Primary hyperoxaluria mouse model and gene therapy.

Posted by & filed under Part 15: PEROXISOMES, Treatment.

Primary hyperoxaluria mouse model and gene therapy.
Proc Natl Acad Sci U S A. 2006 Nov 28;103(48):18249-54.

Alanine-glyoxylate aminotransferase-deficient mice, a model for primary
hyperoxaluria that responds to adenoviral gene transfer.

Salido EC, Li XM, Lu Y, Wang X, Santana A, Roy-Chowdhury N, Torres A, Shapiro
LJ, Roy-Chowdhury J.
In this important PNAS paper, the authors describe the creation of a mouse model for primary hyperoxaluria type 1. They also provide a proof of concept for liver directed gene therapy of primary hyperoxaluria using an adenoviral vector. However, I would have liked to see if the vector-treated mice were protected against ethylene glycol induced nephrocalcinosis.

For more information on primary hyperoxaluria, you can refer to chapter 133 of OMMBID.

Thank you very much in advance for your contributions to this blog (Click on login to register and post a message).
Philippe Campeau, MD
Resident in Medical Genetics at McGill University
OMMBID Blog Administrator

Leave a Reply

You must be logged in to post a comment.