Diseases associated with aging and development are already a leading cause of death and disability, and their prevalence is rising fast.
By 2050, the number of Alzheimer’s patients age 65 and older may nearly triple from 5 million to 13.8 million. Our researchers are discovering the etiological pathways as well as small-molecule and stem cell-based treatments to address the clinical unmet need of patients.

Translating basic science discoveries into new treatments
Our mission is to translate discoveries from basic molecular research and apply a systems approach to generate breakthrough therapies for devastating neurologic and cardiac diseases.
Programs
Degenerative Diseases Program
Every degenerative disease is associated with misfolded proteins. Studying these proteins provides new clues to protecting our health.
Human Genetics Program
We are discovering the genetic roots of human disorders and translating that knowledge for patient well-being and potential treatments.
Development, Aging and Regeneration Program
By building, analyzing and probing models of disease, we can pursue innovative treatment strategies for challenging health conditions.
Publications
Nuclear Pore Complexes Are Key Regulators of Oligodendrocyte Differentiation and Function.
Raices M, D'Angelo MA
Neuron 2019 May 8 ;102(3):509-511
Disulfiram (Antabuse) Activates ROS-Dependent ER Stress and Apoptosis in Oral Cavity Squamous Cell Carcinoma.
Shah O'Brien P, Xi Y, Miller JR, Brownell AL, Zeng Q, Yoo GH, Garshott DM, O'Brien MB, Galinato AE, Cai P, Narula N, Callaghan MU, Kaufman RJ, Fribley AM
J Clin Med 2019 May 6 ;8(5)
Deficient Endoplasmic Reticulum-Mitochondrial Phosphatidylserine Transfer Causes Liver Disease.
Hernández-Alvarez MI, Sebastián D, Vives S, Ivanova S, Bartoccioni P, Kakimoto P, Plana N, Veiga SR, Hernández V, Vasconcelos N, Peddinti G, Adrover A, Jové M, Pamplona R, Gordaliza-Alaguero I, Calvo E, Cabré N, Castro R, Kuzmanic A, Boutant M, Sala D, Hyotylainen T, Orešič M, Fort J, Errasti-Murugarren E, Rodrígues CMP, Orozco M, Joven J, Cantó C, Palacin M, Fernández-Veledo S, Vendrell J, Zorzano A
Cell 2019 May 2 ;177(4):881-895.e17
β2-microglobulin functions as an endogenous NMDAR antagonist to impair synaptic function.
Gao Y, Hong Y, Huang L, Zheng S, Zhang H, Wang S, Yao Y, Zhao Y, Zhu L, Xu Q, Chai X, Zeng Y, Zeng Y, Zheng L, Zhou Y, Luo H, Zhang X, Zhang H, Zhou Y, Fu G, Sun H, Huang TY, Zheng Q, Xu H, Wang X
Cell 2023 Mar 2 ;186(5):1026-1038.e20
Sorting nexin 10 sustains PDGF receptor signaling in glioblastoma stem cells via endosomal protein sorting.
Gimple RC, Zhang G, Wang S, Huang T, Lee J, Taori S, Lv D, Dixit D, Halbert ME, Morton AR, Kidwell RL, Dong Z, Prager BC, Kim LJ, Qiu Z, Zhao L, Xie Q, Wu Q, Agnihotri S, Rich JN
JCI Insight 2023 Mar 22 ;8(6)
Extraction and Purification of Single Nuclei from Frozen Human Brain Tissue.
Palmer CR, Chun J
Methods Mol Biol 2023 ;2561:31-42
Development and use of a high-throughput screen to identify novel modulators of the corticotropin releasing factor binding protein.
Haass-Koffler CL, Francis TC, Gandhi P, Patel R, Naemuddin M, Nielsen CK, Bartlett SE, Bonci A, Vasile S, Hood BL, Suyama E, Hedrick MP, Smith LH, Limpert AS, Roberto M, Cosford NDP, Sheffler DJ
SLAS Discov 2022 Dec ;27(8):448-459
A tetravalent TREM2 agonistic antibody reduced amyloid pathology in a mouse model of Alzheimer's disease.
Zhao P, Xu Y, Jiang L, Fan X, Li L, Li X, Arase H, Zhao Y, Cao W, Zheng H, Xu H, Tong Q, Zhang N, An Z
Sci Transl Med 2022 Sep 7 ;14(661):eabq0095
Novel Antagonist of the Type 2 Lysophosphatidic Acid Receptor (LPA(2)), UCM-14216, Ameliorates Spinal Cord Injury in Mice.
Khiar-Fernández N, Zian D, Vázquez-Villa H, Martínez RF, Escobar-Peña A, Foronda-Sainz R, Ray M, Puigdomenech-Poch M, Cincilla G, Sánchez-Martínez M, Kihara Y, Chun J, López-Vales R, López-Rodríguez ML, Ortega-Gutiérrez S
J Med Chem 2022 Aug 25 ;65(16):10956-10974