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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