Corrigendum to “Preclinical Safety and Efficacy of Human CD34+ Cells Transduced With Lentiviral Vector for the Treatment of Wiskott-Aldrich Syndrome”

Human pluripotent stem cells (PSCs) in the form of human embryonic stem cells (hESCs) or induced pluripotent stem cells (iPSCs) are capable of growing indefinitely in vitro, maintaining their capacity to differentiate into the three primary germ layers: mesoderm, endoderm and ectoderm. Different protocols have been developed to differentiate PSCs into almost any cellular type with different degree of success. This technology has allowed scientists to use patient-derived iPSCs to study the physiopathology of the disease by analyzing the phenotype of the cells derived from these iPSCs. However, control iPSCs obtained from healthy individuals will always have different genomic environment than patient's iPSCs, making it difficult the interpretation of the cells phenotype. The recent appearance of specific nucleases [zinc-finger nucleases (ZFNs), the transcription activator-like effector nucleases (TALENs) and the clustered regularly interspaced short palindromic repeats (CRISPR)] has made it possible to edit the genome of PSCs. We can now generate syngeneic hESCs or iPSCs harboring the desired mutation and comparing the emerging cells with those derived from genetically identical PSCs that will differ only in the mutated gene. In this chapter, we summarize the progress made in this field and discuss the different approaches that have been used recently for the generation of syngeneic human pluripotent cellular models for different pathologies.

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Stem Cells for Modeling Human Disease

Martín¹,

Sánchez‐Gilabert²,

Tristán‐Manzano³

et al. 2016

Pluripotent Stem Cells - From the Bench to the Clinic

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Corrigendum to “Preclinical Safety and Efficacy of Human CD34+ Cells Transduced With Lentiviral Vector for the Treatment of Wiskott-Aldrich Syndrome”

Abstract: In Supplementary Table 7, the sequence of the primer hTelo forward was incorrectly listed as 5'-GGA CAC GTT ATT TAC CCT GTT TCG-3'. The correct sequence is 5'-GGC ACA CGT GGC TTT TCG-3' .

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Stem Cells for Modeling Human Disease

Stem Cells for Modeling Human Disease

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