Gene Therapy

Background:



Human gene therapy is a procedure that is being used in an attemt to treat genetic and other diseases (Anderson,1992).

It is obviously seen that,there is direct relation between gene therapy and genetic diseases e.g. Cystic fibrosis (Bragonzi, et al 2002), but what is amazing is that, gene therapy could also be applied for other diseases which of no genetical basis,e.g. diabetes (Xu, et al 2003),.
Ischemic brain diseases(Saitoh, et al 2003), Cancer(Andres,et al 1992),disordes of the bone marrow,liver,and central nervous system,and deficiences of circulating enzymes,hormones,and coagulation factors(Friedmann,1989).

From a historical point of view,the concept of gene therapy arose during the 1960s and early 1970s (Friedmann,1992).
In 1979,the first clinical trial for gene transfer was conducted by French Anderson who is now considered as the pioneer of gene therapy(Anderson,1980).

The first gene therapy journal ,Human Gene Therapy,began publication on january 1990.On September 14.1990,the first approved gene therapy clinical trial was applied on Ashanthi DeSilva, a 4 year old girl with ADA-deficient Severe Combined Immunodeficiency by National Institute Of   Health team of Anderson/Blaese/Rosenberg(Blaese, et al 1995).

Theoretically, gene therapy can involve gene replacement, correction, or augmentation (Robert,et al 1999).
There are two main types of gene therapy:
Stem cell gene therapy,stem cells are multipotent cells that differentiate into a variety of cells.
Somatic cell gene therapy,somatic cells are the cells being completely differentiated (Ledley,et al 1992).

Delivering genes into a group of cells in a patient,s body could be done using one of two ways:
In vivo technique,where the genetic material is injected directly into the body and specifically target affected cells.
Ex vivo technique , is to deliver the gene to cells while they are outside the body and then their delivery again to the body (Hudock,1973).... [continues]

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