CA3236365A1 - Tcf7l2 mediated remyelination in the brain - Google Patents

Tcf7l2 mediated remyelination in the brain Download PDF

Info

Publication number
CA3236365A1
CA3236365A1 CA3236365A CA3236365A CA3236365A1 CA 3236365 A1 CA3236365 A1 CA 3236365A1 CA 3236365 A CA3236365 A CA 3236365A CA 3236365 A CA3236365 A CA 3236365A CA 3236365 A1 CA3236365 A1 CA 3236365A1
Authority
CA
Canada
Prior art keywords
gat
vector
cat
cell
glial progenitor
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
CA3236365A
Other languages
English (en)
French (fr)
Inventor
Steven Goldman
Abdellatif Benraiss
John MARIANI
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 CA3236365A1 publication Critical patent/CA3236365A1/en
Pending legal-status Critical Current

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06Animal cells or tissues; Human cells or tissues
    • C12N5/0602Vertebrate cells
    • C12N5/0618Cells of the nervous system
    • C12N5/0622Glial cells, e.g. astrocytes, oligodendrocytes; Schwann cells
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2506/00Differentiation of animal cells from one lineage to another; Differentiation of pluripotent cells
    • C12N2506/08Differentiation of animal cells from one lineage to another; Differentiation of pluripotent cells from cells of the nervous system

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Genetics & Genomics (AREA)
  • Biotechnology (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Cell Biology (AREA)
  • Microbiology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
CA3236365A 2021-11-02 2022-11-01 Tcf7l2 mediated remyelination in the brain Pending CA3236365A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202163274763P 2021-11-02 2021-11-02
US63/274,763 2021-11-02
US202263378092P 2022-10-03 2022-10-03
US63/378,092 2022-10-03
PCT/US2022/079028 WO2023081633A1 (en) 2021-11-02 2022-11-01 Tcf7l2 mediated remyelination in the brain

Publications (1)

Publication Number Publication Date
CA3236365A1 true CA3236365A1 (en) 2023-05-11

Family

ID=84569688

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3236365A Pending CA3236365A1 (en) 2021-11-02 2022-11-01 Tcf7l2 mediated remyelination in the brain

Country Status (5)

Country Link
US (1) US20230270818A1 (https=)
EP (1) EP4426331A1 (https=)
JP (1) JP2024542015A (https=)
CA (1) CA3236365A1 (https=)
WO (1) WO2023081633A1 (https=)

Family Cites Families (55)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4650764A (en) 1983-04-12 1987-03-17 Wisconsin Alumni Research Foundation Helper cell
US5059421A (en) 1985-07-26 1991-10-22 The Liposome Company, Inc. Preparation of targeted liposome systems of a defined size distribution
US4980289A (en) 1987-04-27 1990-12-25 Wisconsin Alumni Research Foundation Promoter deficient retroviral vector
US5082670A (en) 1988-12-15 1992-01-21 The Regents Of The University Of California Method of grafting genetically modified cells to treat defects, disease or damage or the central nervous system
US5124263A (en) 1989-01-12 1992-06-23 Wisconsin Alumni Research Foundation Recombination resistant retroviral helper cell and products produced thereby
US5328470A (en) 1989-03-31 1994-07-12 The Regents Of The University Of Michigan Treatment of diseases by site-specific instillation of cells or site-specific transformation of cells and kits therefor
US5399346A (en) 1989-06-14 1995-03-21 The United States Of America As Represented By The Department Of Health And Human Services Gene therapy
US5585362A (en) 1989-08-22 1996-12-17 The Regents Of The University Of Michigan Adenovirus vectors for gene therapy
US6497872B1 (en) 1991-07-08 2002-12-24 Neurospheres Holdings Ltd. Neural transplantation using proliferated multipotent neural stem cells and their progeny
US5350674A (en) 1992-09-04 1994-09-27 Becton, Dickinson And Company Intrinsic factor - horse peroxidase conjugates and a method for increasing the stability thereof
EP1024198A3 (en) 1992-12-03 2002-05-29 Genzyme Corporation Pseudo-adenoviral vectors for the gene therapy of haemophiliae
US5631237A (en) 1992-12-22 1997-05-20 Dzau; Victor J. Method for producing in vivo delivery of therapeutic agents via liposomes
FR2705361B1 (fr) 1993-05-18 1995-08-04 Centre Nat Rech Scient Vecteurs viraux et utilisation en thérapie génique.
FR2705686B1 (fr) 1993-05-28 1995-08-18 Transgene Sa Nouveaux adénovirus défectifs et lignées de complémentation correspondantes.
CA2163860A1 (en) 1993-06-30 1995-01-12 Chung C. Hsu Method for preparing liposomes
SK282843B6 (sk) 1993-07-13 2002-12-03 Rhone-Poulenc Rorer S. A. Defektný rekombinantný adenovírus a farmaceutický prostriedok s jeho obsahom
US5885613A (en) 1994-09-30 1999-03-23 The University Of British Columbia Bilayer stabilizing components and their use in forming programmable fusogenic liposomes
IL116816A (en) 1995-01-20 2003-05-29 Rhone Poulenc Rorer Sa Cell for the production of a defective recombinant adenovirus or an adeno-associated virus and the various uses thereof
US5643599A (en) 1995-06-07 1997-07-01 President And Fellows Of Harvard College Intracellular delivery of macromolecules
WO1998011244A2 (en) 1996-09-11 1998-03-19 The Government Of The United States Of America, Represented By The Secretary, Department Of Health And Human Services Aav4 vector and uses thereof
US6245564B1 (en) 1997-01-23 2001-06-12 Cornell Research Foundation, Inc. Method for separating cells
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
WO1999061601A2 (en) 1998-05-28 1999-12-02 The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Aav5 vector and uses thereof
ATE362542T1 (de) 1998-11-05 2007-06-15 Univ Pennsylvania Nukleinsäuresequenzen des adeno-assoziierten virus des serotyps i, und vektoren und wirtszellen, die diese enthalten
AU780231B2 (en) 1998-11-10 2005-03-10 University Of North Carolina At Chapel Hill, The Virus vectors and methods of making and administering the same
HK1047109A1 (zh) 1999-10-15 2003-02-07 University Of Massachusetts 作为指定基因干预工具的rna干预轨迹基因
US6326193B1 (en) 1999-11-05 2001-12-04 Cambria Biosciences, Llc Insect control agent
ATE318923T1 (de) 2000-06-01 2006-03-15 Univ North Carolina Doppelsträngige parvovirus-vektoren
AU2001275474A1 (en) 2000-06-12 2001-12-24 Akkadix Corporation Materials and methods for the control of nematodes
WO2003070171A2 (en) 2002-02-15 2003-08-28 Cornell Research Foundation, Inc. Myelination of congenitally dysmyelinated forebrains using oligodendrocyte progenitor cells
US20040029269A1 (en) 2002-05-07 2004-02-12 Goldman Steven A Promoter-based isolation, purification, expansion, and transplantation of neuronal progenitor cells, oligodendrocyte progenitor cells, or neural stem cells from a population of embryonic stem cells
EP2345731B1 (en) 2003-09-30 2015-10-21 The Trustees of the University of Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof
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
US20100215724A1 (en) 2005-11-22 2010-08-26 Mcgill University Microcapsule Nanotube Devices for Targeted Delivery of Therapeutic Molecules
US8048999B2 (en) 2005-12-13 2011-11-01 Kyoto University Nuclear reprogramming factor
WO2008089267A1 (en) 2007-01-16 2008-07-24 University Of Rochester Non-human animals with human-glial chimeric brains
JP5813321B2 (ja) 2007-03-23 2015-11-17 ウィスコンシン アラムニ リサーチ ファンデーション 体細胞の再プログラム化
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
EP2315829B1 (en) 2008-08-08 2015-02-11 Mayo Foundation For Medical Education And Research Induced pluripotent stem cells
US20100183558A1 (en) 2008-10-17 2010-07-22 Zhennan Lai Safe lentiviral vectors for targeted delivery of multiple therapeutic molecules
WO2011062963A2 (en) 2009-11-17 2011-05-26 Vitro Diagnositics, Inc. Induced puripotent stem cells and related methods
US9005967B2 (en) 2010-01-22 2015-04-14 Kyoto University Myc variants improve induced pluripotent stem cell generation efficiency
CA2853482C (en) 2011-10-28 2020-05-19 Joshua Grieger Cell line for production of adeno-associated virus
EP2900686B1 (en) 2012-09-28 2020-06-10 The University of North Carolina At Chapel Hill Aav vectors targeted to oligodendrocytes
JP6541577B2 (ja) 2013-02-06 2019-07-10 ユニバーシティー オブ ロチェスター ミエリン障害の治療のための誘導多能性細胞由来オリゴデンドロサイト前駆細胞
EP3003391B1 (en) 2013-05-31 2021-09-22 The Regents of The University of California Adeno-associated virus variants and methods of use thereof
IL297919A (en) 2013-07-22 2023-01-01 Childrens Hospital Philadelphia Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues
GB201403684D0 (en) 2014-03-03 2014-04-16 King S College London Vector
MX390848B (es) 2015-06-23 2025-03-21 Childrens Hospital Philadelphia Factor ix modificado, y composiciones, metodos y usos para la transferencia de genes a celulas, organos y tejidos.
RS67119B1 (sr) 2017-11-14 2025-09-30 Arcellx Inc Polipeptidi koji obuhvataju d domen i njihove upotrebe
AU2019222560B2 (en) 2018-02-17 2025-07-03 Flagship Pioneering Innovations V, Inc. Compositions and methods for membrane protein delivery
CA3103675A1 (en) * 2018-06-21 2019-12-26 University Of Rochester Methods of treating or inhibiting onset of huntington's disease
CN112955174A (zh) 2018-07-09 2021-06-11 旗舰先锋创新V股份有限公司 融合剂脂质体组合物和其用途
US20230057355A1 (en) * 2019-02-13 2023-02-23 University Of Rochester Gene networks that mediate remyelination of the human brain
EP4142759A4 (en) 2020-04-28 2024-05-29 Pfizer Inc. MODIFIED NUCLEIC ACIDS ENCODING ASPARTOACYLASE (ASPA) AND VECTOR FOR GENE THERAPY

Also Published As

Publication number Publication date
JP2024542015A (ja) 2024-11-13
EP4426331A1 (en) 2024-09-11
US20230270818A1 (en) 2023-08-31
WO2023081633A1 (en) 2023-05-11

Similar Documents

Publication Publication Date Title
CN111630170B (zh) 眼部疾病的细胞模型及用于眼部疾病的疗法
CN109069544B (zh) 脑内生成gaba能神经元
US20230057355A1 (en) Gene networks that mediate remyelination of the human brain
IL300409A (en) Gene therapies for neurodegenerative disorders
CA3136004A1 (en) Gene therapies for lysosomal disorders
WO2021031810A1 (zh) Ptbp1抑制剂在预防和/或治疗功能性神经元死亡相关的神经系统疾病中的应用
US20230241118A1 (en) Rejuvenation treatment of age-related white matter loss
US11666628B2 (en) Compositions and method for the treatment of x-linked centronuclear myopathy
CA3215353A1 (en) Casrx/cas13d systems targeting c9orf72
JP2023544702A (ja) Tcf4遺伝子発現を調節するため、およびピット・ホプキンス症候群を治療するための組成物および方法
CN113056560B (zh) 能够实现反馈的合成基因、靶点种子匹配盒及其应用
CN114828897A (zh) 细胞活力的调节
WO2021032068A1 (zh) Ptbp1抑制剂在预防和/或治疗功能性神经元死亡相关的神经系统疾病中的应用
US20250281648A1 (en) Use of transdifferentiation of glial cells into neurons in prevention or treatment of diseases associated with neuron loss-of-function or death
US10024870B2 (en) Therapeutic and diagnostic methods and compositions for neurodegenerative diseases
KR20230123925A (ko) Neurod1 및 dlx2 벡터
CA3236365A1 (en) Tcf7l2 mediated remyelination in the brain
US20240392279A1 (en) Modified protein disulfide isomerase and uses thereof
CN120435307A (zh) 在阿尔茨海默病中使用络丝蛋白的组合物和方法
EP4577654A1 (en) Engineered constructs for increased transcription of rna payloads
JP2026505771A (ja) グリア細胞の競合的置換
Pagliari Optimization of AAV9 gene therapy for Spinal Muscular Atrophy with Respiratory Distress type 1 using in vivo disease models
AU2024277300A1 (en) Compositions and methods for treatment and prevention of neurodegenerative diseases and disorders
Abela-Arnold DNAJC6-related Parkinsonism: Disease Characterisation and Development of a Patient-Derived Dopaminergic Neuronal Cell Model
이상환 Effects of AAV-Mediated Delivery of HGF Gene on the Muscular and Nerve Systems in the Nerve Crush and SOD1-G93A Transgenic Mouse Models

Legal Events

Date Code Title Description
P13 Application amended

Free format text: ST27 STATUS EVENT CODE: A-1-1-P10-P13-P146 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: BSL VERIFIED - NO DEFECTS

Effective date: 20241009

MFA Maintenance fee for application paid

Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 2ND ANNIV.) - STANDARD

Year of fee payment: 2

U00 Fee paid

Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED

Effective date: 20241025

U11 Full renewal or maintenance fee paid

Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT DETERMINED COMPLIANT

Effective date: 20241025

Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL

Effective date: 20241025

MFA Maintenance fee for application paid

Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 3RD ANNIV.) - STANDARD

Year of fee payment: 3

U00 Fee paid

Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED

Effective date: 20251024

U11 Full renewal or maintenance fee paid

Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL

Effective date: 20251024