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Abstract
Systemic amyloidosis occurs when one of a growing list of circulating proteins acquires
an abnormal fold, aggregates and gives rise to extracellular amyloid deposits in different
body sites, leading to organ dysfunction and eventually death. Current approaches
are mainly aimed at lowering the supply of the amyloidogenic precursor or at stabilizing
it in a non-amyloidogenic state, thus interfering with the initial phases of amyloid
formation and toxicity. Areas covered: Improved understanding of the pathophysiology
is indicating novel steps and molecules that could be therapeutically targeted. Here,
we will review emerging molecular targets and therapeutic approaches against the main
forms of systemic amyloidosis at the early preclinical level. Expert opinion: Conspicuous
efforts in drug design and drug discovery have provided an unprecedented list of potential
new drugs or therapeutic strategies, from gene-based therapies to small molecules
and peptides, from novel monoclonal antibodies to engineered cell-based therapies.
The challenge will now be to validate and optimize the most promising candidates,
cross the bridge from the preclinical phase to the clinics and identify, through innovative
trials design, the safest and most effective combination therapies, striving for a
better care, possibly a definitive cure for these diseases.
Abbreviated Title:
Expert Opinion on Therapeutic Targets
Publisher:
Informa UK Limited
ISSN
(Print):
1472-8222
ISSN
(Electronic):
1744-7631
Publication date Created:
October
30 2017
Publication date Created:
December
02 2017
Publication date
(Electronic):
November
06 2017
Publication date
(Print):
December
02 2017
Volume: 21
Issue: 12
Pages: 1095-1110
Affiliations
[1
] Amyloidosis Research and Treatment Center, Foundation IRCCS Policlinico San Matteo,
Department of Molecular Medicine, University of Pavia, Pavia, Italy