[Molecular therapeutic strategies for Huntington's disease].
Huntington's disease is a progressive neurodegenerative disorder of genetic origin that still lacks an effective treatment. Recently, a number of new attempts have been undertaken to develop a successful molecular therapy for this incurable condition. The novel approaches employ, among others, some new methods to selectively silence the mutated gene or to neutralize its toxic protein product. This paper reviews all major strategies that are currently considered for molecular therapy of Huntington's disease while discussing their potential effectiveness regarding the treatment of both the Huntington's disease and a large group of related neurodegenerative disorders associated with abnormal protein aggregation.
Michał
M
Milewski
Dorota
D
Hoffman-Zacharska
Jerzy
J
Ball
D020868:Gene Silencing; D006801:Humans; D006816:Huntington Disease / Q000235:genetics / Q000628:therapy*; D058990:Molecular Targeted Therapy / Q000379:methods*