CA3122289A1 - Methods of treating schizophrenia and other neuropsychiatric disorders - Google Patents

Methods of treating schizophrenia and other neuropsychiatric disorders Download PDF

Info

Publication number
CA3122289A1
CA3122289A1 CA3122289A CA3122289A CA3122289A1 CA 3122289 A1 CA3122289 A1 CA 3122289A1 CA 3122289 A CA3122289 A CA 3122289A CA 3122289 A CA3122289 A CA 3122289A CA 3122289 A1 CA3122289 A1 CA 3122289A1
Authority
CA
Canada
Prior art keywords
smad4
subject
glial
cells
scz
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
CA3122289A
Other languages
English (en)
French (fr)
Inventor
Steven A. Goldman
Zhengshan LIU
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.)
University of Rochester
Original Assignee
University of Rochester
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 University of Rochester filed Critical University of Rochester
Publication of CA3122289A1 publication Critical patent/CA3122289A1/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
    • 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
    • C12N15/1138—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 against receptors or cell surface proteins
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/18—Antipsychotics, i.e. neuroleptics; Drugs for mania or schizophrenia
    • 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
    • C12N2310/12—Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • C12N2310/122—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
    • C12N2310/10—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
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Neurosurgery (AREA)
  • Psychiatry (AREA)
  • Neurology (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Medicinal Preparation (AREA)
  • Peptides Or Proteins (AREA)
CA3122289A 2018-12-11 2019-12-11 Methods of treating schizophrenia and other neuropsychiatric disorders Pending CA3122289A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862778145P 2018-12-11 2018-12-11
US62/778,145 2018-12-11
PCT/US2019/065742 WO2020123663A1 (en) 2018-12-11 2019-12-11 Methods of treating schizophrenia and other neuropsychiatric disorders

Publications (1)

Publication Number Publication Date
CA3122289A1 true CA3122289A1 (en) 2020-06-18

Family

ID=69160318

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3122289A Pending CA3122289A1 (en) 2018-12-11 2019-12-11 Methods of treating schizophrenia and other neuropsychiatric disorders

Country Status (7)

Country Link
US (1) US20220025379A1 (https=)
EP (1) EP3894556A1 (https=)
JP (2) JP7518547B2 (https=)
CN (2) CN119925614A (https=)
AU (1) AU2019396450B2 (https=)
CA (1) CA3122289A1 (https=)
WO (1) WO2020123663A1 (https=)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20110011658A (ko) 2008-05-08 2011-02-08 유니버시티 오브 로체스터 최적화된 세포 제제에 의한 수초 질환의 치료
EP2954046B1 (en) 2013-02-06 2025-10-15 University Of Rochester Induced pluripotent cell-derived oligodendrocyte progenitor cells for the treatment of myelin disorders
US9724432B2 (en) 2015-04-30 2017-08-08 University Of Rochester Non-human mammal model of human degenerative disorder, uses thereof, and method of treating human degenerative disorder
JP7457505B2 (ja) 2017-05-10 2024-03-28 ユニバーシティー オブ ロチェスター 神経精神障害を処置する方法
WO2019246262A2 (en) 2018-06-21 2019-12-26 University Of Rochester Methods of treating or inhibiting onset of huntington's disease
CN116350758B (zh) * 2023-03-16 2024-08-06 郑州大学 肿瘤共享新抗原表位肽或其编码核酸在制备药物中的应用

Family Cites Families (25)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
US6794499B2 (en) 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
US6838283B2 (en) 1998-09-29 2005-01-04 Isis Pharmaceuticals Inc. Antisense modulation of survivin expression
CZ296576B6 (cs) 1999-02-12 2006-04-12 Sankyo Company Limited Nukleosidový analog a oligonukleotidový analog a farmaceutický prostredek, sonda a primer s jeho obsahem
US6013787A (en) 1999-02-23 2000-01-11 Isis Pharmaceuticals Inc. Antisense modulation of Smad4 expression
US7098192B2 (en) 1999-04-08 2006-08-29 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of STAT3 expression
US7179796B2 (en) 2000-01-18 2007-02-20 Isis Pharmaceuticals, Inc. Antisense modulation of PTP1B expression
WO2003070918A2 (en) 2002-02-20 2003-08-28 Ribozyme Pharmaceuticals, Incorporated Rna interference by modified short interfering nucleic acid
JP2005504020A (ja) 2001-07-03 2005-02-10 アイシス・ファーマシューティカルス・インコーポレーテッド ヌクレアーゼ耐性キメラオリゴヌクレオチド
AU2003260370B2 (en) 2002-08-05 2008-05-22 Silence Therapeutics Gmbh Further novel forms of interfering RNA molecules
KR100750788B1 (ko) 2003-09-18 2007-08-20 아이시스 파마수티컬즈 인코포레이티드 ｅＩＦ4Ｅ 발현의 조정
AU2005252663B2 (en) 2004-06-03 2011-07-07 Isis Pharmaceuticals, Inc. Double strand compositions comprising differentially modified strands for use in gene modulation
US7128264B2 (en) 2004-07-23 2006-10-31 Symbol Technologies, Inc: Electro-optical reader with improved performance in high intensity ambient light
JP4428296B2 (ja) 2005-06-10 2010-03-10 セイコーエプソン株式会社 表示パネルモジュールおよび表示装置
US9962558B2 (en) * 2005-08-05 2018-05-08 Gholam A. Peyman Methods to regulate polarization and enhance function of cells
BRPI0619794B8 (pt) 2005-12-13 2022-06-14 Univ Kyoto Uso de um fator de reprogramação, agente para a preparação de uma célula-tronco pluripotente induzida a partir de uma célula somática e métodos para preparar uma célula- tronco pluripotente induzida método e para preparar uma célula somática e uso de células-tronco pluripotentes induzidas
DK3399025T3 (da) 2007-03-23 2025-06-16 Wisconsin Alumni Res Found Omprogrammering af somatiske celler
JP2008307007A (ja) 2007-06-15 2008-12-25 Bayer Schering Pharma Ag 出生後のヒト組織由来未分化幹細胞から誘導したヒト多能性幹細胞
US10047346B2 (en) 2008-08-08 2018-08-14 Mayo Foundation For Medical Education And Research Method of treating heart tissue using induced pluripotent stem cells
US20120276070A1 (en) 2009-11-17 2012-11-01 Vitro Diagnositics, Inc. Induced Pluripotent Stem Cells and Related Methods
US9005967B2 (en) 2010-01-22 2015-04-14 Kyoto University Myc variants improve induced pluripotent stem cell generation efficiency
EP2723861A4 (en) * 2011-06-21 2014-12-10 Alnylam Pharmaceuticals Inc COMPOSITIONS AND METHODS FOR INHIBITING HEPCIDINE ANTIMICROBIAL PEPTIDE (HAMP) OR THE ASSOCIATED GENE EXPRESSION
PT2773748T (pt) * 2011-11-04 2020-03-26 Memorial Sloan Kettering Cancer Center Neurónios dopaminérgicos (da) do mesencéfalo para enxerto
US9035039B2 (en) 2011-12-22 2015-05-19 Protiva Biotherapeutics, Inc. Compositions and methods for silencing SMAD4
WO2016115558A2 (en) 2015-01-16 2016-07-21 Bruno Doiron Compositions and methods for creating pancreatic cancer animal model

Also Published As

Publication number Publication date
EP3894556A1 (en) 2021-10-20
CN113728103B (zh) 2025-02-28
JP2022511568A (ja) 2022-01-31
AU2019396450A1 (en) 2021-06-24
AU2019396450B2 (en) 2025-08-28
JP2024129091A (ja) 2024-09-26
WO2020123663A1 (en) 2020-06-18
CN119925614A (zh) 2025-05-06
JP7518547B2 (ja) 2024-07-18
CN113728103A (zh) 2021-11-30
US20220025379A1 (en) 2022-01-27

Similar Documents

Publication Publication Date Title
AU2019396450B2 (en) Methods of treating schizophrenia and other neuropsychiatric disorders
KR102847197B1 (ko) 정신분열증 및 다른 신경정신장애를 치료하는 방법
US20230287411A1 (en) Methods for engineering non-neuronal cells into neurons and using newly engineered neurons to treat neurodegenerative diseases
KR102450757B1 (ko) 안 질환 치료용 안티센스 올리고뉴클레오타이드
US12303549B2 (en) Methods of treating or inhibiting onset of Huntington's disease
JP2021520200A (ja) 非神経細胞のニューロンへの再プログラミング、及び神経変性疾患または障害を治療するための方法と組成物
CN110438125A (zh) 通过抑制脑源神经营养因子(bdnf)的天然反义转录物治疗bdnf相关疾病
CA2804847A1 (en) Sirna targeting vegfa and methods for treatment in vivo
EP4249589A1 (en) In vitro genetic disease model cell and method for producing the same
JP7435942B2 (ja) 神経幹細胞または神経前駆細胞からドーパミン神経細胞への分化方法
JP7834290B2 (ja) 医薬組成物
US20260022377A1 (en) Polynucleotide compositions and methods for treatment of neurodegenerative diseases
JP2025535055A (ja) Ｕｎｃ１３ａアンチセンスオリゴヌクレオチド及びその使用
HK40129245A (zh) Unc13a反义寡核苷酸和其用途
KR20190071930A (ko) 신경줄기세포 또는 신경전구세포에서 도파민 신경세포로의 분화방법

Legal Events

Date Code Title Description
EEER Examination request

Effective date: 20231114

MFA Maintenance fee for application paid

Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 5TH ANNIV.) - STANDARD

Year of fee payment: 5

U00 Fee paid

Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED

Effective date: 20241206

U11 Full renewal or maintenance fee paid

Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT DETERMINED COMPLIANT

Effective date: 20241206

Free format text: ST27 STATUS EVENT CODE: A-2-2-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL

Effective date: 20241206

D15 Examination report completed

Free format text: ST27 STATUS EVENT CODE: A-2-2-D10-D15-D126 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: EXAMINER'S REPORT

Effective date: 20250131

P11 Amendment of application requested

Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P11-P100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: AMENDMENT RECEIVED - RESPONSE TO EXAMINER'S REQUISITION

Effective date: 20250430

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-2-2-W10-W00-W111 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: CORRESPONDENT DETERMINED COMPLIANT

Effective date: 20250430

P11 Amendment of application requested

Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P11-P102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: AMENDMENT DETERMINED COMPLIANT

Effective date: 20250723