Misfolding diseases are a wide group of devastating disorders characterized by the accumulation of pathological protein aggregates. Although these disorders still lack an effective treatment, new antibody-based strategies are emerging and entering clinical trials. The intrabody approach is a gene-based technology developed to neutralize or modify the function of intracellular and extracellular target antigens. Because intrabodies can potentially target all the different isoforms of a misfolding-prone protein, including pathological conformations, they are emerging as therapeutic molecules for the treatment of misfolding diseases, including Alzheimer's, Parkinson's, Huntington's and prion diseases. This review will provide a description of the intrabody approach, an update of preclinical studies on misfolding diseases and an outlook on the intrabody delivery issue for therapeutic purposes. © 2008 Elsevier Ltd. All rights reserved.

Cardinale, A., Biocca, S. (2008). The potential of intracellular antibodies for therapeutic targeting of protein-misfolding diseases. TRENDS IN MOLECULAR MEDICINE, 14(9), 373-380 [10.1016/j.molmed.2008.07.004].

The potential of intracellular antibodies for therapeutic targeting of protein-misfolding diseases

BIOCCA, SILVIA
2008-01-01

Abstract

Misfolding diseases are a wide group of devastating disorders characterized by the accumulation of pathological protein aggregates. Although these disorders still lack an effective treatment, new antibody-based strategies are emerging and entering clinical trials. The intrabody approach is a gene-based technology developed to neutralize or modify the function of intracellular and extracellular target antigens. Because intrabodies can potentially target all the different isoforms of a misfolding-prone protein, including pathological conformations, they are emerging as therapeutic molecules for the treatment of misfolding diseases, including Alzheimer's, Parkinson's, Huntington's and prion diseases. This review will provide a description of the intrabody approach, an update of preclinical studies on misfolding diseases and an outlook on the intrabody delivery issue for therapeutic purposes. © 2008 Elsevier Ltd. All rights reserved.
2008
Pubblicato
Rilevanza internazionale
Articolo
Sì, ma tipo non specificato
Settore BIO/12 - BIOCHIMICA CLINICA E BIOLOGIA MOLECOLARE CLINICA
English
Con Impact Factor ISI
alpha synuclein; alpha synuclein antibody; amyloid beta protein; amyloid beta protein antibody; amyloid beta protein[1-16] antibody; amyloid beta protein[1-40] antibody; amyloid beta protein[1-42] antibody; amyloid precursor protein antibody; cationic peptide; huntingtin; huntingtin antibody; immunoliposome; intracellular antibody; liposome; monoclonal antibody; parvovirus vector; peptide; polymer; prion protein; prion protein antibody; protein antibody; recombinant antibody; single chain fragment variable antibody; small interfering RNA; tau protein; unclassified drug; Alzheimer disease; amino terminal sequence; antibody engineering; antigen recognition; article; atomic force microscopy; blood brain barrier; carboxy terminal sequence; cell based gene therapy; down regulation; electron microscopy; gene expression regulation; gene mutation; human; Huntington chorea; immunotherapy; liposomal gene delivery system; neuropathology; neurotoxicity; nonhuman; nonviral gene delivery system; oculopharyngeal muscular dystrophy; Parkinson disease; prion disease; protein folding; protein targeting; recombinant DNA technology; RNA interference; scrapie; viral gene delivery system; Animals; Antibodies; Brain Diseases; Humans; Intracellular Space; Protein Engineering; Protein Folding
Cardinale, A., Biocca, S. (2008). The potential of intracellular antibodies for therapeutic targeting of protein-misfolding diseases. TRENDS IN MOLECULAR MEDICINE, 14(9), 373-380 [10.1016/j.molmed.2008.07.004].
Cardinale, A; Biocca, S
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/2108/45784
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