Neurologix shows mutated gene can improve motor deficits of Huntington's disease

By Ted A. Knutson

Washington, Nov. 18 - Neurologix, Inc. announced findings Friday 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.

"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," said Michael Sorell, Neurologix chief executive officer, in a news release. "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."

The research results were presented in a poster session during the 35th Annual Meeting of the Society for Neuroscience in Washington, D.C., by Sergei A. Musatov, Susan E. Browne, Matthew S. Henning, Mihaela A. Stavarache, Joshua A. Goldfein and Michael G. Kaplitt. 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 may significantly 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 lifespan of the mice by 16%. Furthermore, no adverse effects due to dXIAP over-production were observed.

The announcement was made in an 8-K filing with the Securities and Exchange Commission.

Fort Lee, N.J.-based Neurologix is a development-stage company, which through its subsidiary, Neurologix Research Inc., is engaged in the research and development of proprietary treatments for disorders of the brain and central nervous system, primarily using gene therapies.

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