Proteome Sciences results in big news CJD + Stroke tests

Here is the third patent application published on 18 December.



PUBLISHED INTERNATIONAL APPLICATION
(11) WO 03/104412 (13) A2
(21) PCT/US03/17913
(22) 05 June 2003 (05.06.2003)
(25) ENG (26) ENG
(31) 60/386,689 (32) 05 June 2002 (05.06.2002) US
(43) 18 December 2003 (18.12.2003)
(51)7 C12N
(54) SPLICEOSOME MEDIATED RNA TRANS-SPLICING AND CORRECTION OF FACTOR VIII GENETIC DEFECTS USING SPLICEOSOME MEDIATED RNA TRANS SPLING

(71) INTRONN, INC. 910 Clopper Road, Suite 210, Gaithersburg, MD 20878; (US). [US/US].(for all designated States except US)

Abstract
The compositions of the invention include pre-trans-splicing molecules (PTMs) designed to interact with a target precursor messenger RNA molecule (target pre-mRNA) and mediate a trans-splicing reaction resulting in the generation of a novel chimeric RNA molecule (chimeric RNA). In particular, the PTMs of the present invention are genetically engineered to interact with factor VIII (FVIII) target pre-mRNA so as to result in correction of clotting FVIII genetic defects responsible for hemophilia A. The compositions of the invention further include recombinant vector systems capable of expressing the PTMs of the invention and cells expressing said PTMs. The methods of the invention encompass contacting the PTMs of the invention with a FVIII target pre-mRNA under conditions in which a portion of the PTM is trans-spliced to a portion of the target pre-mRNA to form a RNA molecule wherein the genetic defect in the FVIII gene has been corrected. The methods and compositions of the present invention can be used in gene therapy for correction of FVIII disorders such as hemophilia A.