Alexa Garcia
Alexa Garcia is a second year undergraduate student at UCLA majoring in Molecular, Cellular and Developmental Biology (MCDB). She is in Dr. Gerald Lipshutz’s Hepatic Regenerative Medicine Lab at UCLA. They primarily work with enzymatic deficiencies in the liver and brain, and subsequently develop gene therapies that are able to remedy these genetic diseases. Currently, she is working on an initial trial of Carbamoyl Phosphate Synthetase 1 (CPS1) deficiency. CPS1 deficiency is a rare genetic disorder that presents itself in humans through hyperammonemia, abnormal gait, vomiting and more. Previous research conducted on murine models of CPS1 deficiency is met with difficulty in expressing the large size of the CPS1 transgene compared to other hepatic enzymes. The Lipshutz Lab are utilizing a split adeno-associated virus gene therapy approach that can help mitigate the complications that arise from the large size of the CPS1 transgene. The initial trial has now reached a stage to expand to a larger, more permanent project that will allow us to observe the impact of the gene therapy on murine models with CPS1 deficiency. She would like to thank Dr. Lipshutz for the opportunity to grow and learn in research, as well as the rest of the Lipshutz Lab for all their guidance throughout this time. She would also like to thank the CARE Fellows program and Dr. Tama Hasson for providing me the opportunity to expand my skills in research.