**3.20 Other diseases**

118 Non-Viral Gene Therapy

These 5 mouse models of Fanconi anemia will be used to prompt the research of gene therapy of these 5 subtypes of Fanconi anemia, because we can use these mouse models to do animal experiments. Normal genomic DNA or normal DNA fragments can be microinjected/electrotransfered, and transfected into stem cells and somatic cells from a mutated mouse; the corrected stem cells and somatic cells can be transplanted back to the same mouse, to see if the Fanconi anemia mouse model's physiological function is improved

Sickle-cell anemia is an autosomal recessive genetic disease. It results from a mutation at the sixth codon of the β hemoglobin gene on chromosome 11 (the hydrophilic amino acid glutamic acid is replaced by the hydrophobic amino acid valine). This mutation causes red blood cells to become rigid and inflexible. The patient's red blood cells are difficult to go through small capillaries, leading to stroke, chronic pain, anemia, and infection. This disease affects more than 300, 000 people worldwide (Ataga, 2009; Chang et al., 2006;

Cystic fibrosis is a common autosomal recessive genetic disease caused by mutations of cystic fibrosis transmembrane conductance regulator (CFTR) gene on chromosome 7 in Caucasian population (Kerem et al., 1989; Riordan et al., 1989; Rommens et al., 1989). CFTR is a cAMP-regulated chloride channel; the CFTR gene mutations lead to the cAMP-induced chloride channel dysfunction, thereby alter the transport of chloride and associated liquid, cause problems in several organ systems including respiratory system, sweat glands, pancreas, intestine, liver and gallbladder. There are more than 1800 CFTR gene mutations in the world. Cystic fibrosis affacts more than 70, 000 individuals worldwide. In 2006, the median survival age for a person with cystic fibrosis was 37 (M. Anderson et al., 1991; Collaco & Cutting, 2008; Collins, 1992; Cutting, 2010; Lee et al., 2005; Rowntree & Harris,

Duchenne muscular dystrophy (DMD) is the most common form of muscular dystrophy, it affacts about one of every 3500 males. DMD is an X-linked recessive muscle degenerative disease caused by the mutations of dystrophin. As the above stated, the DMD gene is the largest human gene (>2.4 million bp on chromosome X), its cDNA is 14 kb long. DMD gene encodes a single 427 kDa protein-dystrophin. Patient's muscle fibers do not have the 427 kDa dystrophin (Burghes et al., 1987; Campeau et al., 2001; Hoffmanet al., 1987; Koenig et

Huntington's disease (HD) was first described by George Huntington in 1872. HD is an autosomal dominant neurodegenerative disease caused by the mutation of the huntingtin (HTT) gene. HTT gene located at 4p16.3; it has longer CAG trinucletide repeats (more than 40 CAG repeats) in the first exon of the HTT gene than the normal gene. There are transgenic mouse, sheep and monkey models available for conducting animal experiments currently (Bates et al., 1997; Beilby, 2007; S. Davies & Ramsden, 2001; Jacobsen et al., 2010;

al., 1987, 1988; Kunkel, 2004; Monaco et al., 1986; Nelson et al., 2009).

by this kind of gene therapy.

Ingram, 1956, 1957; Pawliuk et al., 2001; Wu et al., 2006).

2003; G. Wang et al., 2005; Zielenski, 2000).

**3.15 Duchenne muscular dystrophy** 

MacDonald et al., 1993; Yang et al., 2008).

**3.16 Huntington's disease** 

**3.13 Sickle-cell anemia**

**3.14 Cystic fibrosis** 

This possible gene therapy method also might be used to cure other diseases such as Alzheimer's disease (Rogaev et al., 1995; Sherrington et al., 1995), Parkinson's disease (Terzi & Zachariou, 2008; Veeriah et al., 2010), X-chronic granulomatous disease (CGD) (Aiuti & Roncarolo, 2009; Kang et al., 2010), type I (insulin-dependent) (Efrat, 1998) and type II (noninsulin-dependent) (Freeman et al., 1999) diabetes.
