Proposal 6 Rescue Of An Immunedeficient System Via Gene Therapy

It is well known that immune deficiency, such as HIV, is the disease of greatest concern to the public at the present time. Many important proteins, such as the CD families, have been discovered to play a significant role in the deficient immune system. In order to increase the immune capabilities of patients, gene therapy has been established. Due to the great public interest in this type of therapy, research funding is virtually unlimited. Therefore, one may switch to this new research avenue that involves several collaborators. In view of recent advances in molecular biology, a proposal regarding gene therapy will be a very good approach. Specifically, one may transfect human cells such as bone marrow stem cells with overexpression of sense cDNA constructs. Stably transfected cells that constitu-tively express proteins (e.g., CD4) can be directly injected into the immune system of the patient. An alternative is to overexpress and purify a large amount of the proteins in bacteria or yeast, and then inject an appropriate dose of the protein molecules into the patient to activate the immune system using appropriate methodologies. Figure 1.6 illustrates the relevant research design strategies and methods covered in this book.

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