4.3.3. Gene therapy

Principles of stem cell biology and cancer: future applications and therapeutics. Edited by T. Regad, T. J. Sayers and R. C. Rees. John Wiley & Sons (2015)

Part I. Stem Cells


HSCs are a favourite target for gene therapy, due to the fact that they are well characterized and are routinely transplanted in clinical settings. Gene therapy – the ability to change the genetic makeup of specific cells, either by introduction of genes or, more recently, by the removal (inactivation) or alteration of genes – has developed through numerous clinical trials during the past 40 years (Gillet et al., 2009). Much progress has been made during that time, but setbacks have slowed the process and continue to drive the need for further improvements. The field continues to show great potential, however (Kay, 2011). The large strides made in vector design and gene control will enable much of the use currently anticipated. Technical developments continue to increase our options. Especially important in this respect is the increasingly routine use of whole-genome sequencing techniques, which allow for exact identification of mutated genes or control regions. The ongoing development of advanced techniques such as nucleotide editing at the genome level using oligonucleotides or CRISPR/Cas (Aarts and te Riele, 2011; Wang et al., 2013; Wei et al., 2013) allows for far more precise approaches to gene therapy. Developments like these mean that gene therapy, even after 40 years of work, continues to hold great promise for the expansion of the clinical use of HSCs, both in treating cancers and in other indications.

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