Family Updates

The most important family update is gene therapy.


In the last several years, recombinant adenoviruses have been used as delivery vectors with curative and preventative purposes. These vectors have been used in experimental gene therapy for genetic disorders, in immuno and molecular therapy for cancers, and as experimental candidates for vaccination against infectious diseases. To do this, several changes must be made to the genome. The early region E3 is deleted completely, as it is not essential for viral replication. This makes room for the eukaryotic delivery gene of interest. Also, the E1 region is mutated (truncated) to offer further space. A good example of how this works involves the genetic disease cystic fibrosis. The gene, 'cystic fibrosis conductance regulator' (CFTR) is mutated in patients suffering from this disease, causing sever symptoms in the respiratory and gastrointestinal tracts due largely to the increased viscosity of fluids. This gene can be delivered via an adenovirus, thereby facilitating the synthesis of CFTR in the infected host cell. A similar approach, often combined with chemotherapy, is becoming increasingly promising for cancer patients as well.