Some Human Diseases Targeted for gene therapy, Cystic Fibrosis, Diabetes mellitus, Pituituary dwarfism, Emphysema

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	Some human diseases targeted for gene therapy. I. Diseases caused by single gene defects shown in parentheses (targets for gene therapy) I. Cystic fibrosis (CFTR); 2. Diabetes mellitus; 3. Pituitary dwarfism (hGH); 4. Emphysema (α-I-antitrypsin); 5. Familial hypercholesterolemia (LDL receptor); 6. Haemoglobinopathies - thalassemia major and sickle cell anaemia - ( β-globin); 7. Haemophilia - A, B (Factor VIII, Factor IX); 8. Lysosomal disease;
9. Gauchers disease (glucocerebrosidase); 10. Glycogen storage diseases; II. Phenylketonuria (phenylalanine hydroxylase); 12. Severe combined immunodeficiency diseases or SCID (ADA); 13. Duchenne muscular dystrophy (dystrophin); 14. CNS disorders - Leisch - Nyhan syndrome (hypoxanthine phosphoribosyl transferase) and Tay-Sachs disease (hexosaminidase). 15. Immunodeficiency (adenosine deaminase); 16. Hyperammonaemia (ornithine transcarbamylase).
II. Diseases acquired due to genetic predisposition (amenable to gene therapy using gene shown in parentheses) I. Cancer (interleukins, TK, tumour suppressor); 2. Cardiovascular diseases - Myocardial infarcts or MI (tPA); prevention of blood clots (tPA). hypercholesterolaemia (LDL receptor) 3. Neurodegenerative diseases - Alzheimer's disease (NGF), Parkinson's disease (TH); 4. Joint disorders - Rheumatoid arthritis (cytokine, IL-l antagonists); 5. Infectious disorders - AIDS (HIV antigens. Cytokines, TK genes, cytomegalovirus or CMV infections).