Title:
NUCLEIC ACID-ENCAPSULATING AAV EMPTY PARTICLES
Document Type and Number:
WIPO Patent Application WO/2018/139637
Kind Code:
A1
Abstract:
The present invention provides a method for manufacturing nucleic acid-encapsulating empty particles of the adeno-associated virus (AAV), the method comprising the following steps: (1) a step in which linear nucleic acid fragments are prepared, the nucleic acid fragments comprising (i) the sequence of the A region of an AAV inverted terminal repeat sequence (ITR), (ii) the sequence of the A' region of the AAV ITR, and (iii) the sequence of the D region and/or the sequence of the D' region of the AAV ITR; (2) a step in which the nucleic acid fragments from (1) are introduced into cells that generate AAV empty particles; and (3) a step in which the cells from (2) are cultured.
Inventors:
OKADA TAKASHI (JP)
OKADA HIRONORI (JP)
MIYAGAWA YOSHITAKA (JP)
MINENO JUNICHI (JP)
CHONO HIDETO (JP)
OKADA HIRONORI (JP)
MIYAGAWA YOSHITAKA (JP)
MINENO JUNICHI (JP)
CHONO HIDETO (JP)
Application Number:
PCT/JP2018/002687
Publication Date:
August 02, 2018
Filing Date:
January 29, 2018
Export Citation:
Assignee:
NIPPON MEDICAL SCHOOL FOUNDATION (JP)
TAKARA BIO INC (JP)
TAKARA BIO INC (JP)
International Classes:
C12N7/01; C12N15/09
Foreign References:
| US20070280906A1 | 2007-12-06 |
Other References:
CHIORINI, JOHN A. ET AL.: "Adeno-associated virus (AAV) type 5 Rep protein cleaves a unique terminal resolution site compared with other AAV serotypes", J. VIROL., vol. 73, no. 5, 1999, pages 4293 - 4298, XP055529796, ISSN: 1098-5514
NAHREINI, PIRUZ ET AL.: "Cloning and integration of DNA fragments in human cells via the inverted terminal repeats of the adeno-associated virus 2 genome", GENE, vol. 119, 1992, pages 265 - 272, XP023540634, ISSN: 0378-1119
CHIORINI, JOHN A.: "Determination of adeno-associated virus Rep68 and Rep78 binding sites by random sequence oligonucleotide selection", J. VIROL., vol. 69, no. 11, 1995, pages 7334 - 7338, XP055529800, ISSN: 1098-5514
MUSATOV, SERGEI ET AL.: "A cis-acting element that directs circular adeno-associated virus replication and packaging", J. VIROL., vol. 76, no. 24, 2002, pages 12792 - 12802, XP002325443, ISSN: 1098-5514
PETRAS, OGNJEN: "Generation of Novel Double-Stranded AAV Vectors with an Expanded Packaging Capacity Using Linearized 1-AAV ITR DNA Molecules", MOLECULAR THERAPY, vol. 9, 2004, pages S284 - S285, XP004635114, ISSN: 1525-0016
NAHREINI, PIRUZ ET AL.: "Cloning and integration of DNA fragments in human cells via the inverted terminal repeats of the adeno-associated virus 2 genome", GENE, vol. 119, 1992, pages 265 - 272, XP023540634, ISSN: 0378-1119
CHIORINI, JOHN A.: "Determination of adeno-associated virus Rep68 and Rep78 binding sites by random sequence oligonucleotide selection", J. VIROL., vol. 69, no. 11, 1995, pages 7334 - 7338, XP055529800, ISSN: 1098-5514
MUSATOV, SERGEI ET AL.: "A cis-acting element that directs circular adeno-associated virus replication and packaging", J. VIROL., vol. 76, no. 24, 2002, pages 12792 - 12802, XP002325443, ISSN: 1098-5514
PETRAS, OGNJEN: "Generation of Novel Double-Stranded AAV Vectors with an Expanded Packaging Capacity Using Linearized 1-AAV ITR DNA Molecules", MOLECULAR THERAPY, vol. 9, 2004, pages S284 - S285, XP004635114, ISSN: 1525-0016
Attorney, Agent or Firm:
SAMEJIMA, Mutsumi et al. (JP)
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