ES2629380T3 - Viriones de AAV con inmunorreactividad reducida y usos de los mismos - Google Patents

Viriones de AAV con inmunorreactividad reducida y usos de los mismos Download PDF

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Publication number
ES2629380T3
ES2629380T3 ES13163182T ES13163182T ES2629380T3 ES 2629380 T3 ES2629380 T3 ES 2629380T3 ES 13163182 T ES13163182 T ES 13163182T ES 13163182 T ES13163182 T ES 13163182T ES 2629380 T3 ES2629380 T3 ES 2629380T3
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Spain
Prior art keywords
aav
amino acid
aav virions
virions
reduced immunoreactivity
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Expired - Lifetime
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ES13163182T
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Alejandra Elena Arbetman
Peter C. Colosi
Michael A Lochrie
Richard T Surosky
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Genzyme Corp
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Genzyme Corp
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    • 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
    • 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)
    • A61K38/48—Hydrolases (3) acting on peptide bonds (3.4)
    • A61K38/482—Serine endopeptidases (3.4.21)
    • A61K38/4846—Factor VII (3.4.21.21); Factor IX (3.4.21.22); Factor Xa (3.4.21.6); Factor XI (3.4.21.27); Factor XII (3.4.21.38)
    • 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
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    • 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
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    • 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
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    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
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    • 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
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    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/21—Serine endopeptidases (3.4.21)
    • C12Y304/21022—Coagulation factor IXa (3.4.21.22)
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
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    • C12N2750/14011—Parvoviridae
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    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • C12N2830/80—Vector systems having a special element relevant for transcription from vertebrates
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Abstract

Uso de una proteína de la cápside de virus adeno-asociado (AAV) recombinante, en un virión de AAV recombinante, para reducir la inmunorreactividad de los viriones en comparación con los correspondientes viriones de tipo salvaje, en donde dicha proteína de la cápside de AAV mutado comprende una sustitución del aminoácido que se produce en una posición correspondiente a la posición en la cápside VPA de AAV-2 seleccionada del grupo que consiste en el aminoácido 357 y el aminoácido 395.
ES13163182T 2003-06-19 2004-06-21 Viriones de AAV con inmunorreactividad reducida y usos de los mismos Expired - Lifetime ES2629380T3 (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US48039503P 2003-06-19 2003-06-19
US56731004P 2004-04-30 2004-04-30
US57650104P 2004-06-03 2004-06-03

Publications (1)

Publication Number Publication Date
ES2629380T3 true ES2629380T3 (es) 2017-08-09

Family

ID=33545345

Family Applications (5)

Application Number Title Priority Date Filing Date
ES17162137T Expired - Lifetime ES2862206T3 (es) 2003-06-19 2004-06-21 Viriones de AAV con inmunorreactividad disminuida y sus usos
ES04755800.2T Expired - Lifetime ES2563064T3 (es) 2003-06-19 2004-06-21 Viriones de AAV con inmunorreactividad reducida y usos de los mismos
ES13163168.1T Expired - Lifetime ES2629087T3 (es) 2003-06-19 2004-06-21 Viriones de AAV con inmunorreactividad reducida y usos de los mismos
ES13163182T Expired - Lifetime ES2629380T3 (es) 2003-06-19 2004-06-21 Viriones de AAV con inmunorreactividad reducida y usos de los mismos
ES11158742.4T Expired - Lifetime ES2627913T3 (es) 2003-06-19 2004-06-21 Viriones de AAV con inmunorreactividad reducida y usos de los mismos

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ES17162137T Expired - Lifetime ES2862206T3 (es) 2003-06-19 2004-06-21 Viriones de AAV con inmunorreactividad disminuida y sus usos
ES04755800.2T Expired - Lifetime ES2563064T3 (es) 2003-06-19 2004-06-21 Viriones de AAV con inmunorreactividad reducida y usos de los mismos
ES13163168.1T Expired - Lifetime ES2629087T3 (es) 2003-06-19 2004-06-21 Viriones de AAV con inmunorreactividad reducida y usos de los mismos

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ES11158742.4T Expired - Lifetime ES2627913T3 (es) 2003-06-19 2004-06-21 Viriones de AAV con inmunorreactividad reducida y usos de los mismos

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Country Link
US (6) US7259151B2 (es)
EP (5) EP1633772B1 (es)
JP (7) JP2007524386A (es)
CY (5) CY1117575T1 (es)
DK (5) DK3235827T3 (es)
ES (5) ES2862206T3 (es)
HU (5) HUE029556T2 (es)
PL (5) PL1633772T3 (es)
PT (5) PT2657247T (es)
SI (5) SI3235827T1 (es)
WO (1) WO2004112727A2 (es)

Families Citing this family (214)

* Cited by examiner, † Cited by third party
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EP1082413B1 (en) 1998-05-28 2008-07-23 THE GOVERNMENT OF THE UNITED STATES OF AMERICA, as represented by THE SECRETARY, DEPARTMENT OF HEALTH AND HUMAN SERVICES Aav5 vector and uses thereof
JP4969002B2 (ja) 1999-06-08 2012-07-04 ユニバーシテイ・オブ・アイオワ・リサーチ・フアウンデーシヨン ｒＡＡＶ形質導入を増加するための化合物および方法
NZ532635A (en) 2001-11-13 2007-05-31 Univ Pennsylvania A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method
US7419817B2 (en) 2002-05-17 2008-09-02 The United States Of America As Represented By The Secretary Department Of Health And Human Services, Nih. Scalable purification of AAV2, AAV4 or AAV5 using ion-exchange chromatography
JP2007524379A (ja) 2003-03-31 2007-08-30 ユニバーシテイ・オブ・アイオワ・リサーチ・フアウンデーシヨン 上皮ナトリウムチャンネル関連障害のＰｈａｒｍｉｃｏ−遺伝子治療
WO2005017101A2 (en) 2003-05-19 2005-02-24 THE GOVERNMENT OF THE UNITED STATES OF AMERICA, as represented by THE SECRETARY, DEPARTMENT OF HEALTH & HUMAN SERVICES, NATIONAL INSTITUTES OF HEALTH Avian adenoassociated virus (aaav) and uses thereof
SI3235827T1 (sl) 2003-06-19 2021-05-31 Genzyme Corporation AAV virioni z zmanjšano imunoreaktivnostjo in uporaba za ta namen
US9233131B2 (en) * 2003-06-30 2016-01-12 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
US9441244B2 (en) 2003-06-30 2016-09-13 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
WO2005056807A2 (en) * 2003-12-04 2005-06-23 The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services, National Institutes Of Health Bovine adeno-associated viral (baav) vector and uses thereof
WO2006029196A1 (en) * 2004-09-08 2006-03-16 The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services, National Institutes Of H Ealth Transcytosis of adeno-associated viruses
EP1828390B1 (en) * 2004-12-15 2012-06-13 The University Of North Carolina At Chapel Hill Chimeric vectors
WO2006110689A2 (en) * 2005-04-07 2006-10-19 The Trustees Of The University Of Pennsylvania Method of increasing the function of an aav vector
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