Thursday, June 5, 2014
over two decades, treatment of acute porphyrias has relied on hemin
infusions that restore heme levels, but the therapy is slow to work, has side
effects and is expensive. Now, a team led by Alnylam Pharmaceuticals Inc. has created an
siRNA therapeutic that reduces disease symptoms in mice.1
team tested 45 siRNAs to find the most potent repressor of Alas-1 mRNA
expression in cultured mouse hepatocytes. The researchers then formulated the
optimal siRNA, dubbed Alas1-siRNA1, in lipid nanoparticles to enable
i.v. delivery to the liver.
to Bissell and the study's authors, one key benefit of the siRNA is its fast
kinetics versus hemin.
Donner, A. SciBX
Published online June 5, 2014
1. Yasuda, M. et al. Proc. Natl. Acad. Sci. USA;
published online May 12, 2014; doi:10.1073/pnas.1406228111 Contact:
Robert J. Desnick, Icahn School of Medicine at Mount Sinai, New York, N.Y. e-mail:
firstname.lastname@example.org Contact: Maria I. New, same affiliation as above e-mail: email@example.com
AND INSTITUTIONS MENTIONED
Alnylam Pharmaceuticals Inc. (NASDAQ:ALNY), Cambridge, Mass.
American Porphyria Foundation, Houston, Texas
Icahn School of Medicine at Mount Sinai, New York, N.Y.
National Institutes of Health, Bethesda, Md.
Recordati S.p.A. (Milan:REC), Milan, Italy
(NASDAQ:QURE) Amsterdam, the Netherlands
University of California, San Francisco, Calif.
The University of Texas Medical Branch, Galveston, Texas