MX394648B - Composiciones que contienen vectores modificados de virus adeno-asociados para usarse en el tratamiento de esclerosis lateral amiotrófica. - Google Patents

Composiciones que contienen vectores modificados de virus adeno-asociados para usarse en el tratamiento de esclerosis lateral amiotrófica.

Info

Publication number
MX394648B
MX394648B MX2018011744A MX2018011744A MX394648B MX 394648 B MX394648 B MX 394648B MX 2018011744 A MX2018011744 A MX 2018011744A MX 2018011744 A MX2018011744 A MX 2018011744A MX 394648 B MX394648 B MX 394648B
Authority
MX
Mexico
Prior art keywords
cdna insert
aav
factor
cntfra
clf
Prior art date
Application number
MX2018011744A
Other languages
English (en)
Other versions
MX2018011744A (es
Inventor
Alexander John Maclennan
Original Assignee
Univ Cincinnati
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 Cincinnati filed Critical Univ Cincinnati
Publication of MX2018011744A publication Critical patent/MX2018011744A/es
Publication of MX394648B publication Critical patent/MX394648B/es

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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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular 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
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/71—Receptors; Cell surface antigens; Cell surface determinants for growth factors; for growth regulators
    • 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
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/715—Receptors; Cell surface antigens; Cell surface determinants for cytokines; for lymphokines; for interferons
    • 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
    • A01K2267/0318—Animal model for neurodegenerative disease, e.g. non- Alzheimer's
    • 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)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Biophysics (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Neurology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • General Chemical & Material Sciences (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Cell Biology (AREA)
  • Immunology (AREA)
  • Toxicology (AREA)
  • Wood Science & Technology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Engineering & Computer Science (AREA)
  • Neurosurgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Hospice & Palliative Care (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Psychiatry (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Orthopedic Medicine & Surgery (AREA)

Abstract

La presente invención se refiere a un vector de virus adeno-asociado modificado (AAV) que empaca un genoma basado en AAV recombinante (rAVV) para usarse en el tratamiento de un trastorno degenerativo de las neuronas motoras, en donde el vector de virus adeno-asociado modificado (AAV) que empaca un genoma basado en AAV recombinante ( rAVV) está adaptado para ser administrable al sujeto que padece el trastorno degenerativo de las neuronas motoras, en donde el genoma basado en r AAV comprende un inserto de ADNc del receptor de factor neurotrófico ciliar alfa (CNTRF¿), y en donde el uso comprende la regulación por incremento de la expresión de CNTR¿ ARN en el músculo esquelético
MX2018011744A 2016-03-31 2017-03-31 Composiciones que contienen vectores modificados de virus adeno-asociados para usarse en el tratamiento de esclerosis lateral amiotrófica. MX394648B (es)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201662315988P 2016-03-31 2016-03-31
US201662433987P 2016-12-14 2016-12-14
US201662433985P 2016-12-14 2016-12-14
PCT/US2017/025315 WO2017173234A1 (en) 2016-03-31 2017-03-31 Methods and compositions for the treatment of als

Publications (2)

Publication Number Publication Date
MX2018011744A MX2018011744A (es) 2019-05-30
MX394648B true MX394648B (es) 2025-03-24

Family

ID=59965184

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2018011744A MX394648B (es) 2016-03-31 2017-03-31 Composiciones que contienen vectores modificados de virus adeno-asociados para usarse en el tratamiento de esclerosis lateral amiotrófica.

Country Status (4)

Country Link
EP (2) EP4008356A1 (es)
CA (1) CA3018224A1 (es)
MX (1) MX394648B (es)
WO (1) WO2017173234A1 (es)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4025258A4 (en) 2019-09-03 2023-09-06 University of Cincinnati METHODS AND COMPOSITIONS FOR THE TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP4301347B2 (ja) * 1996-03-14 2009-07-22 ジェネンテク， インコーポレイテッド Ｇｄｎｆおよびｇｄｎｆ受容体の用途
FR2753379B1 (fr) * 1996-09-13 1998-10-30 Rhone Poulenc Rorer Sa Methode de traitement de la sclerose laterale amyotrophique
JP2002531418A (ja) 1998-12-03 2002-09-24 アビジェン， インコーポレイテッド アデノ随伴ウイルスの薬学処方物における使用のための賦形剤、およびそれにより構成される薬学処方物
FR2804435A1 (fr) * 2000-01-27 2001-08-03 Pf Medicament COMPLEXE ISOLE COMPRENANT UNE PROTEINE NNT-1 ET EN OUTRE AU MOINS UNE PROTEINE CLF-1 ET/OU UNE PROTEINE sCNTFRalpha
EP1290206A2 (en) * 2000-06-09 2003-03-12 University of Florida Recombinant aav vectors for gene therapy of obesity
DK2826860T3 (en) * 2010-04-23 2018-12-03 Univ Massachusetts CNS targeting AAV vectors and methods for their use
EP4410805A3 (en) * 2014-03-18 2024-11-27 University of Massachusetts Raav-based compositions and methods for treating amyotrophic lateral sclerosis

Also Published As

Publication number Publication date
WO2017173234A1 (en) 2017-10-05
CA3018224A1 (en) 2017-10-05
MX2018011744A (es) 2019-05-30
EP3436078A4 (en) 2019-10-16
EP3436078A1 (en) 2019-02-06
EP3436078B1 (en) 2021-10-27
EP4008356A1 (en) 2022-06-08

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