Therapeutic Strategies for Huntington’s Disease

  • Ladislav Mrzljak
  • Ignacio Munoz-Sanjuan
Part of the Current Topics in Behavioral Neurosciences book series (CTBN, volume 22)


Huntington’s disease (HD) is a devastating autosomal dominant neurodegenerative disease, caused by expansion of the CAG repeat in the huntingtin (HTT) gene and characterized pathologically by the loss of pyramidal neurons in several cortical areas, of striatal medium spiny neurons, and of hypothalamic neurons. Clinically, a distinguishing feature of the disease is uncontrolled involuntary movements (chorea, dyskensias) accompanied by progressive cognitive, motor, and psychiatric impairment. This review focuses on the current state of therapeutic development for the treatment of HD, including the preclinical and clinical development of small molecules and molecular therapies.


Huntington’s disease Huntingtin CAG repeats Kynurenine PDE HDACs ASO siRNA Zinc-finger repressors Dopamine Glutamate GABA Adenosine 


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Copyright information

© Springer-Verlag Berlin Heidelberg 2013

Authors and Affiliations

  1. 1.CHDI Management/CHDI Foundation Inc.Los AngelesUSA
  2. 2.CHDI Management/CHDI Foundation Inc.PrincetonUSA

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