Understanding HSP genetics to translate into drug therapy

A positive projection for the future has been made by a Miami researcher based on a review of current and emerging understanding of the mechanisms of HSP

Hereditary spastic paraplegia (HSP) comprises a group of clinically and genetically heterogeneous diseases that affect the upper motor neurons and their axonal projections. A total of 30 chromosomal loci have been identified for autosomal dominant, recessive and X-linked HSP. The underlying genes for 15 of these loci have been described.

The molecular dissection of the cellular functions of the related gene products has already greatly advanced our understanding of the most critical pathways involved in HSP.

It is hoped that in the foreseeable future this knowledge will begin to translate into novel pharmacological approaches for this devastating disease.

SOURCE: Expert Opin Pharmacother. 2007 Jul;8(10):1433-9. Review.

The genetics of hereditary spastic paraplegia and implications for drug therapy.

Züchner S.

University of Miami Miller School of Medicine, Miami Institute of Human Genomics, Miami, FL 33101, USA. [email protected]

PMID: 17661726 [PubMed – indexed for MEDLINE]