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November 24, 2005
Another Gene Therapy Treatment For HD
Neurologix, another struggling biotech company, has a promising gene therapy treatment for Huntington's Disease that has done well in pre-clinical testing. From their press release:
Neurologix, Inc. today 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. ...Using cell culture models of the disease, the researchers showed that a truncated form of XIAP lacking the RING domain (RING) 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 life-span of the mice by 16%. Furthermore, no adverse effects due to dXIAP over-production were observed.
Posted by Dave at November 24, 2005 11:59 AM
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