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

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.

• Authors: Alessio Cardinale, Silvia Biocca
• Journal: Trends in molecular medicine (Trends Mol Med) Vol. 14 Issue 9 Pg. 373-80 (Sep 2008) ISSN: 1471-4914 [Print] England
• PMID: 18693139 (Publication Type: Journal Article, Review)

Chemical References

• Antibodies

Topics

• Animals
• Antibodies (administration & dosage, therapeutic use)
• Brain Diseases (drug therapy, metabolism)
• Humans
• Intracellular Space (immunology)
• Protein Engineering
• Protein Folding