Gene Therapy of Some Genetic Diseases by Transferring Normal Human Genomic DNA into Somatic Cells and Stem Cells from Patients
1.1 Viral vectors for gene therapy Gene therapy is a way to correct mutated genes in vivo by transferring normal genes into cells of patients with genetic diseases or cancers, or to introduce new genes into cells to express therapeutic proteins. Several viruses like adenoviruses (Nayak & Herzog, 2010; Raper et al., 2003), alphaviruses (Lundstrom, 2001, 2005), retroviruses (Aiuti et al., 2009; Bordignon et al., 1989, 1995; Cavazzana-Calvo et al., 2000; Ferrari et al., 1991; Halatsch et al., 2000), lentiviruses (Dupre et al., 2004; Mortellaro et al., 2006; Nayak & Herzog, 2010), adeno-associated viruses (AAV) (Jayandharan et al., 2011; Nayak & Herzog, 2010; Terzi & Zachariou, 2008), herpes simplex viruses type 1 (HSV-I) (Epstein, 2009), have been used as vectors to deliver normal genes into cells of patients for gene therapy. However, there were limitations and hurdles in using these vectors. Some viruses like retroviruses, lentiviruses might integrate into human genomic DNA and cause cancers (Dave et al., 2004; Du et al., 2005; Hacein-Bey-Abina et al., 2003a, 2003b; Z. Li et al., 2002; Modlich et al., 2005; Seggewiss et al., 2006). Most viruses can infect both normal cells and defective/cancer cells of patients, as long as the cells have receptors of the viruses (Antar et al., 2009; K. Holmes et al., 1997; Norkin, 1995; L. Song, 2010; L. Song et al., 2009; van den Wollenberg et al., 2008; van Houdt et al., 2008), and this might lead to serious infections, inflammatory responses, and immunological reactions (Nayak & Herzog, 2010).
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