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Titolo:
STABLE DELIVERY OF PHYSIOLOGICAL LEVELS OF RECOMBINANT ERYTHROPOIETINTO THE SYSTEMIC CIRCULATION BY INTRAMUSCULAR INJECTION OF REPLICATION-DEFECTIVE ADENOVIRUS
Autore:
TRIPATHY SK; GOLDWASSER E; LU MM; BARR E; LEIDEN JM;
Indirizzi:
UNIV CHICAGO,DEPT PATHOL,5841 S MARYLAND AVE CHICAGO IL 60637 UNIV CHICAGO,DEPT BIOCHEM & MOLEC BIOL CHICAGO IL 60637 UNIV CHICAGO,DEPT MED CHICAGO IL 60637
Titolo Testata:
Proceedings of the National Academy of Sciences of the United Statesof America
fascicolo: 24, volume: 91, anno: 1994,
pagine: 11557 - 11561
SICI:
0027-8424(1994)91:24<11557:SDOPLO>2.0.ZU;2-X
Fonte:
ISI
Lingua:
ENG
Soggetto:
GENETICALLY MODIFIED MYOBLASTS; DUCHENNE MUSCULAR-DYSTROPHY; HAMSTER OVARY CELLS; HUMAN FACTOR-IX; GENE-THERAPY; HUMAN ALPHA-1-ANTITRYPSIN; MEDIATED TRANSFER; MUSCLE-CELLS; EXPRESSION; INVIVO;
Tipo documento:
Article
Natura:
Periodico
Settore Disciplinare:
Science Citation Index Expanded
Citazioni:
40
Recensione:
Indirizzi per estratti:
Citazione:
S.K. Tripathy et al., "STABLE DELIVERY OF PHYSIOLOGICAL LEVELS OF RECOMBINANT ERYTHROPOIETINTO THE SYSTEMIC CIRCULATION BY INTRAMUSCULAR INJECTION OF REPLICATION-DEFECTIVE ADENOVIRUS", Proceedings of the National Academy of Sciences of the United Statesof America, 91(24), 1994, pp. 11557-11561

Abstract

A number of inherited and acquired serum protein deficiencies including hemophilias A and B, diabetes mellitus, and the erythropoietin-responsive anemias are currently treated with repeated subcutaneous or intravenous infusions of purified or recombinant proteins. The development of an in vivo gene-transfer approach to deliver physiologic levels of recombinant proteins to the systemic circulation would represent a significant advance in the treatment of these disorders. Here we describe the construction of a replication-defective adenovirus (AdEF1hEpo) containing the human erythropoietin (hEpo) cDNA under the transcriptional control of the cellular elongation factor 1 alpha (EP1 alpha) promoter and the 4F2 heavy chain (4F2HC) enhancer. Neonatal CD-1 and adultSCID mice injected once intramuscularly (i.m.) with 10(7) to 10(9) plaque-forming units (pfu) of this virus displayed significant dose-dependent elevations of serum hEpo levels and increased hematocrits, whichwere stable over the 4-month time course of these experiments. Adenovirus injected i.m. remained localized at the site of injection and there was no evidence of either systemic infection or a localized inflammatory response. These results suggest that i.m. injection of recombinant replication-defective adenovirus vectors may serve as a paradigm for the treatment of human serum protein deficiencies.

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Documento generato il 23/09/20 alle ore 20:48:13