A representative culture from a brain organoid in which the gene MECP2 — causative in Rett syndrome — has been “knocked out,” as shown through a fluorescent microscope. Because the culture was treated with experimental cancer drug ADH-503, new synapses formed. Credit: Muotri Lab / UC San Diego Health Sciences
New study identifies a potential therapeutic target to improve cognitive function in patients with various neurological disorders.
New research from the University of California San Diego suggests that an experimental cancer drug may improve cognitive function in individuals with Rett syndrome, a rare condition associated with autism. This discovery holds potential for developing treatments for other neurological disorders.
The findings, published July 25 in Stem DOI: 10.1016/j.stemcr.2024.06.013
Co-authors of the study include Christopher LaRock, with the Department of Pediatrics at the University California San Diego School of Medicine and Skaggs School of Pharmacy and Pharmaceutical Sciences; Jacob J. Jeziroski, Natalia Chermount, Tomoka Ozaki, Aurian Saleh, Cedric E. Snethlage, Sandra Sanchez, Gabriela Goldberg, Clever A. Trujillo and Kinichi Nakashima, with the University of California San Diego School of Medicine and Department of Pediatrics at Rady Children’s Hospital San Diego, and the Department of Cellular & Molecular Medicine; Hideyuki Nakashima, with the Department of Stem Cell Biology and Medicine at Kyushu University’s Graduate School of Medicine; Adriano Ferrasa, with the Experimental Multiuser Labratory at the Graduate Program in Health Sciences at the School of Medicine at Pontifícia Universidade Católica do Paraná in Curitiba, Paraná, Brazil, as well as the Department of Informatics at the Universidade Estadual de Ponta Grossa in Ponta Grossa, Parana, Brazil; Roberto H. Herai, with the Experimental Multiuser Labratory at the Graduate Program in Health Sciences at the School of Medicine at Pontifícia Universidade Católica do Paraná in Curitiba, Paraná, Brazil, and the Research Department at Lico Kaesemodel Institute in Curitiba, Paraná, Brazil; and Victor Nizet, with the Department of Pediatrics at the University California San Diego School of Medicine and Skaggs School of Pharmacy and Pharmaceutical Sciences.
This work was made possible in part by the California Institute for Regenerative Medicine (CIRM) Major Facilities grant (FA1-00607) to the Sanford Consortium for Regenerative Medicine. Muotri is supported by the
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