Clinical and molecular characterization of a re-established line of sheep exhibiting hemophilia A.

ABSTRACT:

Background

Large animal models that accurately mimic human hemophilia A (HA) are in great demand for developing and testing novel therapies to treat HA.

Objectives

To re-establish a line of sheep exhibiting a spontaneous bleeding disorder closely mimicking severe human HA, fully characterize their clinical presentation, and define the molecular basis for disease.

Patients/methods

Sequential reproductive manipulations were performed with cryopreserved semen from a deceased affected ram. The resultant animals were examined for hematologic parameters, clinical symptoms, and responsiveness to human FVIII (hFVIII). The full coding region of sheep FVIII mRNA was sequenced to identify the genetic lesion.

Results and conclusions

The combined reproductive technologies y

SUBMITTER: Porada CD 

PROVIDER: S-EPMC2826196 | biostudies-literature | 2010 Feb

REPOSITORIES: biostudies-literature