Gene-Based Antibody Strategies for Prion Diseases

Prion diseases or transmissible spongiform encephalopathies (TSE) are a group of neurodegenerative and infectious disorders characterized by the conversion of a normal cellular protein PrPC into a pathological abnormally folded form, termed PrPSc. There are neither available therapies nor diagnostic...

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Main Authors: Alessio Cardinale, Silvia Biocca
Format: Article
Language:English
Published: Wiley 2013-01-01
Series:International Journal of Cell Biology
Online Access:http://dx.doi.org/10.1155/2013/710406
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author Alessio Cardinale
Silvia Biocca
author_facet Alessio Cardinale
Silvia Biocca
author_sort Alessio Cardinale
collection DOAJ
description Prion diseases or transmissible spongiform encephalopathies (TSE) are a group of neurodegenerative and infectious disorders characterized by the conversion of a normal cellular protein PrPC into a pathological abnormally folded form, termed PrPSc. There are neither available therapies nor diagnostic tools for an early identification of individuals affected by these diseases. New gene-based antibody strategies are emerging as valuable therapeutic tools. Among these, intrabodies are chimeric molecules composed by recombinant antibody fragments fused to intracellular trafficking sequences, aimed at inhibiting, in vivo, the function of specific therapeutic targets. The advantage of intrabodies is that they can be selected against a precise epitope of target proteins, including protein-protein interaction sites and cytotoxic conformers (i.e., oligomeric and fibrillar assemblies). Herein, we address and discuss in vitro and in vivo applications of intrabodies in prion diseases, focussing on their therapeutic potential.
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publishDate 2013-01-01
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spelling doaj-art-75392c4ea4bf4a6b8deac40fbd3067b32025-08-20T03:33:34ZengWileyInternational Journal of Cell Biology1687-88761687-88842013-01-01201310.1155/2013/710406710406Gene-Based Antibody Strategies for Prion DiseasesAlessio Cardinale0Silvia Biocca1IRCCS San Raffaele Pisana, Via di Val Cannuta 247, 00166 Rome, ItalyDepartment of Systems Medicine, University of Rome “Tor Vergata,” Via Montpellier 1, 00133 Rome, ItalyPrion diseases or transmissible spongiform encephalopathies (TSE) are a group of neurodegenerative and infectious disorders characterized by the conversion of a normal cellular protein PrPC into a pathological abnormally folded form, termed PrPSc. There are neither available therapies nor diagnostic tools for an early identification of individuals affected by these diseases. New gene-based antibody strategies are emerging as valuable therapeutic tools. Among these, intrabodies are chimeric molecules composed by recombinant antibody fragments fused to intracellular trafficking sequences, aimed at inhibiting, in vivo, the function of specific therapeutic targets. The advantage of intrabodies is that they can be selected against a precise epitope of target proteins, including protein-protein interaction sites and cytotoxic conformers (i.e., oligomeric and fibrillar assemblies). Herein, we address and discuss in vitro and in vivo applications of intrabodies in prion diseases, focussing on their therapeutic potential.http://dx.doi.org/10.1155/2013/710406
spellingShingle Alessio Cardinale
Silvia Biocca
Gene-Based Antibody Strategies for Prion Diseases
International Journal of Cell Biology
title Gene-Based Antibody Strategies for Prion Diseases
title_full Gene-Based Antibody Strategies for Prion Diseases
title_fullStr Gene-Based Antibody Strategies for Prion Diseases
title_full_unstemmed Gene-Based Antibody Strategies for Prion Diseases
title_short Gene-Based Antibody Strategies for Prion Diseases
title_sort gene based antibody strategies for prion diseases
url http://dx.doi.org/10.1155/2013/710406
work_keys_str_mv AT alessiocardinale genebasedantibodystrategiesforpriondiseases
AT silviabiocca genebasedantibodystrategiesforpriondiseases