As I have described previously, we are working toward developing agents for gene
therapy by redesigning homing endonucleases (DNA cleavage enzymes) to
cut within genes containing mutations responsible for various
diseases. It has been shown that cleavage near a disease-causing
mutation will induce cellular recombination pathways leading to
correction of that mutation. Our focus is on diseases amenable to
gene therapy, where the healthy cells have an advantage over the
diseased cells, and correction of the genomes of even a fraction of
the cells can cure the disease. The diseases on our list include (but
not limited too), fanconi anemia, cystic fibrosis, haemophila A,
XSCID, ADASCID, and tyrosinemia I. Recently, we have produced
successful endonuclease designs toward partial target sequences near
mutations in genes responsible for fanconi anemia, haemophilia A, and
tyrosinemia I, bringing us steps closer to our goal of developing proteins
that can help cure these diseases.

We are all excited about the increase in computational throughput in the last month which
is letting us test many more ideas more quicly. Thanks to all of you!