CO2023005403A2 - Vector dlx2 - Google Patents

Vector dlx2

Info

Publication number
CO2023005403A2
CO2023005403A2 CONC2023/0005403A CO2023005403A CO2023005403A2 CO 2023005403 A2 CO2023005403 A2 CO 2023005403A2 CO 2023005403 A CO2023005403 A CO 2023005403A CO 2023005403 A2 CO2023005403 A2 CO 2023005403A2
Authority
CO
Colombia
Prior art keywords
dlx2
vector
neurons
compositions
conversion
Prior art date
Application number
CONC2023/0005403A
Other languages
English (en)
Inventor
Jie Xu
Original Assignee
Neuexcell Therapeutics Inc
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 Neuexcell Therapeutics Inc filed Critical Neuexcell Therapeutics Inc
Publication of CO2023005403A2 publication Critical patent/CO2023005403A2/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
    • 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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • 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
    • A61P25/28—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
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
    • 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
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/80—Vector systems having a special element relevant for transcription from vertebrates
    • C12N2830/85—Vector systems having a special element relevant for transcription from vertebrates mammalian

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)

Abstract

La presente divulgación se refiere a vectores AAV, composiciones y métodos relacionados con la conversión de neurogliocitos en neuronas mediante el uso de una secuencia codificante de Dlx2 en un vector AAV.
CONC2023/0005403A 2020-09-29 2023-04-27 Vector dlx2 CO2023005403A2 (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063084927P 2020-09-29 2020-09-29
US202163247417P 2021-09-23 2021-09-23
PCT/US2021/052302 WO2022072310A1 (en) 2020-09-29 2021-09-28 Dlx2 vector

Publications (1)

Publication Number Publication Date
CO2023005403A2 true CO2023005403A2 (es) 2023-08-18

Family

ID=80931164

Family Applications (1)

Application Number Title Priority Date Filing Date
CONC2023/0005403A CO2023005403A2 (es) 2020-09-29 2023-04-27 Vector dlx2

Country Status (12)

Country Link
US (1) US20220106614A1 (es)
EP (1) EP4221835A1 (es)
JP (1) JP2023543360A (es)
KR (1) KR20230123926A (es)
AU (1) AU2021352917A1 (es)
CA (1) CA3197320A1 (es)
CL (1) CL2023000922A1 (es)
CO (1) CO2023005403A2 (es)
IL (1) IL301747A (es)
MX (1) MX2023003656A (es)
PE (1) PE20231569A1 (es)
WO (1) WO2022072310A1 (es)

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6897061B1 (en) * 2000-06-16 2005-05-24 Spinal Cord Society Transdifferentiation of glial cells
US6602680B2 (en) * 2000-07-14 2003-08-05 The Regents Of The University Of California Production of gabaergic cells
US9388427B2 (en) * 2002-12-02 2016-07-12 Biovec, Llc In vivo and ex vivo gene transfer into renal tissue using gutless adenovirus vectors
EP1995309A1 (en) * 2007-05-21 2008-11-26 Vivalis Recombinant protein production in avian EBx® cells
CA2725953A1 (en) * 2007-05-29 2008-12-11 Christopher B. Reid Methods for production and uses of multipotent cell populations, pluripotent cell populations, neuronal cell populations, and muscle cell populations
WO2013071440A1 (en) * 2011-11-18 2013-05-23 UNIVERSITé LAVAL Methods and products for increasing frataxin levels and uses thereof
WO2014003553A1 (en) * 2012-06-27 2014-01-03 Arthrogen B.V. Combination for treating an inflammatory disorder
EP3233131A1 (en) * 2014-12-16 2017-10-25 Board of Regents of the University of Nebraska Gene therapy for juvenile batten disease
MA41346A (fr) * 2015-01-12 2017-11-21 Juno Therapeutics Inc Eléments régulateurs post-transcriptionnels d'hépatite modifiée
US9840542B2 (en) * 2015-09-11 2017-12-12 Nomadogen Biotechnologies Inc. Methods and compositions for the packaging of nucleic acids into microglial exosomes for the targeted expression of polypeptides in neural cells
CN116763942A (zh) * 2016-02-18 2023-09-19 宾州研究基金会 脑内生成gaba能神经元

Also Published As

Publication number Publication date
WO2022072310A1 (en) 2022-04-07
US20220106614A1 (en) 2022-04-07
CA3197320A1 (en) 2022-04-07
MX2023003656A (es) 2023-06-22
JP2023543360A (ja) 2023-10-13
CL2023000922A1 (es) 2023-11-17
EP4221835A1 (en) 2023-08-09
AU2021352917A1 (en) 2023-05-25
IL301747A (en) 2023-05-01
KR20230123926A (ko) 2023-08-24
PE20231569A1 (es) 2023-10-04

Similar Documents

Publication Publication Date Title
BR112021026376A2 (pt) Proteínas de fusão flt3l-fc e métodos de uso
Erdös The dimension of the rational points in Hilbert space
MX2022000893A (es) Metodo de obtencion de mitocondrias de celulas y mitocondrias obtenidas.
WO2021257730A3 (en) Cells modified by a cas12i polypeptide
AR100606A1 (es) Variantes de lipasas y polinucleótidos que las codifican
AR110093A1 (es) D-psicosa 3-epimerasa y método para preparar d-psicosa utilizando la misma
WO2019094447A3 (en) Production of mycosporine-like amino acids in cyanobacteria
WO2018120843A9 (zh) 一种三功能分子及其应用
AR073492A1 (es) Metodo para dar forma al cabello
CO2023005403A2 (es) Vector dlx2
WO2020259721A3 (zh) Mcu随机间隔转换电桥电压的真随机数产生器及产生方法
CO2023005298A2 (es) Vector neurod1
CO2023005303A2 (es) Vector neurod1 y dlx2
EA202190876A1 (ru) Композиции, содержащие бактериальные штаммы
TR201914163A2 (tr) Epokside doğal kauçuk içeren, yüksek yapışma düzeyine sahip ve dayanımı artırılmış kuşingam hamuru.
RU2011113233A (ru) Способ и устройство для выполнения цифровой криптографической функции
AU2018233017A1 (en) Tarpaulin solar energy generation system and solar tent
ECSP23086870A (es) Un fab anti-tslp con estabilidad mejorada
WO2018039100A3 (en) Stem cell-produced microvesicles for treating tendon pathologies
Plotkin Action of endomorphism semigroups on definable sets
JP1724041S (ja) 工業用グリップ
Saidova EXPORT POTENTIAL OF HYDROPOWER OF THE REPUBLIC OF TAJIKISTAN AND THE EFFICIENCY OF ITS IMPLEMENTATION
Mamontov Recognizing small finite simple groups by element orders in the class of all groups
JP1750036S (ja) 電解質を用いた歯ブラシ
Péter Korai vízerőművek a Hegyi-Bánságban: Early hydro plants in West-Transsylvania