Overview of the current status of gene therapy for primary immune deficiencies (PIDs)

Abstract

Over 3 decades, gene therapy has advanced from a logical idea to becoming a clinical reality for several of the most severe primary immune deficiencies, as well as other inherited disorders. The first gene therapy medicines have been licensed for marketing and several more are advancing toward that goal to make them widely available, beyond clinical trials. Although common platforms of cells, vectors, or editing reagents are used for these disorders, each individual genetic cause of an immune deficiency requires its own vector or editing tools and a package of preclinical data on efficacy and safety to initiate clinical trials. One-by-one, gene therapy for primary immune deficiencies is being brought to the clinic and hopefully will provide safe and effective therapies.

Cavitation is the determining mechanism for the atomization of high-viscosity liquid

“…In recent years, the introduction of exogenous normal genes into target cells by gene transfer technology to treat diseases caused by genetic abnormalities has been called gene therapy technology, which can treat difficult diseases such as cancer, thalassemia, and autoimmune diseases. Gene therapy has become the development trend of modern medicine, 39 , 40 , 41 , 42 , 43 and inhaled gene therapy is the current research area in the field of inhaled medication delivery. 44 , 45 , 46 Under the single-dimensional energy supply, achieving a high energy supply that is necessary for the atomization of high-viscosity liquids is difficult.…”