The Wiskott–Aldrich Syndrome is a complex primary immunodeficiency disorder, and HSC (Hematopoietic stem cells) gene therapy has emerged as an innovative therapeutic strategy for various primary immunodeficiency disorders. The disease arises from the multiple dysfunctions of different subgroups of leukocytes, including defective function of T and B cells, disturbed formation of the NK-cell immunological synapse, and impaired migratory responses in all leukocyte subgroups.

WAS is a suitable candidate for stem cell therapy as the WASP expression is restricted to cells of the hematopoietic system, which grants an advantage over WASP-negative cells. After the therapy, the patient’s frequency and severity of infections decreased.

Advantages of Stem Cell Banking

The umbilical cord is a source of rare and precious cells which have the power of regeneration and potentially save lives from many severe diseases, like WAS. However, stem cell preservation at baby’s birth is a once-in-a-lifetime opportunity and here is why you should not miss it:

• Cord blood stеm cеlls can quickly adapt and have the potential to rеplacе damagеd or malfunctioning cells within thе body.
• The benefits of stem cells don’t end at thе child, but they also еxtеnds to the trеatmеnt to siblings and family members who may sharе a gеnеtic match.
• The stem cells extracted from umbilical cord blood can treat a wide variety of diseases, like cardiovascular, neurodegenerative, autoimmune, and orthopaedic diseases.