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New Hope for Dominant Retinal Conditions
An announcement at the Congress pre-empted the official report published
in the August edition of Nature magazine on the success of Ribozyme
therapy for Autosomal Dominant forms of retinal degeneration.
In this type of inheritance, there is a 50% chance on each pregnancy of
the condition being passed on to the child from an affected parent -
either mother or father.
A research team led by Dr William Hauswirth and Dr Alfred Lewin of the
University of Florida, have shown that Ribozyme therapy dramatically
decreases vision loss in an animal model with RP.
Photoreceptor cell function was as much as 93% better in the treated eye
than in the untreated control eye. Ribozyme molecules can be encoded to
disrupt the message from the incorrect copy of the gene, stopping the
production of incorrect protein material. With the mutant DNA
inactivated, the healthy copy of the gene (present in all dominant
conditions) can then supply the cells with the needed protein to ensure
cell survival.
With this "proof of principle" established, it is hoped that clinical
trials for this type of retinal degeneration will begin soon.
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