Though my primary focus is on gene transfer, I will occasionally wander into other areas of translational pharmaceutical research, including cell transfer (also sometimes called “cell therapy”), monoclonal antibodies, small-molecule drugs that target novel biological mechanisms, or large-molecule drugs. These types of translational clinical research raise a similar cluster of issues– namely, high degrees of… Continue reading The Scope of this Blog
Tag: Introductory material
Introduction to Lost in Translation
Within the past six months, a fifth patient developed a lympho-proliferative disorder in an X-SCID gene transfer trial. The RAC has all but cleared AAV vector as a causal agent in the death of Jolee Mohr in a rheumatoid arthritis study. A Merck HIV-vaccine trial involving adenoviral vectors was terminated after a data safety monitoring… Continue reading Introduction to Lost in Translation