Neural transplantation for Huntington's disease: Experimental rationale and recommendations for clinical trials

K. M. Shannon, J. H. Kordower

Research output: Contribution to journalArticlepeer-review

29 Scopus citations


Huntington's disease (HD) is a neurodegenerative disorder affecting motor function, personality, and cognition. This paper reviews the experimental data that demonstrate the potential for transplantation of fetal striatum and trophic factor secreting cells to serve as innovative treatment strategies for HD. Transplantation strategies have been effective in replacing lost neurons or preventing the degeneration of neurons destined to die in both rodent and nonhuman primate models of HD. In this regard, a logical series of investigations has proven that grafts of fetal striatum survive, reinnervate the host, and restore function impaired following excitotoxic lesions of the striatum. Furthermore, transplants of cells genetically modified to secrete trophic factors such as nerve growth factor protect striatal neurons from degeneration due to excitotoxicity or mitochondrial dysfunction. Given the disabling and progressive nature of HD, coupled with the absence of any meaningful medical therapy, it is reasonable to consider clinical trials of neural transplantation for this disease. Fetal striatal implants will most likely be the first transplant strategy attempted for HD. This paper describes the variable parameters we believe to be critical for consideration for the design of clinical trials using fetal striatal implants for the treatment of HD.

Original languageEnglish (US)
Pages (from-to)339-352
Number of pages14
JournalCell Transplantation
Issue number2
StatePublished - 1996
Externally publishedYes


  • Brain tissue transplantation
  • Clinical trials
  • Huntington's disease
  • Nerve growth factor
  • Review

ASJC Scopus subject areas

  • Biomedical Engineering
  • Cell Biology
  • Transplantation


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