MX2022004524A - Tratamiento génico para la enfermedad de alzheimer. - Google Patents

Tratamiento génico para la enfermedad de alzheimer.

Info

Publication number
MX2022004524A
MX2022004524A MX2022004524A MX2022004524A MX2022004524A MX 2022004524 A MX2022004524 A MX 2022004524A MX 2022004524 A MX2022004524 A MX 2022004524A MX 2022004524 A MX2022004524 A MX 2022004524A MX 2022004524 A MX2022004524 A MX 2022004524A
Authority
MX
Mexico
Prior art keywords
disease
alzheimer
gene therapy
apoe4
mammal
Prior art date
Application number
MX2022004524A
Other languages
English (en)
Spanish (es)
Inventor
Ronald G Crystal
Katie Stiles
Original Assignee
Univ Cornell
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 Univ Cornell filed Critical Univ Cornell
Publication of MX2022004524A publication Critical patent/MX2022004524A/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
    • C12N15/63—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
    • 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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal 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
    • 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
    • A61K48/005—Medicinal 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • 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
    • C07K14/775—Apolipopeptides
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/05—Animals comprising random inserted nucleic acids (transgenic)
    • A01K2217/052—Animals comprising random inserted nucleic acids (transgenic) inducing gain of function
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/0306—Animal model for genetic diseases
    • A01K2267/0312—Animal model for Alzheimer's disease
    • 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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141—MicroRNAs, miRNAs
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/31—Combination therapy
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Toxicology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Virology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Hospice & Palliative Care (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Psychiatry (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
MX2022004524A 2019-10-16 2020-10-16 Tratamiento génico para la enfermedad de alzheimer. MX2022004524A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962915988P 2019-10-16 2019-10-16
PCT/US2020/056051 WO2021076941A1 (en) 2019-10-16 2020-10-16 Gene therapy for alzheimer's disease

Publications (1)

Publication Number Publication Date
MX2022004524A true MX2022004524A (es) 2022-07-21

Family

ID=73288693

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2022004524A MX2022004524A (es) 2019-10-16 2020-10-16 Tratamiento génico para la enfermedad de alzheimer.

Country Status (13)

Country Link
US (1) US20230405148A1 (https=)
EP (1) EP4045653A1 (https=)
JP (2) JP2023500793A (https=)
KR (1) KR20220082050A (https=)
CN (1) CN114761569A (https=)
AU (2) AU2020367437B2 (https=)
BR (1) BR112022007010A2 (https=)
CA (1) CA3157864A1 (https=)
CO (1) CO2022006367A2 (https=)
IL (1) IL292148A (https=)
MX (1) MX2022004524A (https=)
WO (1) WO2021076941A1 (https=)
ZA (1) ZA202205344B (https=)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP7616995B2 (ja) * 2018-11-28 2025-01-17 プリベイル セラピューティクス，インコーポレーテッド 神経変性疾患のための遺伝子治療
EP4065600A1 (en) 2019-11-25 2022-10-05 Cornell University Apoe gene therapy
JP2025513066A (ja) * 2022-04-12 2025-04-22 ユニキュアー バイオファーマ ビー．ブイ． Ａｐｏｅの核酸制御
EP4551711A1 (en) * 2022-07-08 2025-05-14 Cornell University Methods and pharmaceutical compositions for the treatment and the prevention of alzheimers disease
WO2025213111A2 (en) * 2024-04-05 2025-10-09 Cornell University Aav expression of mirna to suppress human apoe mrna

Family Cites Families (34)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4797368A (en) 1985-03-15 1989-01-10 The United States Of America As Represented By The Department Of Health And Human Services Adeno-associated virus as eukaryotic expression vector
US4863457A (en) 1986-11-24 1989-09-05 Lee David A Drug delivery device
US5378475A (en) 1991-02-21 1995-01-03 University Of Kentucky Research Foundation Sustained release drug delivery devices
AU4198793A (en) 1992-07-24 1994-01-27 Takeda Chemical Industries Ltd. Microparticle preparation and production thereof
FR2695563B1 (fr) 1992-09-11 1994-12-02 Pasteur Institut Microparticules portant des antigènes et leur utilisation pour l'induction de réponses humorales ou cellulaires.
ES2113094T3 (es) 1993-03-09 1998-04-16 Epic Therapeutics Inc Las microparticulas macromoleculares y metodos de obtencion.
US5981719A (en) 1993-03-09 1999-11-09 Epic Therapeutics, Inc. Macromolecular microparticles and methods of production and use
US6090925A (en) 1993-03-09 2000-07-18 Epic Therapeutics, Inc. Macromolecular microparticles and methods of production and use
US5603960A (en) 1993-05-25 1997-02-18 O'hagan; Derek T. Preparation of microparticles and method of immunization
US5464758A (en) 1993-06-14 1995-11-07 Gossen; Manfred Tight control of gene expression in eucaryotic cells by tetracycline-responsive promoters
US5814618A (en) 1993-06-14 1998-09-29 Basf Aktiengesellschaft Methods for regulating gene expression
US6913767B1 (en) 1993-10-25 2005-07-05 Genentech, Inc. Compositions for microencapsulation of antigens for use as vaccines
US5443505A (en) 1993-11-15 1995-08-22 Oculex Pharmaceuticals, Inc. Biocompatible ocular implants
US6342390B1 (en) 1994-11-23 2002-01-29 The United States Of America As Represented By The Secretary Of Health And Human Services Lipid vesicles containing adeno-associated virus rep protein for transgene integration and gene therapy
EP0930874A2 (en) 1996-10-09 1999-07-28 Takeda Chemical Industries, Ltd. A method for producing a microparticle
US6210707B1 (en) 1996-11-12 2001-04-03 The Regents Of The University Of California Methods of forming protein-linked lipidic microparticles, and compositions thereof
US6884435B1 (en) 1997-01-30 2005-04-26 Chiron Corporation Microparticles with adsorbent surfaces, methods of making same, and uses thereof
US6406719B1 (en) 1998-05-13 2002-06-18 Microbiological Research Authority Encapsulation of bioactive agents
GB9810236D0 (en) 1998-05-13 1998-07-08 Microbiological Res Authority Improvements relating to encapsulation of bioactive agents
US6264987B1 (en) 2000-05-19 2001-07-24 Alkermes Controlled Therapeutics Inc. Ii Method for preparing microparticles having a selected polymer molecular weight
FR2814642B1 (fr) 2000-10-03 2005-07-01 Ass Pour Le Dev De La Rech En Souris transgenique pour la recombinaison ciblee mediee par la cre-er modifiee
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
WO2005116204A1 (ja) * 2004-05-11 2005-12-08 Rnai Co., Ltd. Ｒｎａ干渉を生じさせるポリヌクレオチド、および、これを用いた遺伝子発現抑制方法
US20070081972A1 (en) 2005-09-30 2007-04-12 The University Of Iowa Research Foundation Polymer-based delivery system for immunotherapy of cancer
US20070248599A1 (en) * 2006-02-21 2007-10-25 Jordan Tang Treatment of alzheimer's disease with inhibitors of apoe binding to apoe receptor
EP2114153B1 (en) * 2007-02-06 2016-11-23 Tai June Yoo Treatment and prevention of neurodegenerative diseases using gene therapy
WO2012054425A2 (en) 2010-10-18 2012-04-26 University Of Iowa Research Foundation Biodegradable particulate formulations
US9345776B2 (en) 2011-02-24 2016-05-24 The University Of Iowa Research Foundation Biodegradable polymers with sulfenamide bonds for drug delivery applications
CN104540952A (zh) * 2012-05-18 2015-04-22 衣阿华大学研究基金会 用于治疗淀粉状蛋白沉积的方法和组合物
WO2013181618A2 (en) * 2012-05-31 2013-12-05 The Trustees Of Columbia University In The City Of New York Methods to treat alzheimer's disease using apoe inhibitors
IL245703B (en) * 2013-11-20 2022-07-01 Univ Iowa Res Found Methods and compositions for treating amyloid deposits
JP7610923B2 (ja) * 2016-09-02 2025-01-09 スパーク セラピューティクス インコーポレイテッド Ｃｎｓ障害を治療するための方法及びベクター
US11071791B2 (en) * 2018-01-26 2021-07-27 Wisconsin Alumni Research Foundation Vector for gene silencing and replacement and methods of use thereof
JP7616995B2 (ja) * 2018-11-28 2025-01-17 プリベイル セラピューティクス，インコーポレーテッド 神経変性疾患のための遺伝子治療

Also Published As

Publication number Publication date
ZA202205344B (en) 2023-03-29
WO2021076941A1 (en) 2021-04-22
IL292148A (en) 2022-06-01
CA3157864A1 (en) 2021-04-22
BR112022007010A2 (pt) 2022-07-12
WO2021076941A9 (en) 2021-10-07
JP2023500793A (ja) 2023-01-11
CO2022006367A2 (es) 2022-08-09
US20230405148A1 (en) 2023-12-21
AU2020367437A1 (en) 2022-06-02
EP4045653A1 (en) 2022-08-24
KR20220082050A (ko) 2022-06-16
CN114761569A (zh) 2022-07-15
AU2025200812A1 (en) 2025-02-27
AU2020367437B2 (en) 2024-11-07
JP2026041903A (ja) 2026-03-10

Similar Documents

Publication Publication Date Title
MX2022004524A (es) Tratamiento génico para la enfermedad de alzheimer.
PH12020551425A1 (en) Rimegepant for cgrp related disorders
PH12021552763A1 (en) Treatment and prevention of metabolic diseases
PH12020552135A1 (en) Kdm1a inhibitors for the treatment of disease
MX2021015427A (es) Vesiculas extracelulares microbianas procesadas.
PH12019501010A1 (en) Arginase inhibitor combination therapies
WO2016106403A3 (en) Therapeutic compositions and methods for malignant tumors with rnai molecules targeted to hsp47 and p21
WO2019246262A3 (en) Methods of treating or inhibiting onset of huntington's disease
WO2021183318A3 (en) Methods and compositions relating to improved combination therapies
MX2025005489A (es) Uso de lumateperona para el tratamiento del trastorno bipolar ii
MX2020008680A (es) Terapia de combinacion con apilimod y agentes glutamatergicos.
EP2294184A4 (en) TREATMENT OF EYE DISEASES AND EXCESSIVE NEOVASCULARIZATION WITH THE HELP OF COMBINED THERAPY
ZA202110111B (en) Methods and compositions for treating liver disorders
MY179105A (en) Methods of treating alzheimer's disease
BR112022002609A2 (pt) Métodos de tratamento de tumores estromais gastrointestinais
GB2503852A (en) Compounds for the treatment of neuropsychiatric disorders
PH12021551036A1 (en) Anti-liv1 immune cell cancer therapy
EP4484443A3 (en) Materials and methods for treatment of friedreich ataxia and other related disorders
PH12023552830A1 (en) Compositions and methods for inhibiting ketohexokinase
MX2022006533A (es) Un esteroide c21-n-pirazolilo 19-nor c3,3-disustituido y metodos de uso del mismo.
WO2022031847A3 (en) Compositions and methods for inhibiting plp1 expression
EP4289820A3 (en) Compounds for use in treating alzheimer's disease in apoe4+/+ patients
EP4286013A3 (en) Lrrc33 inhibitors and use thereof
MX2021008081A (es) Tratamiento de la enfermedad de sjogren con proteinas de fusion de nucleasa.
MX2019000677A (es) Células miméticas de células b.