Alana Yang Rose Hills
An Assay to Characterize Mutations and Cell Therapies for ART-SCID
The development of programmable nuclease CRISPR-Cas9 has given scientists the opportunity to correct disease-causing genes in patients with unprecedented precision. Building upon these advancements, my collaborators and I are developing a non-viral CRISPR-Cas therapeutic platform for rare inherited immune diseases. Our current focus is the specific application of this platform approach to treating Artemis-deficient severe combined immunodeficiency (ART-SCID), which is the most difficult form of SCID to treat. Furthermore, the current method of diagnosis for ART-SCID is a radiosensitivity assay that takes 6-8 weeks–far too long for patients whose life expectancies can be a mere 6 months. For my ongoing research project, I am leading the development of a functional assay for Artemis activity to both experimentally validate our cell therapy and streamline diagnosis for novel mutations causing ART-SCID.
Message To Sponsor
Thank you for your support of my summer research! It's thanks to you that I have the opportunity to work on life-saving applications of science and pursue what I love to do the most. I am very grateful for your generosity.