A Big Hope for Bubble Boys

A new groundbreaking study, led by Dr. Ayal Hendel from the Mina and Everard Goodman Faculty of Life Sciences at Bar-Ilan University, in collaboration with researchers from Tel Aviv University and senior physicians from Sheba Hospital in Tel Hashomer, suggests an innovative genetic treatment strategy for a severe genetic disorder – Severe Combined Immunodeficiency (SCID), also known as "bubble boy disease". SCID is a general name for a group of diseases, in which a defect in a single gene causes a failure in T-cell development (a certain type of white blood cells), which severely impedes the immune system's ability to respond to pathogens. In the study, published in the prestigious journal Molecular Therapy Nucleic Acids, by using CRISPR gene editing technology, researchers developed a model for SCID in immune progenitor cells. Next, by using CRISPR, researchers repaired the mutant gene that caused the SCID , both in the model they developed and in immune progenitor cells taken from a patient, and obtained normal T-cells. The SCID model developed by Dr. Hendel and his colleagues will serve as an important platform for the SCID study, while the gene correction that the researchers achieved is a proof-of-concept for SCID gene therapy by using CRISPR.

Read more >>