The potential of intracellular antibodies for therapeutic targeting of protein-misfolding diseases.
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.
Cardinale
Alessio
A
IRCCS San Raffaele, Rome, Italy.
Biocca
Silvia
S
D000818:Animals; D000906:Antibodies / Q000008:administration & dosage / Q000627:therapeutic use*; D001927:Brain Diseases / Q000188:drug therapy* / Q000378:metabolism; D006801:Humans; D042541:Intracellular Space / Q000276:immunology*; D015202:Protein Engineering; D017510:Protein Folding
D000906:Antibodies