GENE THERAPY, The state of the art

GENE THERAPY
THE STATE OF THE ART

Dr. Abdel Aziz El Bayoumi
Professor of Genetics

Dr. Khalid Al Ali
Lecturer of Genetics

Department of Biological Sciences
University of Qatar, Doha

10. CANDIDATES OF INHERITED DISEASES OF THE LUNG

There are a large number of inherited human diseases. However, not all are subject for gene therapy. The ideal candidate should satisfy the following:

1. It must be caused by a single gene defect.

2. It affects only one cell type which is ready for removal or replacement

3. The gene responsible for the disease must be well identified and cloned.

4. The regulated expression of the gene is not required to correct the defect.

5. The disease represents a significant cause of mortality and no effective conventional treatment is available.

Figure 15 : Production of transgenic mice as a model for gene therapy

Table 1 represents some common inherited diseases that could be candidate for gene therapy. Most of these diseases are controlled by single gene. There are however other diseases that are controlled by more than one gene. These are so far very difficult for gene therapy

Table 1 Some common inherited diseases that could be candidate for gene therapy

Disease	Defective Gene
Sickle cell disease	Beta chain of hemoglobin
Cystic fibrosis	ATP-binding cassette (ABC) family protein
Huntington’s disease	Identified;function unknown
Duchenne’s muscular dystrophy	Dystrophin
Phenylketouria mental retardation	Phenylaianine hydrroxylase
Lesch-Nyhan syndrome	Hypoxanthineguanine – phosphoribosy ltransferase
Gaucher’s disease	Glucocerebrosidase
Tay-Sach’s disease	Alpha chain of lysosomal hexosaminidase A
Galactosemia	Galactose accumulation
Maple syrup urine disease	Amino acids and ketoacid accumulation
Adenosine deaminase (ADA) deficiency	Adenosine deaminase
Thalassemias	Beta chain of hemoglobin
Alphal-antitrypsin deficiency	Alphal- antitrypsin
X-linked chronic granulomatous disease (CGD)	Cytochrome b558 gp91-phox
Autosomal CGD	P47-phox
Familial hypercholesteremia	Receptor for low-density lipoprotein
Ornithine transcarbamylase deficiency	Ormithine transcarbamylase

Modified from, Maulik, S. and S. Potel 1997.

Notice