Gene Therapy Archive

Ribozymes in Gene Therapy

RNA molecules with catalytic ability are known as ribozymes . Some ribozymes act by cleaving other RNA molecules. Such ribozymes have catalytic domains that carry out the reaction fused to domains that recognize the substrate RNA by base pairing. If …

Aggressive Gene Therapy For Cancer

Although most cancers are not inherited via the germ line, cancer is nonetheless a genetic disease. In the case of hereditary disease we may attempt to replace the defective component, thus preventing cell death. In contrast, when dealing with a …

Retrovirus Gene Therapy for SCID

Severe combined immunodeficiency (SCID) occurs when both the B cells and T cells of the immune system are defective and results in an almost totally defective immune response. Children with SCID have to be shielded from all contact with other …

Retrovirus Gene Therapy

Retroviruses infect many types of cells in mammals. They need dividing cells for successful infection, and will not infect many tissues where host cell growth and division have come to a standstill. Moreover, the genetic material of retroviruses passes through …

Adenovirus Vectors in Gene Therapy

Adenoviruses are relatively simple, double-stranded DNA viruses that infect humans and other vertebrates. The virus particle consists of a simple icosahedral shell, or capsid, containing a single linear dsDNA molecule of approximately 36,000 base pairs. A terminal protein protects each end …

Aggressive Gene Therapy

The original idea behind gene therapy was curing hereditary defects by replacing defective genes. However, there is no inherent reason why gene therapy must only be “defensive” and suppress defects. We can go on the offensive and provide genes whose …

Gene Patching by Oligonucleotide Crossover

During replacement gene therapy, we normally think of replacing the whole defective gene with a complete functional copy. However, some genetic defects consist of just a single base change, or perhaps a cluster of closely linked base alterations. In this …

Gene Therapy

Genetic engineering means that we alter an organism permanently so that the changes will be stably inherited. For multicellular organisms this implies deliberate alteration of the DNA in the germline cells. In contrast, gene therapy (occasionally called genetic surgery ) …