Muna I. Naash
University of Houston, TX·Biomedical Engineering
Inherited retinal diseases slowly destroy photoreceptors, the light-sensing cells of the eye, and most have no treatment. This research asks how two disease genes keep photoreceptors healthy, and how working copies of those genes can be delivered safely to the retina without using viruses.
- Fields
Biomedical Engineering
Neuroscience
Genetics
- Topics
inherited retinal degeneration
Usher syndrome type 2A
usherin
- Grants
- 2 known grants, through Feb 2030
What they work on
- Map which usherin isoforms are made in the retina and the cochlea, using knock-in mice with fluorescently tagged usherin, and find out how the c.2299delG mutation causes hearing and vision defects in ush2aG/G mice.
- Test whether CK30PEG DNA nanoparticles, combined with hyaluronic acid nanospheres and sulfotyrosine, can deliver the large Ush2a gene to photoreceptors after intravitreal injection, comparing this route with subretinal injection in ush2aG/G mice.
- Measure whether the best-performing gene carrier and usherin isoform give lasting rescue of retinal structure and function in ush2aG/G mice followed over time.
- Determine how complexes of PRPH2 and ROM1 form the curved disc rims, close new discs and form incisures in photoreceptor outer segments, using mouse models and imaging.
- Identify proteins that bind PRPH2 and could explain why patients carrying the same PRPH2 variant develop different diseases, and develop therapeutic platforms for severe PRPH2-related disease.
More about the research
The two projects are linked: one builds a deeper understanding of how disease develops, and the other uses nanoparticle carriers to turn that understanding into gene therapy.
Why it matters
Patients with Usher syndrome type 2A and PRPH2-linked retinal dystrophies currently have no approved treatments. A non-viral way to deliver very large genes such as USH2A, together with a clearer picture of why PRPH2 patients differ in severity, could guide future gene therapies aimed at slowing or preventing the vision loss these patients experience.
Known grants
From NIH and NSF research grants. Labs often have other funding we can't see.
Closest labs
Visvanathan Ramamurthy
West Virginia University, WV·Neuroscience
Identify the best AAV gene therapy regimen for PROM1-associated blindness, delivering working gene copies with a harmless virus in animal models that mimic the disease and measuring retinal light responses by electrophysiology.
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.
Aparna Lakkaraju
University of California-San Francisco, CA·Biology
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.
The school
University of Houston: 21 labs on PI Pages, $239M a year on research, 374 research doctorates a year.
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