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Hereditary Disease Foundation
"Pushing for Answers: Strategies for Inactivation of Mutant Genes"
May 2-5, 1999
Cold Spring Harbor, New York
Report prepared by Lisa J. Bain
Abstract
A diverse group of scientists met for three days in May 1999, at a symposium co-sponsored by the Hereditary Disease Foundation and the Amyotrophic Lateral Sclerosis Association and its Greater New York Chapter. They discussed a wide array of tools and technologies being developed both to understand the basic biological mechanisms of these two diseases and develop strategies for correcting mutant genes. Three major challenges emerged: first, to understand more fully the pathogenic mechanisms underlying these diseases and identify the appropriate targets for gene therapy approaches; second, to develop specific tools that will correct the mutant genes; and third, to deliver these constructs to the relevant targets. Approaches discussed included viral and non-viral vectors engineered to deliver constructs such as ribozymes, chimeric oligonucleotides, and peptide nucleic acids. It will most likely be not one, but a combination of these strategies that will ultimately prove effective.
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