Abstract: A non-human transgenic mammalian animal, as described above, contains an exogenous double stranded DNA sequence stably integrated into the genome of the animal, which comprises cis-acting regulatory units operably linked to a DNA sequence encoding human FIX protein without the benefit of the presence of a complete milk gene sequence for gene rescue, and a signal sequence is active in directing newly expressed Factor IX into the milk of the animal at levels in an unactivated form that is suitable for subsequent processing for therapeutic applications in treating Hemophilia B. The transgenic mammals are preferably pigs, cows, sheep, goats and rabbits. The applications include milk derivatives for oral delivery and oral tolerization in the treatment of Hemophilia B.
Type:
Grant
Filed:
October 21, 2008
Date of Patent:
February 15, 2011
Assignee:
Progenetics LLC
Inventors:
Julian D. Cooper, William Hugold Velander, Tanya K. O'Sickey
Abstract: A non-human transgenic mammalian animal, as described above, contains one or more exogenous double stranded DNA sequence(s) stably integrated into the genome of the animal, which comprises trans-acting regulatory units controlling expression of DNA sequences encoding proteins to be secreted into the milk of transgenic mammals. The DNA sequence of the trans-regulatory gene encodes transcriptional activating proteins, which are not secreted but made in a temporally controlled and mammary tissue specific manner. The DNA sequence containing the protein to be secreted in the milk is constructed on a separate gene sequence under the regulation of a minimal promoter and a trans-activation binding domain. The transgenic mammals are preferably pigs, cows, sheep, goats and rabbits. A related composition and method for making transgenic proteins which require specialized propeptides for proper post-translational processing is also described.
Type:
Application
Filed:
May 24, 2010
Publication date:
December 30, 2010
Applicant:
PROGENETICS LLC
Inventors:
Julian D. Cooper, Tanya K. O'Sickey, Stephen P. Butler
Abstract: A non-human transgenic mammalian animal, as described above, contains one or more exogenous double stranded DNA sequence(s) stably integrated into the genome of the animal, which comprises trans-acting regulatory units controlling expression of DNA sequences encoding proteins to be secreted into the milk of transgenic mammals. The DNA sequence of the trans-regulatory gene encodes transcriptional activating proteins, which are not secreted but made in a temporally controlled and mammary tissue specific manner. The DNA sequence containing the protein to be secreted in the milk is constructed on a separate gene sequence under the regulation of a minimal promoter and a trans-activation binding domain. The transgenic mammals are preferably pigs, cows, sheep, goats and rabbits. A related composition and method for making transgenic proteins which require specialized propeptides for proper post-translational processing is also described.
Type:
Grant
Filed:
March 11, 2002
Date of Patent:
May 25, 2010
Assignee:
Progenetics LLC
Inventors:
Julian D. Cooper, Tanya K. O'Sickey, Stephen P. Butler