Blood online
Home About Blood Authors Subscriptions Permission Advertising Public Access contact us
 

 
Advanced
Current Issue
First Edition
Future Articles
Archives
Submit to Blood
Search
American Society of Hematology
Meeting Abstracts
Email Alerts
 Blood, 1 October 2005, Vol. 106, No. 7, pp. 2280-2286.
Prepublished online as a Blood First Edition Paper on March 29, 2005; DOI 10.1182/blood-2004-11-4174.

This Article
Right arrow Full Text
Right arrow Full Text (PDF)
Right arrow All Versions of this Article:
2004-11-4174v1
106/7/2280    most recent
Right arrow Alert me when this article is cited
Right arrow Alert me if a correction is posted
Right arrow Citation Map
Services
Right arrow Email this article to a friend
Right arrow Similar articles in this journal
Right arrow Similar articles in PubMed
Right arrow Alert me to new issues of the journal
Right arrow Download to citation manager
Right arrow reprints & permissions
Right arrow Rights and Permissions
Citing Articles
Right arrow Citing Articles via HighWire
Right arrow Citing Articles via CrossRef
Right arrow Citing Articles via Google Scholar
Google Scholar
Right arrow Articles by Lippin, Y.
Right arrow Articles by Galun, E.
Right arrow Search for Related Content
PubMed
Right arrow PubMed Citation
Right arrow Articles by Lippin, Y.
Right arrow Articles by Galun, E.
Related Collections
Right arrow Hematopoiesis and Stem Cells
Right arrow Red Cells
Right arrow Gene Therapy
Social Bookmarking
Add to CiteULike   Add to Connotea   Add to Del.icio.us   Add to Digg   Add to Reddit   Add to Technorati  
What's this?

arrow to previous article Previous Article  |  Table of Contents  |  Next Article next article arrow

GENE THERAPY

Human erythropoietin gene therapy for patients with chronic renal failure

Yitzhak Lippin, Michal Dranitzki-Elhalel, Einat Brill-Almon, Chava Mei-Zahav, Sarah Mizrachi, Yael Liberman, Adrian Iaina, Eli Kaplan, Eduardo Podjarny, Evelyne Zeira, Menahem Harati, Nicole Casadevall, Noam Shani, and Eithan Galun

From the Department of Plastic Surgery, Rambam Medical Center, Haifa, Israel; the Department of Medicine, Hadassah-Hebrew University Hospital, Jerusalem, Israel; Medgenics, Biogenics, Misgav, Israel; the Goldyne Savad Institute of Gene Therapy, Hadassah-Hebrew University Hospital, Jerusalem, Israel; the Department of Nephrology, Sourasky Medical Center, Tel Aviv, Israel; Medical Clinic, Herzliah, Israel; the Laboratoire d'Hematologie, Hôtel Dieu, Paris, France; and Institut National de la Santé et de la Recherche Médicale (INSERM) 362, Paris, France.

Gene therapy holds a major promise. However, until now, this promise was fulfilled only in few cases, in rare genetic diseases. One very common clinical condition is anemia. Patients with anemia of chronic renal failure are treated with erythropoietin. The objective of this study was to develop a therapeutic platform for serum-secreted proteins like erythropoietin. We developed a tissue protein factory based on dermal cores (Biopump) harvested and implanted autologously. In this study, an adenovector was designed to express the human erythropoietin under the control of the cytomegalovirus (CMV) promoter. This vector transduced the harvested dermal cores ex vivo. The transduced cores were implanted, and erythropoietin and reticulocyte counts were measured. Dermal cores were harvested from 13 patients with chronic renal failure, and implantation was performed in 10. There were no significant drug-related side effects to this procedure. Erythropoietin serum levels increased significantly to therapeutic levels from day 1 after implantation reaching a peak during the first week of follow-up. The expression period was transient for up to 14 days. The rise of erythropoietin was followed by a transient significant increase in reticulocyte counts. The decrease of erythropoietin expression coincided with a significant dermal infiltrate of CD8 cytotoxic T cells. Antierythropoietin antibodies were not detected until day 90 following implantation. Implantation of dermal cores ex vivo transduced with human genes could eventually be used in the clinical setting to express therapeutic serum proteins. However, nonimmunogenic delivery system should be tested as gene vehicles.


Add to CiteULike CiteULike   Add to Connotea Connotea   Add to Del.icio.us Del.icio.us   Add to Digg Digg   Add to Reddit Reddit   Add to Technorati Technorati    What's this?


This article has been cited by other articles:


Home page
 HeartHome page
A R Lyon, M Sato, R J Hajjar, R J Samulski, and S E Harding
Gene therapy: targeting the myocardium
Heart, January 1, 2008; 94(1): 89 - 99.
[Abstract] [Full Text] [PDF]

Home page
 CJASNHome page
I. C. Macdougall
Novel Erythropoiesis-Stimulating Agents: A New Era in Anemia Management
Clin. J. Am. Soc. Nephrol., January 1, 2008; 3(1): 200 - 207.
[Abstract] [Full Text] [PDF]

Home page
 BloodHome page
M. M. Hsieh, N. S. Linde, A. Wynter, M. Metzger, C. Wong, I. Langsetmo, A. Lin, R. Smith, G. P. Rodgers, R. E. Donahue, et al.
HIF prolyl hydroxylase inhibition results in endogenous erythropoietin induction, erythrocytosis, and modest fetal hemoglobin expression in rhesus macaques
Blood, September 15, 2007; 110(6): 2140 - 2147.
[Abstract] [Full Text] [PDF]

Home page
 J. Am. Soc. Nephrol.Home page
N. Eliopoulos, R. F. Gagnon, M. Francois, and J. Galipeau
Erythropoietin Delivery by Genetically Engineered Bone Marrow Stromal Cells for Correction of Anemia in Mice with Chronic Renal Failure
J. Am. Soc. Nephrol., June 1, 2006; 17(6): 1576 - 1584.
[Abstract] [Full Text] [PDF]


click for free articles
home about blood authors subscriptions permissions advertising public access contact us
  Copyright © 2005 by American Society of Hematology         Online ISSN: 1528-0020