Aparna Lakkaraju
University of California-San Francisco, CA·Biology
More than 300 different genes can cause inherited retinal degenerations, and most of these diseases have no treatment. Each gene would need its own gene therapy, so the lab asks a different question: can a single drug protect the retina regardless of which gene is broken?
- Fields
Biology
Pharmacology
Neuroscience
- Topics
inherited retinal degeneration
inherited retinal dystrophy
retinal pigment epithelium
- Grants
- 2 known grants, through Jun 2029
What they work on
- Test whether a clinically approved bisphosphonate drug, which blocks cholesterol production and ceramide generation, protects the retinal pigment epithelium and prevents retinal degeneration in mouse models of Stargardt and Batten disease.
- Evaluate whether a drug that activates adiponectin receptors increases ceramide breakdown and the formation of new mitochondria, preventing microglial activation and photoreceptor loss in these mouse models.
- Track drug distribution and mitochondrial dynamics in the living mouse retina using intravital imaging and super-resolution microscopy, combined with lipidomics and transcriptomics.
- Provide vision researchers with shared advanced microscopes and quantitative image analysis to capture how cells in eye tissues change over time at high resolution.
More about the research
The lab also helps run a shared imaging and image-analysis resource for vision scientists studying the eye at the level of individual cells.
Why it matters
Inherited retinal degenerations cause progressive, irreversible vision loss, often in people aged 15 to 45, and most have no treatment. Drugs that target a disease mechanism shared across many gene defects could treat multiple conditions, including Stargardt and Batten disease, with one approach. Because these drugs are already known to be safe and reach the retina without eye injections, they may offer a practical, gene-independent therapy.
Known grants
From NIH and NSF research grants. Labs often have other funding we can't see.
Closest labs
Hans Grossniklaus
Emory University, GA·Medicine & Clinical Research
Provide histology, electron microscopy, confocal and super-resolution microscopy, and animal surgery imaging to determine how tissue structure changes in eye diseases, disease models and after experimental treatments.
Tylor Lewis
University of Alabama at Birmingham, AL·Neuroscience·Early-career
Comprehensively characterize retinal degeneration in global ADAM9 knockout mice, focusing on the interface between photoreceptor outer segments and the retinal pigment epithelium.
Paul Bernstein
University of Utah, UT·Biochemistry
Test whether orally delivered synthetic VLC-PUFAs, retina-specific fats made by the enzyme ELOVL4, protect photoreceptor function, using mouse models, cell culture and model membranes.
The school
University of California-San Francisco: 89 labs on PI Pages, $2.1B a year on research, 164 research doctorates a year.
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