Human Diseases Targeted for Gene Therapy, Single genes, Heterozygous condition, Homozygous condition, Dominant allele

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Gene Theraphy
Gene Theraphy Introduction
Human Diseases Targeted for Gene Therapy
Some Human Diseases Targeted for gene therapy
Vectors and Other Delivery Systems for Gene therapy
Viruses as vectors
Retrovirus (RV) as a vector
Adenovirus (AV) as a vector
Adeno associated virus (AAV) as a vector
Lentiviruses as a vector
Non-viral DNA delivery systems
Synthetic particles as vectors
Transient Expression for gene therapy
Splicesome mediated trans-RNA splicing (SMART)
Ex Vivo vs In Vivo Gene Therapy
Target Tissue of Choice for Gen-Delivery System

	Human Diseases Targeted for Gene Therapy Human diseases, that are targeted for gene therapy, may be due to defects in single genes or may be acquired under a variety of circumstances due to genetic predisposition to these diseases. Diseases belonging to both these classes can be treated through gene therapy. Single gene controlled diseases are transferred from generation to generation. The genes for these diseases are generally carried in heterozygous condition in the normal individuals and the disease is caused by the recessive allele in homozygous condition. If the disease is caused by the dominant allele. gene therapy becomes difficult, because, not only a normal allele needs to be introduced, but the defective dominant
allele needs to be removed. The heritable Mendelian diseases include phenylketonuria, haemophilia, cystic fibrosis (CF), etc. Acquired diseases include diseases, which are caused mainly due to somatic mutations in some genes, so that they are caused due to specific genes, but seldom transmitted to the germ line. Majority of cancers belong to this class, although heritable cancers are also known now. Acquired diseases are also caused due to genes, which predispose an individual to a disease, like cancer, coronary artery disease, Alzheimer's disease, etc. These diseases are sometimes Mendelian, but are more often caused by a combination of genetic predisposition and environmental factors, or even solely due to environmental factors.
For instance, among heart diseases, a small proportion (e.g. type III hypercholesterolaemia) is caused due to a deletion or mutation either in the gene encoding low density lipoprotein (LDL) receptor, which imports cholesterol into the cell, or in a gene encoding carrier protein apolipoprotein B. But most heart attacks are caused due to a mixture of genetic and environmental causes, which means that there is a predisposition to heart disease, which requires environmental triggers or abuse, to become apparent. Knowledge about such predisposition through genetic testing in such cases may enable the predisposed person to modify the environment. Somatic gene therapy can be used for all the above. classes of diseases.