Gene therapy in the clinic

Clinicians have generally been quick to embrace the concept of gene therapy and are already testing a variety of novel strategies that hinge on the transfer of genetic material. The targeted diseases have already been discussed in the previous sections but, for a variety of reasons, most of the clinical gene therapy protocols that have been approved to date are for the treatment of cancer patients. Most of these early trials are phase I studies performed in poor-prognosis patients with...

Gaagtc

Figure 2.3 Restriction fragment length polymorphisms. DNA polymorphisms provide the major means of discriminating allelic markers. The principles underlying RFLPs are shown. The restriction enzyme EcoRI cuts genomic DNA specifically at the nucleotide sequence GAATTC. At a given genetic locus, variation of a single nucleotide is sufficient to create or destroy an EcoRI restriction site to generate two distinct alleles (A or a). Southern blot analysis using a DNA probe specific for this locus is...