Neural transplantation in patients with Huntington's disease

Rosser, Anne Elizabeth ORCID: https://orcid.org/0000-0002-4716-4753 and Dunnett, Stephen Bruce ORCID: https://orcid.org/0000-0003-1826-1578 2003. Neural transplantation in patients with Huntington's disease. CNS Drugs 17 (12) , pp. 853-867. 10.2165/00023210-200317120-00001

Full text not available from this repository.

Abstract

The gene for Huntington’s disease was identified in 1993 as being a CAG repeat expansion in exon 1 of a gene now known as huntingtin on chromosome 4. Although many of the downstream effects of this mutant gene were identified in the subsequent years, a more detailed understanding of these events will be necessary in order to design specific interventions to interfere with the disease process and slow disease progression. In parallel, a number of groups have been investigating alternative approaches to treatment of Huntington’s disease, including cell and tissue transplantation. As the brunt of cell dysfunction and loss is borne by the striatum, at least in the early to mid-stages of disease, the goal is to identify methods for replacing lost cells with fetal neuroblasts that can develop, integrate into the host circuitry and thereby restore lost function. Clinical studies in which primary fetal neuroblasts were transplanted into the brains of patients with advanced Parkinson’s disease have demonstrated benefit when the transplant methodology closely follows the biological principles established in animal experiments. On the basis of demonstrated benefit following striatal cell transplantation in animal models of Huntington’s disease, a small number of studies have now commenced in patients with Huntington’s disease. To date, these clinical studies have demonstrated the feasibility and safety of transplantation in this condition, but it will require several more years yet before the efficacy of the procedure can be confidently established

Item Type:	Article
Date Type:	Publication
Status:	Published
Schools:	Biosciences Medicine MRC Centre for Neuropsychiatric Genetics and Genomics (CNGG) Neuroscience and Mental Health Research Institute (NMHRI)
Subjects:	Q Science > QP Physiology R Medicine > R Medicine (General)
Publisher:	Springer
ISSN:	1172-7047
Last Modified:	27 Oct 2022 08:28
URI:	https://orca.cardiff.ac.uk/id/eprint/62401

Citation Data

Cited 38 times in Scopus. View in Scopus. Powered By Scopus® Data

Actions (repository staff only)

Edit Item