Kaposiform Lymphangiomatosis (KLA) is a rare, life-threatening disease where abnormal lymphatic vessels grow throughout the body, causing bleeding disorders and fluid buildup around the heart and lungs. KLA is caused by a single genetic mistake in a small fraction of blood vessel cells. Current treatments can slow the disease but cannot cure it, leaving patients dependent on medications for life.
I aim to develop a gene therapy that finds and fixes only the cells carrying this genetic error while leaving healthy cells untouched. My approach combines three targeting layers: custom-designed genetic switches that turn on only in diseased cells, delivery particles that specifically seek out KLA cells, and a gene editor that corrects the typo. I will use artificial intelligence to design these genetic switches, test which cell surface markers can guide delivery particles to the right cells, and optimize the gene editor to fix the mutation safely.
This research will provide a one-time cure for KLA patients instead of lifelong treatment. The platform will also be adapted for other diseases caused by genetic errors in scattered cells, including certain cancers and other vascular disorders.