Therapeutic application of intrabodies against age-related neurodegenerative disorders

Alessio Cardinale, Daniela Merlo, Paolo Giunchedi, Silvia Biocca

Research output: Contribution to journalArticle

Abstract

Many neurodegenerative diseases, referred to as misfolding diseases, are characterized by the formation and accumulation of pathological extracellular and intracellular misfolded aggregates. Ageing is considered the major risk factor for neurodegenerative disorders and, due to increase of mean lifespan, the clinical relevance is growing dramatically with a urgent need to find new effective therapeutic approaches. The intracellular antibody technology is a gene-based strategy which exploits the specificity of recombinant antibodies to neutralize or modify the function of intracellular and extracellular target antigens. Intrabodies can potentially recognize all the pathological conformers of a misfolding-prone protein, and therefore they are emerging as therapeutic agents for the treatment of misfolding diseases as well as molecular tools for the understanding of their pathogenesis. Here we focus on the application of intrabodies against two major age-related neurodegenerative disorders, Alzheimer’s disease (AD) and Parkinson’s disease (PD) and the description of in vivo gene delivery systems available for their potential entering in the clinical setting

Original languageEnglish
Pages (from-to)6028-6036
Number of pages9
JournalCurrent Pharmaceutical Design
Volume20
Issue number38
Publication statusPublished - Jan 1 2014

Keywords

  • Gene delivery
  • Intrabody
  • Nanotechnology
  • Neurodegenerative diseases
  • ScFv

ASJC Scopus subject areas

  • Drug Discovery
  • Pharmacology
  • Medicine(all)

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