SERPINA1 iRNA COMPOSITIONS AND METHODS OF USE THEREOF
SG10201901482XA (en) Oncolytic adenovirus encoding a b7 protein
WO2016106404A3 (en) Methods and compositions for treating malignant tumors associated with kras mutation
UY36106A (es) Vectores aav para terapia génica retiniana y del snc.
EA201500802A1 (ru) Способы профилактики и/или лечения кардиомиопатии и устранения симптомов кардиомиопатии, фармацевтическая композиция, векторы
CL2017002729A1 (es) Proteína de unión a rgma y su uso
EA201890861A1 (ru) Композиции, содержащие лентивирусные векторы, экспрессирующие il-12, и способы их применения
EA202190474A1 (ru) Терапия на основе микро-рнк, направленная против lcp-1-позитивных раковых опухолей
MX2021005980A (es) Adenovirus y metodos para usar adenovirus.
BR112022006476A2 (pt) Oligonucleotídeos com análogos de nucleosídeos
BR112016010342A2 (pt) sequência de rna isolada ou sintética, sequência de dna, vetor que permite a expressão de uma sequência de rna, célula que compreende uma sequência de rna, composição que compreende uma sequência de rna, utilização de uma sequência de rna, camundongo transgênico, método que permite avaliar a eficácia cosmética de um composto de teste, e, método que permite avaliar a eficácia terapêutica de um composto de teste
MX2021005435A (es) Transgen abcb11 con codones optimizados para el tratamiento de colestasis intrahepatica familiar progresiva tipo 2 (pfic2).
BR112018006480A2 (pt) ?vetor, método de tratamento de uma doença ou condição, composição, combinação de um vetor, kit, uso de um vetor, método para a modulação in vivo da atividade eletrofisiológica de uma célula
DOP2017000185A (es) Arni variante
MX2022001864A (es) Construcciones de arni para inhibir la expresión de slc30a8 y métodos de uso de las mismas.
AR106868A1 (es) Métodos de tratamiento de mieloma múltiple y leucemia de células plasmáticas por medio de terapia de células t