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`US007345025B2
`
`(12) United States Patent
`Symonds et al.
`
`(10) Patent No.:
`(45) Date of Patent:
`
`US 7,345,025 B2
`Mar.18,2008
`
`(54) METHODS FOR GENETIC MODIFICATION
`OF HEMATOPOIETIC PROGENITOR CELLS
`AND USES OF THE MODIFIED CELLS
`
`(75)
`
`Inventors: Geoffrey P. Symonds, Rosebay (AU);
`Rafael G. Amado, Encino, CA (US);
`Lun-Quan Sun, Eastwood (AU); Janet
`L. MacPherson, Leichhardt (AU);
`Gregory C. Fanning, Bronte (AU);
`Wayne Gerlach, East Killara (AU)
`
`(73) Assignee: Johnson & Johnson Research Pty.
`Limited, Eveleigh (AU)
`
`( *) Notice:
`
`Subject to any disclaimer, the tenn of this
`patent is extended or adjusted under 35
`U.S.C. 154(b) by 0 days.
`
`(21) Appl. No.: 10/192,980
`
`(22) Filed:
`
`Jul. 10, 2002
`
`(65)
`
`Prior Publication Data
`
`US 2004/0072771 Al
`
`Apr. 15, 2004
`
`(60)
`
`(51)
`
`(52)
`
`(58)
`
`Related U.S. Application Data
`
`Provisional application No. 60/386,063, filed on Jun.
`4, 2002, provisional application No. 60/343,484, filed
`on Dec. 21, 2001, provisional application No. 60/304,
`283, filed on Jul. 10, 2001, provisional application
`No. 60/304,127, filed on Jul. 10, 2001.
`
`Int. Cl.
`AOlN 43104
`(2006.01)
`C12Q 1168
`(2006.01)
`CJ2N 15100
`(2006.01)
`C07H 21102
`(2006.01)
`C07H 21104
`(2006.01)
`U.S. Cl. ........................... 514/44; 435/6; 435/91.1;
`435/91.31; 435/320.l; 435/455; 435/458;
`536/23.1; 536/24.5
`Field of Classification Search .................. 514/44;
`424/93.l; 435/320.1, 6, 91.1, 91.31, 455,
`435/458, 375; 536/23.1, 24.5
`See application file for complete search history.
`
`(56)
`
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`
`(Continued)
`
`Primary Examiner-Jane Zara
`(74) Attorney, Agent, or Firm-John P. White; Cooper &
`Dunham LLP
`
`(57)
`
`ABSTRACT
`
`Described are compositions and methods relating to gene
`therapy, particularly as applied to hematopoietic progenitor
`(HP) cells, to transduced cells and methods of obtaining
`them, and to methods of using them to provide prolonged
`engraftment of modified hematopoietic cells in human sub(cid:173)
`jects. The invention particularly relates to ex vivo gene
`therapy of HP cells for treatment or prevention of HIV
`infection.
`
`18 Claims, 21 Drawing Sheets
`
`

`
`US 7,345,025 B2
`2
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`

`
`US 7,345,025 B2
`3
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