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Therapeutic targeting of TMEM97 to restore cholesterol homeostasis in demyelinating eye disease

Award Number: F30EY038545
ORGANIZATION: NATIONAL EYE INSTITUTE
OPDIV: NIH
AWARD CLASS: DISCRETIONARY
AWARD ACTIVITY TYPE: FELLOWSHIP/SCHOLARSHIP/STUDENT LOANS
PERIOD OF PERFORMANCE START DATE: 07/01/2026
PERIOD OF PERFORMANCE END DATE: 06/30/2030

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Therapeutic targeting of TMEM97 to restore cholesterol homeostasis in demyelinating eye disease - PROJECT SUMMARY Optic neuritis (ON) is one of the most common initial manifestations and causes of vision loss in demyelinating diseases like multiple sclerosis (MS). Optic nerve demyelination also occurs in isolated inflammatory episodes, trauma, and glaucoma. Widespread demyelination often exceeds the limited intrinsic remyelination potential of oligodendrocytes (OL) and leads to progressive axon damage and neuronal death. Current treatments rely on immunosuppressant and anti-inflammatory drugs that merely manage symptoms and delay progression. Thus, there is a pressing need for vision-preserving treatments that prevent neurodegeneration and/or promote repair. Cholesterol, a key constituent of the myelin sheath, is essential to central nervous system function and dysreg- ulated in many degenerative diseases, including demyelinating eye disease. Although cholesterol synthesis is amongst the top upregulated pathways in OLs during remyelination, it is insufficient to sustain this process ef- fectively. Given the optic nerve and retina’s reliance on cholesterol for rapid axonal transmission, structural in- tegrity, myelin repair, and cell survival, restoring proper cholesterol synthesis and intracellular trafficking could lead to new vision-preserving interventions, yet few studies have explored this possibility. One regulator of cholesterol trafficking is transmembrane protein 97 (TMEM97). TMEM97 has been implicated in impaired cholesterol egress from late endosomes and lysosomes via interactions with Niemann–Pick C1 in Niemann Pick disease type C. It has also been linked to neurotrauma, retinal ischemia, and in preliminary stud- ies, to MS. The central hypothesis is that TMEM97-mediated dysregulation of cholesterol homeostasis contrib- utes to demyelinating eye disease, and targeting TMEM97 will restore cholesterol homeostasis to favor OL sur- vival and remyelination. This proposal will address a critical gap in ON treatment by (1) describing how TMEM97 influences cholesterol homeostasis in OLs and (2) determining if targeting TMEM97 is a viable approach for promoting OL survival and remyelination in a mouse model of ON. These questions will be addressed with mech- anistic in vitro gain- and loss-of-function studies and in vivo by knocking out TMEM97 in OL lineage cells and assessing molecular, cellular, and functional outcomes of ON. This training project aligns with the NEI’s Strategic Plan and will provide valuable insights into the underlying mechanisms of failed remyelination to inform new therapeutic approaches to myelin repair and neuroaxonal protection in demyelinating eye disease. This fellowship will support the training necessary to lead impactful neuroimmunology and neuro-ophthalmol- ogy research as a physician-scientist. This proposal will provide specialized research training in molecular and cellular neuroscience under Drs. Daniel Liebl and Roberta Brambilla as well as longitudinally integrated clinical training at the world-renowned Bascom Palmer Eye Institute under neuro-ophthalmologist Dr. Maja Kostic.


Issue Date FY	Funding FY	Legal Entity Name	Legal Entity Address	Legal Entity City	Legal Entity State	Legal Entity Zip Code	Legal Entity COUNTY	Legal Entity COUNTRY	Assistance Listing	Award Code	Budget Year	Action Date	Action Type	Action Amount

Issue Date FY: 2026 ( Subtotal = $55,114 )
2026	2026	UNIVERSITY OF MIAMI	1320 S DIXIE HWY STE 1200	CORAL GABLES	FL	33146	MIAMI-DADE	USA	Vision Research	000	1	6/16/2026	NEW	$55,114
														Subtotal = $55,114

Grand Total All Awards = $55,114

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Therapeutic targeting of TMEM97 to restore cholesterol homeostasis in demyelinating eye disease

Award Number: F30EY038545

ORGANIZATION: NATIONAL EYE INSTITUTE

OPDIV: NIH

AWARD CLASS: DISCRETIONARY

AWARD ACTIVITY TYPE: FELLOWSHIP/SCHOLARSHIP/STUDENT LOANS

PERIOD OF PERFORMANCE START DATE: 07/01/2026

PERIOD OF PERFORMANCE END DATE: 06/30/2030

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