Recent advanced protocols on cell reprogramming for the generation of
human induced pluripotent stem cells (hiPSCs) has improved the comprehension of the
pathogenic mechanisms and the development of new drugs. In fact, disease-specific
pluripotent stem cells offer an ideal platform for both cell and gene therapy protocol
applications and represent a good possibility for new and personalized pharmacological
treatments. Without any doubt, the most innovative therapies are those which provide a
site specific gene correction, and are suitable to those diseases for which a drug’s
therapy is not available. In the last decade have emerged ZFNs, TALENs, and the
CRISPR/Cas9 system, tools for genome engineering, consisting of a sequence-specific
DNA-binding domain and a non-specific DNA cleavage domain, that allow to correct
mutated genes in vitro.
In this chapter, we focus on hiPSCs as a target cells for gene manipulation: new
strategies as Zinc-finger nucleases, TALENs and CRISPR/ Cas9 have been developed
to maximize the efficiency of genome editing protocols on human reprogrammed cells.
Indeed, humanized iPSCs-based disease model systems exploit an individualized cellbased
platform that has unlimited growth potential for novel regenerative strategies and
clinical therapeutics, along with companion diagnostics, to predict and prognosticate
the molecular basis of various human diseases.
Keywords: CRISPR/Cas9, Gene editing, HESCs, hiPSCs, Human diseases,
TALENs, ZFN.
