Expression of erythropoietin in cats treated with a recombinant adeno-associated viral vector

Mark C. Walker, Tamara C. Mandell, P. Cynda Crawford, Greg G. Simon, Kevin S. Cahill, Peter J. Fernandes, James N. MacLeod, Barry J. Byrne, Julie K. Levy

Research output: Contribution to journalArticlepeer-review

6 Scopus citations


Objective - To characterize the biological effects of IM administration of a recombinant adeno-associated virus serotype 2 (rAAV2) vector containing feline erythropoietin (fEPO) cDNA and determine whether readministration of the vector or removal of muscle tissue at the injection sites alters those effects. Animals - 10 healthy 7-week-old specific pathogen-free cats. Procedure - Cats received 1 × 107 infective units (iU; n = 3), 1 × 108 iU (3), or 1 × 109 iU (2) of rAAV2-fEPO vector IM (day 0). Two control cats received an rAAV2 vector containing the LacZ gene (1 × 109 iU, IM). In all cats, hematologic variables and serum fEPO concentration were measured at intervals; anti-rAAV2 antibody titer was measured on day 227. In cats that did not respond to treatment, the rAAV2-fEPO vector was readministered. Injection sites were subsequently surgically removed. Results - Compared with control cats, cats treated with 1 × 109 iU of rAAV2-fEPO vector had increased Hct and serum fEPO concentrations. One of these cats developed pure BBC aplasia; its Hct normalized following injection site excision. Cats receiving lower doses of vector had no response; on retreatment, 1 of those cats developed sustained erythrocytosis that persisted despite injection site removal and the others did not respond or responded transiently. Antibodies against rAAV2 were detected in all vector-treated cats. Conclusions and clinical relevance - Gene therapy may be an effective treatment for cats with hypoproliferative anemia. However, rAAV2-fEPO vector administration may result in pure BBC aplasia or pathologic erythrocytosis, and injection site removal does not consistently abolish the biological response.

Original languageEnglish
Pages (from-to)450-456
Number of pages7
JournalAmerican Journal of Veterinary Research
Issue number3
StatePublished - Mar 2005

ASJC Scopus subject areas

  • General Veterinary


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