AR070000A1 - Terapia genica para enfermedades de almacenamiento lisosomal - Google Patents

Terapia genica para enfermedades de almacenamiento lisosomal

Info

Publication number
AR070000A1
AR070000A1 ARP080102421A ARP080102421A AR070000A1 AR 070000 A1 AR070000 A1 AR 070000A1 AR P080102421 A ARP080102421 A AR P080102421A AR P080102421 A ARP080102421 A AR P080102421A AR 070000 A1 AR070000 A1 AR 070000A1
Authority
AR
Argentina
Prior art keywords
alpha
acid
beta
viral vector
transgene
Prior art date
Application number
ARP080102421A
Other languages
English (en)
Inventor
James Dodge
Seng H Cheng
Original Assignee
Genzyme Corp
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 Genzyme Corp filed Critical Genzyme Corp
Publication of AR070000A1 publication Critical patent/AR070000A1/es

Links

Classifications

    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • 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
    • 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
    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01045—Glucosylceramidase (3.2.1.45), i.e. beta-glucocerebrosidase

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Obesity (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)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Enzymes And Modification Thereof (AREA)

Abstract

Se proporciona métodos y composiciones para tratar enfermedades de almacenamiento lisosomal en un sujeto. En un aspecto, se suministra un producto transgénico a un sujeto mediante la administracion al cerebro de un vector viral neurotrofico recombinante que contiene el transgén. El vector viral lleva el transgén hacia una region del cerebro que es susceptible a la infeccion por el virus y que expresa el producto génico viral recombinante codificado. También se proporcionan composiciones para el suministro de un producto transgénico a un sujeto mediante la administracion al cerebro del sujeto de un vector viral neurotrofico recombinante que contiene el transgén. El producto transgénico puede ser cualquiera que sea deficiente en una enfermedad de almacenamiento lisosomal. Reivindicacion 2: El método de la reivindicacion 1, caracterizado porque el vector viral es un vector AAV. Reivindicacion 3: El método de la reivindicacion 1, caracterizado porque el vector viral es AAV4. Reivindicacion 4: El método de la reivindicacion 1, caracterizado porque el producto transgénico es esfingomielinasa ácida (aSM). Reivindicacion 13: El método de la reivindicacion 10, caracterizado porque el transgén es seleccionado del grupo formado por aspartilglucosaminidasa, alfa-qalactosidasa A, palmitoilproteína tioesterasa, tripeptidilpeptidasa, proteína de la transmembrana lisosomal, productos génicos multiples, transportador de cisteína, ceramidasa ácida , alfa-L-fucosidasa ácida, proteína/catepsina A protectoras, beta-glucosidasa ácida, o, beta-galactosidasa ácida, iduronato-2-sulfatasa, alfa-L-iduronidasa, galactocerebrosidasa, alfa-mannosidasa ácida, beta-mannosidasa ácida, arilsulfatasa B, arilsulfatasa A, N-acetilgalactosamina-6-sulfato, beta-galactosidasa ácida, N-acetilglucosamina-1, esfingomielinasa ácida, NPC-1, alfa-glucosidasa, beta-hexosaminidasa B, heparan N-sulfatasa, alfa-N-acetilglucosaminidasa, acetil-CoA: alfa-glucosaminida, N-acetilglucosamina-6-sulfato, alfa-N-acetilgalactosaminidasa, alfa-N-acetilgalactosaminidasa, alfa-neuramidasa, beta-glucuronidasa, beta-hexosaminidasa A y lipasa ácida.
ARP080102421A 2007-06-06 2008-06-06 Terapia genica para enfermedades de almacenamiento lisosomal AR070000A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US92494707P 2007-06-06 2007-06-06

Publications (1)

Publication Number Publication Date
AR070000A1 true AR070000A1 (es) 2010-03-10

Family

ID=40130114

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP080102421A AR070000A1 (es) 2007-06-06 2008-06-06 Terapia genica para enfermedades de almacenamiento lisosomal

Country Status (14)

Country Link
US (2) US8796236B2 (es)
EP (2) EP2158322B1 (es)
AR (1) AR070000A1 (es)
CY (1) CY1119295T1 (es)
DK (1) DK2158322T3 (es)
ES (2) ES2905616T3 (es)
HR (1) HRP20171165T1 (es)
HU (1) HUE035779T2 (es)
IL (3) IL202522A (es)
LT (1) LT2158322T (es)
PL (2) PL3252161T3 (es)
PT (2) PT3252161T (es)
SI (1) SI2158322T1 (es)
WO (1) WO2008154198A1 (es)

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DK1988823T3 (en) 2006-02-09 2018-12-03 Genzyme Corp SLOW INTRAVENTRICULAR ADMINISTRATION
PL3252161T3 (pl) 2007-06-06 2022-03-07 Genzyme Corporation Terapia genowa dla lizosomalnych chorób spichrzeniowych
US9217155B2 (en) 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
US8242086B2 (en) * 2008-11-12 2012-08-14 Duke University Methods and compositions for treating disorders caused by a deficiency in a gene product of a CLN gene
DK2424571T3 (da) 2009-04-30 2020-05-04 Ospedale San Raffaele Srl Genvektor
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
CA3049237C (en) 2010-04-23 2024-06-11 University Of Massachusetts Cns targeting aav vectors and methods of use thereof
CA3050894C (en) 2010-04-23 2022-10-18 University Of Massachusetts Multicistronic expression constructs
EP3546584B1 (en) 2011-03-31 2024-02-14 University of Iowa Research Foundation Compositions for treating lysosmal storage disease (lsd)
US20130090374A1 (en) * 2011-04-20 2013-04-11 Miguel Sena-Esteves Methods for the treatment of tay-sachs disease, sandhoff disease, and gm1-gangliosidosis
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US20210228739A1 (en) * 2018-05-15 2021-07-29 University Of Massachusetts Raav vectors encoding of lysosomal beta-galactosidase (glb1) and cathepsin a
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MX2021010266A (es) * 2019-02-26 2021-09-23 Univ Pennsylvania Composiciones utiles en el tratamiento de la enfermedad de krabbe.
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Also Published As

Publication number Publication date
IL252898A0 (en) 2017-08-31
DK2158322T3 (en) 2017-08-28
IL266734B (en) 2021-10-31
PT2158322T (pt) 2017-08-09
PL2158322T3 (pl) 2017-10-31
CY1119295T1 (el) 2018-02-14
US11369693B2 (en) 2022-06-28
EP3252161A1 (en) 2017-12-06
IL266734A (en) 2019-07-31
PL3252161T3 (pl) 2022-03-07
US20100173979A1 (en) 2010-07-08
EP2158322A4 (en) 2011-06-01
US8796236B2 (en) 2014-08-05
EP3252161B1 (en) 2021-11-17
LT2158322T (lt) 2017-08-25
IL202522A0 (en) 2011-08-01
EP2158322A1 (en) 2010-03-03
HRP20171165T1 (hr) 2017-12-15
PT3252161T (pt) 2022-02-01
HUE035779T2 (en) 2018-05-28
EP2158322B1 (en) 2017-05-03
ES2905616T3 (es) 2022-04-11
ES2635726T3 (es) 2017-10-04
IL252898B (en) 2019-06-30
US20150151007A1 (en) 2015-06-04
WO2008154198A1 (en) 2008-12-18
IL202522A (en) 2017-10-31
SI2158322T1 (sl) 2017-10-30

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