AU2021383834A1 - Compositions and methods for suppressing msut2 - Google Patents

Compositions and methods for suppressing msut2 Download PDF

Info

Publication number
AU2021383834A1
AU2021383834A1 AU2021383834A AU2021383834A AU2021383834A1 AU 2021383834 A1 AU2021383834 A1 AU 2021383834A1 AU 2021383834 A AU2021383834 A AU 2021383834A AU 2021383834 A AU2021383834 A AU 2021383834A AU 2021383834 A1 AU2021383834 A1 AU 2021383834A1
Authority
AU
Australia
Prior art keywords
seq
sirna molecule
sirna
group
sequence selected
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
AU2021383834A
Other languages
English (en)
Other versions
AU2021383834A9 (en
Inventor
Jeremy Baker
Brian Kraemer
Pamela McMillan
Timothy J. Strovas
Jeanna M. Wheeler
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
United States Goverment Represented By Department Of Veterans Affairs AS
Original Assignee
United States Goverment Represented By Department Of Veterans Affairs AS
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 United States Goverment Represented By Department Of Veterans Affairs AS filed Critical United States Goverment Represented By Department Of Veterans Affairs AS
Publication of AU2021383834A1 publication Critical patent/AU2021383834A1/en
Publication of AU2021383834A9 publication Critical patent/AU2021383834A9/en
Pending legal-status Critical Current

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
    • A61K31/21—Esters, e.g. nitroglycerine, selenocyanates
    • A61K31/27—Esters, e.g. nitroglycerine, selenocyanates of carbamic or thiocarbamic acids, meprobamate, carbachol, neostigmine
    • 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
    • A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/445—Non condensed piperidines, e.g. piperocaine
    • 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
    • A61K31/55—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having seven-membered rings, e.g. azelastine, pentylenetetrazole
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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 nucleic acids [NA]
    • 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
    • C12N2320/10—Applications; Uses in screening processes
    • C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Emergency Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
AU2021383834A 2020-11-23 2021-11-22 Compositions and methods for suppressing msut2 Pending AU2021383834A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063117213P 2020-11-23 2020-11-23
US63/117,213 2020-11-23
PCT/US2021/060279 WO2022109376A2 (en) 2020-11-23 2021-11-22 Compositions and methods for suppressing msut2

Publications (2)

Publication Number Publication Date
AU2021383834A1 true AU2021383834A1 (en) 2023-07-06
AU2021383834A9 AU2021383834A9 (en) 2024-06-20

Family

ID=81709733

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2021383834A Pending AU2021383834A1 (en) 2020-11-23 2021-11-22 Compositions and methods for suppressing msut2

Country Status (6)

Country Link
US (1) US20240002848A1 (https=)
EP (1) EP4247394A4 (https=)
JP (1) JP2023551223A (https=)
AU (1) AU2021383834A1 (https=)
CA (1) CA3199887A1 (https=)
WO (1) WO2022109376A2 (https=)

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2001053312A1 (en) * 1999-12-23 2001-07-26 Hyseq, Inc. Novel nucleic acids and polypeptides
US20070009899A1 (en) * 2003-10-02 2007-01-11 Mounts William M Nucleic acid arrays for detecting gene expression in animal models of inflammatory diseases
US8598333B2 (en) * 2006-05-26 2013-12-03 Alnylam Pharmaceuticals, Inc. SiRNA silencing of genes expressed in cancer
KR101704988B1 (ko) * 2009-05-28 2017-02-08 큐알엔에이, 인크. 항바이러스 유전자에 대한 천연 안티센스 전사체의 억제에 의한 항바이러스 유전자 관련된 질환의 치료
WO2012040784A1 (en) * 2010-09-30 2012-04-05 Chipdx Llc Gene marker sets and methods for classification of cancer patients
US11439658B2 (en) * 2018-04-12 2022-09-13 United States Government As Represented By The Department Of Veterans Affairs Compositions and methods for suppressing MSUT2

Also Published As

Publication number Publication date
JP2023551223A (ja) 2023-12-07
EP4247394A4 (en) 2025-10-29
US20240002848A1 (en) 2024-01-04
CA3199887A1 (en) 2022-05-27
AU2021383834A9 (en) 2024-06-20
WO2022109376A3 (en) 2022-07-14
EP4247394A2 (en) 2023-09-27
WO2022109376A2 (en) 2022-05-27

Similar Documents

Publication Publication Date Title
KR101749352B1 (ko) Ｓｉｒｔ1에 대한 천연 안티센스 전사체의 억제에 의해 ｓｉｒｔ1 관련된 질환의 치료
US20130005794A1 (en) Methods and sequences to suppress primate huntington gene expression
KR20240004467A (ko) Tdp-43 단백질병증을 치료하기 위한 조성물 및 방법
US10144930B2 (en) Inhibitors of MYH7B and uses thereof
JP6431480B2 (ja) アルツハイマー病の治療および診断のためのビオチン複合体
EP3430143B1 (en) Inhibitors of srsf1 to treat neurodegenerative disorders
KR20180104075A (ko) IL4Rα, TRPA1, 또는 F2RL1을 표적화하는 RNA 복합체를 사용한 아토피 피부염 및 천식의 치료
TW201202418A (en) Treatment of Methionine Sulfoxide Reductase A (MSRA) related diseases by inhibition of natural antisense transcript to MSRA
CN101454335A (zh) 蛋白质错折叠的治疗
KR20210110310A (ko) 올리고머 핵산 분자 및 그의 이용
EP4054639A1 (en) Exon skipping of fc-epsilon-ri-beta and ms4a6a in combination for the treatment of allergic diseases
US10537591B2 (en) Method for promoting muscle regeneration
KR20200014320A (ko) Apcs의 발현을 억제하는 핵산
AU2021383834A9 (en) Compositions and methods for suppressing msut2
Liu et al. RNAi-based gene therapy for dominant Limb Girdle Muscular Dystrophies
WO2024213890A1 (en) Oligonucleotide decoy
EP1670425A2 (en) Bone morphogenetic protein (bmp) 2a and uses thereof
CN113891940A (zh) 视网膜色素变性的新治疗方法
CN112662671B (zh) 靶向SMN2启动子区甲基化位点nt-290所在区域的反义寡核苷酸
WO2013188824A2 (en) Hexim-1 as a target of leptin signaling to regulate obesity and diabetes
WO2025254047A1 (ja) アンドロゲン受容体 ｒｎａ配列に基づく新規二本鎖ｒｎａ及びその利用
WO2024036343A2 (en) Synergistic nucleic acid based therapeutics and methods of use for treating genetic disorders
WO2025159163A1 (ja) Ａｐｐ ｒｎａ配列に基づく新規二本鎖ｒｎａ及びその利用
US20220259596A1 (en) Inhibitors of microRNA 451a for Treatment of Endometriosis
WO2014148216A1 (ja) 炎症性腸疾患の治療用医薬組成物

Legal Events

Date Code Title Description
SREP Specification republished