Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response (original) (raw)

Notes

1. **NOTE:**In the version of this article originally published, Pradip Rustagi was inadvertently omitted from the author list; John Rasko should be listed as John E. J. Rasko; the affiliations of John E. J. Rasko and Katherine A. High were incorrect; and the author order was incorrect. The correct list of authors with their affiliations is:
   Catherine S Manno1,2,15, Glenn F Pierce3,15, Valder R Arruda1,2,15, Bertil Glader4,15, Margaret Ragni5, John J E Rasko6, Margareth C Ozelo7, Keith Hoots8, Philip Blatt9, Barbara Konkle10, Michael Dake4, Robin Kaye1,2, Mahmood Razavi4, Albert Zajko10, James Zehnder4, Pradip K Rustagi11, Hiroyuki Nakai4, Amy Chew1,3, Debra Leonard2,12, J Fraser Wright3, Ruth R Lessard3, Jürg M Sommer3, Michael Tigges3, Denise Sabatino1, Alvin Luk3, Haiyan Jiang3, Federico Mingozzi1, Linda Couto3, Hildegund C Ertl1,13, Katherine A High1,2,14 & Mark A Kay4
   1The Children’s Hospital of Philadelphia, Philadelphia and 2University of Pennsylvania School of Medicine, 3615 Civic Center Boulevard, Philadelphia, Pennsylvania, 19104, USA. 3Avigen Inc., 1301 Harbor Bay Parkway, Alameda, California 94502, USA. 4Stanford University School of Medicine, 300 Pasteur Drive, Room G305, Stanford, California 94305-5208, USA. 5University of Pittsburgh Medical Center, Hemophilia Center of Western Pennsylvania, 3636 Boulevard of the Allies, Pittsburgh, Pennsylvania 15213, USA. 6Royal Prince Alfred Hospital, Centenary Institute of Cancer Medicine & Cell Biology, Locked Bag No. 6, Newtown NSW 2042, Australia. 7University of Campinas, State University of Campinas, Caixa Postal 6198, CEP, 13083-970, Campinas-Sao Paolo, Brazil. 8University of Texas Houston Health Science Center, 1515 Holcombe Boulevard, Houston, Texas 77030, USA. 9Christiana Care, Christiana Hospital, 4755 Ogletown-Stanton Road, Newark, Delaware 19713, USA. 10University of Pennsylvania School of Medicine Presbyterian Hospital, MAB, Suite 103, 39th & Market Street, Philadelphia., Pennsylvania 19104, USA. 11Stanford University, School of Medicine, Department of Hematology, Jane Lathrop Building, 770 Welch Road, Suite 380, Palo Alto, California 94304, USA. 12Weill Medical College of Cornell University, 525 East 68th Street, New York, New York 10012, USA. 13Wistar Institute, 3601 Spruce Street, Philadelphia, Pennsylvania 19104, USA.. 14Howard Hughes Medical Institute, 3615 Civic Center Boulevard, Philadelphia, Pennsylvania 19104, USA. 15These authors contributed equally to this work. Correspondence should be addressed to K.A.H. (high@email.chop.edu).
   The error has been corrected in the HTML and PDF versions of the article.

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Acknowledgements

This work was supported by US National Institutes of Health grants U01 HL66948 (to M.A.K., K.A.H., C.S.M., B.G.), R01 HL71518 (to C.S.M.), P01 HL064190 (to K.A.H.), K01 DK60580 (to V.R.A.) and by the Howard Hughes Medical Institute. We thank each of the subjects for agreeing to participate in this study. We thank J. Bluestone and C. June for their scientific input. We thank J.W. Sun for assistance, critical reading and bioinformatics support.

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Author notes

1. Catherine S Manno, Glenn F Pierce, Valder R Arruda and Bertil Glader: These authors contributed equally to this work.

Authors and Affiliations

1. The Children's Hospital of Philadelphia,
   Catherine S Manno, Valder R Arruda, Robin Kaye, Amy Chew, Denise Sabatino, Federico Mingozzi, Hildegund C Ertl & Katherine A High
2. University of Pennsylvania School of Medicine, 3615 Civic Center Boulevard, Philadelphia, 19104, Pennsylvania, USA
   Catherine S Manno, Valder R Arruda, Robin Kaye, Debra Leonard & Katherine A High
3. Avigen, Inc., 1301 Harbor Bay Parkway, Alameda, 94502, California, USA
   Glenn F Pierce, Amy Chew, J Fraser Wright, Ruth R Lessard, Jürg M Sommer, Michael Tigges, Alvin Luk, Haiyan Jiang & Linda Couto
4. Stanford University School of Medicine, 300 Pasteur Drive, Room G305, Stanford, 94305-5208, California, USA
   Bertil Glader, Michael Dake, Mahmood Razavi, James Zehnder, Hiroyuki Nakai & Mark A Kay
5. University of Pittsburgh Medical Center, Hemophilia Center of Western Pennsylvania, 3636 Boulevard of the Allies, Pittsburgh, 15213, Pennsylvania, USA
   Margaret Ragni
6. Royal Prince Alfred Hospital, Centenary Institute of Cancer Medicine & Cell Biology, Locked Bag No 6, Newtown, 2042, NSW, Australia
   John J E Rasko
7. University of Campinas, State University of Campinas, Caixa Postal 6198, CEP, Campinas-Sao Paolo, 13083-970, Brazil
   Margareth C Ozelo
8. University of Texas Houston Health Science Center, 1515 Holcombe Boulevard, Houston, 77030, Texas, USA
   Keith Hoots
9. Christiana Care, Christiana Hospital, 4755 Ogletown-Stanton Road, Newark, 19713, Delaware, USA
   Philip Blatt
10. University of Pennsylvania School of Medicine Presbyterian Medical Center, MAB, Suite 103, 39th & Market Street, Philadelphia, 19104, Pennsylvania, USA
    Barbara Konkle & Albert Zajko
11. Department of Hematology, Stanford University, School of Medicine, Jane Lathrop Building, 770 Welch Road, Suite 380, Palo Alto, 94304, California , USA
    Pradip Rustagi
12. Weill Medical College of Cornell University, 525 East 68th Street, New York, 10012, New York, USA
    Debra Leonard
13. Wistar Institute, 3601 Spruce Street, Philadelphia, 19104, Pennsylvania, USA
    Hildegund C Ertl
14. Howard Hughes Medical Institute, 3615 Civic Center Boulevard, Philadelphia, 19104, Pennsylvania, USA
    Katherine A High

Authors

1. Catherine S Manno
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2. Glenn F Pierce
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3. Valder R Arruda
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4. Bertil Glader
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5. Margaret Ragni
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6. John J E Rasko
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7. Margareth C Ozelo
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8. Keith Hoots
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9. Philip Blatt
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10. Barbara Konkle
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11. Michael Dake
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12. Robin Kaye
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13. Mahmood Razavi
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14. Albert Zajko
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15. James Zehnder
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16. Pradip Rustagi
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17. Hiroyuki Nakai
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18. Amy Chew
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19. Debra Leonard
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20. J Fraser Wright
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21. Ruth R Lessard
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22. Jürg M Sommer
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23. Michael Tigges
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24. Denise Sabatino
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25. Alvin Luk
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26. Haiyan Jiang
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27. Federico Mingozzi
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28. Linda Couto
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29. Hildegund C Ertl
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30. Katherine A High
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31. Mark A Kay
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Corresponding author

Correspondence toKatherine A High.

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Competing interests

Glenn F. Pierce, Hiroyuki Nakai, Amy Chew, J. Fraser Wright, Ruth Lessard, Jürg M. Sommer, Michael Tigges, Alvin Luk, Haiyan Jiang, Linda Couto and Mark A. Kay are employed by or hold equity in Avigen, Inc., which manufactured the clinical-grade vector used in the study.

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Manno, C., Pierce, G., Arruda, V. et al. Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response.Nat Med 12, 342–347 (2006). https://doi.org/10.1038/nm1358

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• Received: 16 June 2005
• Accepted: 21 December 2005
• Published: 12 February 2006
• Issue Date: 01 March 2006
• DOI: https://doi.org/10.1038/nm1358