Dataset Information

The Current Status of Neural Grafting in the Treatment of Huntington's Disease. A Review.

ABSTRACT: Huntington's disease (HD) is a devastating, fatal, autosomal dominant condition in which the abnormal gene codes for a mutant form of huntingtin that causes widespread neuronal dysfunction and death. This leads to a clinical presentation, typically in midlife, with a combination of motor, psychiatric, cognitive, metabolic, and sleep abnormalities, for which there are some effective symptomatic therapies that can produce some transient benefits. The disease, though, runs a progressive course over a 20-year period ultimately leading to death, and there are currently no proven disease modifying therapies. However whilst the neuronal dysfunction and loss affects much of the central nervous system, the striatum is affected early on in the disease and is one of the areas most affected by the pathogenic process. As a result the prospect of treating HD using neural transplants of striatal tissue has been explored and to date the clinical data is inconclusive. In this review we discuss the rationale for treating HD using this approach, before discussing the clinical trial data and what we have learnt to date using this therapeutic strategy.

SUBMITTER: Wijeyekoon R

PROVIDER: S-EPMC3232470 | biostudies-literature | 2011

REPOSITORIES: biostudies-literature

ACCESS DATA

Publications

The Current Status of Neural Grafting in the Treatment of Huntington's Disease. A Review.

Wijeyekoon Ruwani R Barker Roger A RA

Frontiers in integrative neuroscience 20111207

Huntington's disease (HD) is a devastating, fatal, autosomal dominant condition in which the abnormal gene codes for a mutant form of huntingtin that causes widespread neuronal dysfunction and death. This leads to a clinical presentation, typically in midlife, with a combination of motor, psychiatric, cognitive, metabolic, and sleep abnormalities, for which there are some effective symptomatic therapies that can produce some transient benefits. The disease, though, runs a progressive course over ...[more]

PMID: 22162966

Similar Datasets

Project description:Therapeutic drug monitoring (TDM) receives growing interest in different psychiatric clinical settings (emergency, inpatient, and outpatient services). Despite its usefulness, TDM remains underemployed in mental health. This is partly due to the need for evidence about the relationship between drug serum concentration and efficacy and tolerability, both in the general population and even more in subpopulations with atypical pharmacokinetics. This work aims at reviewing the scientific literature published after 2017, when the most recent guidelines about the use of TDM in mental health were written. We found 164 pertinent records that we included in the review. Some promising studies highlighted the possibility of correlating early drug serum concentration and clinical efficacy and safety, especially for antipsychotics, potentially enabling clinicians to make decisions on early laboratory findings and not proceeding by trial and error. About populations with pharmacokinetic peculiarities, the latest studies confirmed very common alterations in drug blood levels in pregnant women, generally with a progressive decrease over pregnancy and a very relevant dose-adjusted concentration increase in the elderly. For adolescents also, several drugs result in having different dose-related concentration values compared to adults. These findings stress the recommendation to use TDM in these populations to ensure a safe and effective treatment. Moreover, the integration of TDM with pharmacogenetic analyses may allow clinicians to adopt precise treatments, addressing therapy on an individual pharmacometabolic basis. Mini-invasive TDM procedures that may be easily performed at home or in a point-of-care are very promising and may represent a turning point toward an extensive real-world TDM application. Although the highlighted recent evidence, research efforts have to be carried on: further studies, especially prospective and fixed-dose, are needed to replicate present findings and provide clearer knowledge on relationships between dose, serum concentration, and efficacy/safety.

Dataset Information

The Current Status of Neural Grafting in the Treatment of Huntington's Disease. A Review.

Publications

The Current Status of Neural Grafting in the Treatment of Huntington's Disease. A Review.

Similar Datasets

OmicsDI is part of the ELIXIR infrastructure

Tweets