The hereditary retinopathy, retinitis pigmentosa (RP), which affects 1 in 3, 500 people worldwide, is the most common cause of registered visual handicap among those of the working age in developed countries. RP is a highly variable disorder where patients may develop symptomatic visual loss in early childhood, while others may remain asymptomatic until mid-adulthood. Most cases of RP segregate in autosomal dominant, recessive or X-linked recessive modes, with approximately 41 genes being implicated in disease pathology to date (Ret Net). The extensive genetic heterogeneity associated with autosomal dominant RP (ad RP) is an undisputed hindrance to the development of genetically based therapeutics.
Tabella dei contenuti
Preface.- Introduction.- Gene-based Medicines directly targeting genetic defects and molecular pathologies common to multiple forms of disease.- Molecular Medicines.
Lingua Inglese ● Formato PDF ● Pagine 46 ● ISBN 9781461444992 ● Dimensione 0.9 MB ● Casa editrice Springer New York ● Città NY ● Paese US ● Pubblicato 2012 ● Scaricabile 24 mesi ● Moneta EUR ● ID 2662770 ● Protezione dalla copia DRM sociale