About this Research Topic
The improvement of the process of gene discovery in PIDs has led to the identification of novel pathogenic mechanisms and candidate targets to develop precision treatments. Targeted therapies are particularly effective for the treatment of hyperimmune conditions and may be successful in previously incurable conditions. Moreover, the use of targeted therapies may reduce the incidence of side effects compared to conventional treatments. For example, the use of small molecule inhibitors, such as Leniolisib and Ruxolitinib, or the use of immunomodulators, such as Abatacept, have been proven to be effective in reducing the recurrence of infections, in preventing lymphoma development and in treating refractory autoimmune manifestations in patients with Activated PI3K delta syndrome (APDS), STAT1 gain of function mutations and CTLA-4/LRBA deficiency, respectively. Moreover, these targeted treatments may help in reducing the use of steroids and potent immunosuppressive drugs in patients with PIDs with immune-dysregulation, in turn reducing the incidence of adverse drug events. Similarly, using gene therapy in different PIDs prevents the procedure related mortality and the risk of graft versus host disease and solves the problem of donor availability.
This Research Topic is focused to provide a comprehensive overview of the latest research on novel therapeutic approaches to primary immunodeficiencies. We seek Original Research, Reviews, Mini‐Reviews, Methods, and Perspective articles addressing, although not limited to, the following topics:
1. Treating primary immunodeficiencies in the era of precision medicine
2. Gene editing and gene therapy in PIDs
3. Small molecules in PIDs
4. Novel therapeutic approaches to Familial HLH and its neurological manifestations
5. Regenerative medicine in PIDs: where are we?
Important Note: All contributions to this Research Topic must be within the scope of the section and journal to which they are submitted, as defined in their mission statements. Frontiers reserves the right to guide an out-of-scope manuscript to a more suitable section or journal at any stage of peer review.