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Hereditary Retinopathies: Progress in Development of Genetic and Molecular Therapies: SpringerBriefs in Genetics

Autor Pete Humphries, Marian M. Humphries, Lawrence C. S. Tam, G. Jane Farrar, Paul F. Kenna, Matthew Campbell, Anna-Sophia Kiang
en Limba Engleză Paperback – 4 aug 2012
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 (RetNet). The extensive genetic heterogeneity associated with autosomal dominant RP (adRP) is an undisputed hindrance to the development of genetically based therapeutics.
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Specificații

ISBN-13: 9781461444985
ISBN-10: 1461444985
Pagini: 55
Ilustrații: VII, 46 p. 8 illus., 5 illus. in color.
Dimensiuni: 155 x 235 x 10 mm
Greutate: 0.09 kg
Ediția:2012
Editura: Springer
Colecția Springer
Seria SpringerBriefs in Genetics

Locul publicării:New York, NY, United States

Public țintă

Research

Cuprins

Preface.- Introduction.- Gene-based Medicines directly targeting genetic defects and molecular pathologies common to multiple forms of disease.- Molecular Medicines.

Caracteristici

Provides a snap-shot of current gene and drug-based therapies for hereditary retinal disorders The authors explore emerging topics such as novel gene or drug delivery methods to the retina and retinal diseases caused by protein misfolding and aggregation Describes current developments and future prospects for the application of gene therapy Includes supplementary material: sn.pub/extras Includes supplementary material: sn.pub/extras