This paper presents preclinical data on a bispecific antibody, SAR446159, designed to target alpha-synuclein aggregates and cross the blood-brain barrier for treating synucleinopathies.

npj Parkinson s Disease · Aug 22, 2025Peer-reviewedNeuroscience

Elovl1 inhibition reduced very long chain fatty acids in a mouse model of adrenoleukodystrophy

Jeremy Y. Huang, Brian Freed, Martin Hanus, Kelly Keefe, Ming Sum Ruby Chiang, Alexander Brezzani, Yongyi Luo, Yihang Li, Becky Lam, Stephanie Holley, Joseph Gans, Zuzana Dostalova, Buyun Tang, Clifford G. Phaneuf, Erin A. Teeple, , Can Kayatekin

The paper inhibits Elovl1 in a mouse model of ALD to reduce VLCFA accumulation, finding it corrects lipid pathways but also causes unexpected transcriptional changes.

iScience · Jul 31, 2025Peer-reviewedPharmacology & Toxicology

AAV gene therapy for GBA-PD and Gaucher Disease

Swathi Ayloo, Jae Cheon Ryu, Shih-Ching Chou, Lydia Blatnik, Erik Wischhof, Jie Bu, Lilu Guo, Mahmud Hossain, Dhiman Ghosh, William McCarty, Ann Byrne, Lilly Chai, J T Clarke, Can Kayatekin, Bailin Zhang, , Shyam S. Ramachandran

This paper describes an AAV-mediated gene therapy strategy for GBA1-PD and Gaucher Disease, demonstrating robust GCase replenishment and cross-correction in preclinical models.

bioRxiv · Jun 17, 2025 · PreprintNeuroscience

Network Analysis and Human Single Cell Brain Transcriptomics Reveal Novel Aspects of Alpha-Synuclein (SNCA) Biology

Erin A. Teeple, Khushboo Jindal, Beril Kiragasi, Siddharth Annaldasula, Ann Byrne, Lilly Chai, Mahdiar Sadeghi, Can Kayatekin, Srinivas Shankara, Katherine Wood Klinger, S. Pablo Sardi, Stephen L. Madden, Dinesh Kumar

The paper identifies a conserved SNCA co-expression module in human brain single-nucleus RNA-seq data and links it to Parkinson's Disease risk loci through protein-protein interaction networks and CRISPR validation.

bioRxiv · Jun 5, 2020 · PreprintNeuroscience