Gene therapy for Huntington's disease

Shilpa Ramaswamy, Jeffrey H. Kordower

Research output: Contribution to journalReview articlepeer-review

51 Scopus citations


Huntington's disease (HD) is a neurodegenerative disease for which there is no cure. Therapies that are efficacious in animal models have to date shown benefit for humans. One potential powerful approach is gene therapy. The ideal method of administration of gene therapy has been hotly debated and viral vectors have provided one method of long-term and wide-spread delivery to the brain. Trophic factors to protect cells from degeneration and RNAi to reduce mutant huntingtin (mHtt) protein expression are 2 main classes of compounds that demonstrate benefit in animal models. This review will examine some commonly used adeno-associated viral (AAV) vectors and discuss some therapies that hold promise for HD.

Original languageEnglish (US)
Pages (from-to)243-254
Number of pages12
JournalNeurobiology of Disease
Issue number2
StatePublished - Nov 2012
Externally publishedYes


  • Adeno-associated viral (AAV) vectors
  • Allele-specific RNAi
  • Brain-derived neurotrophic factor (BDNF)
  • Ciliary neurotrophic factor (CNTF)
  • Gene therapy
  • GFL
  • Glial cell line-derived neurotrophic factor (GDNF)
  • Intrabody therapy
  • Neurturin (NRTN)
  • Nonallele-specific RNAi
  • Oligonucleotides
  • Pseudotypes
  • RNA interference (RNAi)
  • Serotype

ASJC Scopus subject areas

  • Neurology


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