Gene therapy and AIDS

It is likely that gene therapy will prove useful in treating a far broader range of medical conditions than simply those of inherited genetic disease and cancer. A prominent additional disease target are those diseases caused by infectious agents, particularly intracellular pathogens such as HIV. The main strategic approach adopted entails introducing a gene into pathogen-susceptible cells whose product will interfere with pathogen survival/replication within that cell. Such a strategy is sometimes termed 'intracellular immunization'.

One such anti-AIDS strategy being pursued is the introduction into viral-sensitive cells of a gene coding for an altered (dysfunctional) HIV protein, such as gag, tat or env. The presence of such mutant forms of gag, in particular, was shown to be capable of inhibiting viral replication. This is probably due to interference by the mutated gag product with correct assembly of the viral core. An additional approach entails the transfer to sensitive cells of a gene coding for antibody fragments capable of binding to the HIV envelope proteins. This may also interfere with viral assembly in infected cells.

Scientists have also generated recombinant cells capable of synthesizing and secreting soluble forms of the HIV cell surface receptor, i.e. the CD4 antigen. It has been suggested that release of such soluble viral receptors into the blood would bind circulating virions, hence blocking their ability to 'dock' at sensitive cells. Although this proved to be the case in vitro, early in vivo studies have not proved as encouraging. Yet additional therapeutic approaches to AIDS, based upon antisense technology, will be discussed later in this chapter.

Diabetes 2

Diabetes 2

Diabetes is a disease that affects the way your body uses food. Normally, your body converts sugars, starches and other foods into a form of sugar called glucose. Your body uses glucose for fuel. The cells receive the glucose through the bloodstream. They then use insulin a hormone made by the pancreas to absorb the glucose, convert it into energy, and either use it or store it for later use. Learn more...

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