Results Show Potential for Gene Therapy Treatment of Huntington's Disease
News Nov 21, 2005
Neurologix, Inc. has announced findings from preclinical studies, which showed that the gene XIAP (X-linked inhibitor of apoptosis) may prevent the progression of Huntington's disease.
Neurologix scientists demonstrated that a mutated form of the gene delivered by an adeno-associated virus (AAV) vector, introduced using standard neurosurgical techniques can improve motor deficits associated with the disease.
The findings were presented in a poster session during the 35th Annual Meeting of the Society for Neuroscience in Washington D.C. The title of the poster was "Neuroprotective effects of XIAP in models of Huntington's disease."
Using cell culture models of the disease, the researchers showed that a truncated form of XIAP lacking the RING domain (RING) may reduce cell death caused by a mutated form of human huntingtin gene.
The researchers further investigated the neuroprotective effects of dXIAP in a transgenic animal model by injecting presymptomatic mice with AAV vectors encoding dXIAP into the striatum, an area of the brain largely affected in Huntington's patients.
In the study, mice injected with this vector experienced significant protection from motor dysfunction when compared to mice treated with a control vector.
dXIAP also appeared to prolong the life-span of the mice by 16%. Furthermore, no adverse effects due to dXIAP over-production were observed.
"These preclinical data in the Huntington's disease study show the potential of the Company's neuroprotection approach of using AAV vectors to deliver therapeutic genes for the treatment of serious and debilitating neurological disorders," stated Michael Sorell, Chief Executive Officer.
"These studies and studies on additional compounds will need to be confirmed and the mechanisms and limitations further elucidated before testing in humans can begin," he added.
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