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Authors

Advisor(s)

Bradley Yoder

Committee Member(s)

Kent Keyser

Michael Miller

Michael Crowley

Alecia Gross

Document Type

Dissertation

Date of Award

2015

Abstract

Nephronophthisis (NPHP) is a ciliopathy with diverse clinical features likely caused by genetic modifiers. To identify NPHP modifiers, a screen was conducted on nphp-4(tm925) mutant C. elegans to reveal mutations that exacerbate the NPHP ciliary defects. Ten loci were generated, five of which have now been identified. Three are mutations in ciliopathy genes mks-1, mks-2, and mks-5. The fourth allele (yhw66) is a missense mutation (S316F) in OSM-3, a kinesin required for cilia distal segment assembly. As in osm-3 null mutants, nphp-4(tm925);osm-3(yhw66) mutants lack distal segments, are dye-filing defective (Dyf), and have osmotic avoidance defects (OSM). The osm-3(yhw66) mutant alone has no overt cilia phenotype but do have reduced IFT rates. To evaluate the possibility S316F is a phosphorylation site, we compared the localization and function of phospho-null OSM-3(S316F), phospho-mimetic OSM-3(S316D), and wild type OSM-3. OSM-3(S316F) is enriched at the cilia base compared to wild type OSM-3. In contrast, OSM-3(S316D) accumulates in the cilia distal tip. While OSM-3(S316F) rescues all phenotypes in osm-3(mn357) nulls and OSM-3(S316D) restores distal cilia segments, OSM-3(S316D) does not rescue the Dyf phenotype with or without NPHP-4. Thus, there are NPHP-4 dependent and independent phenotypes associated with these OSM-3 mutations. These data reveal that NPHP-4 can exert regulatory influences on trafficking and function of a ciliary kinesin. The fifth allele from the screen was cca-1, a T-type calcium channel. The genetic interaction between cca-1 and nphp-4 is not critical for building the cilia but is necessary to maintain the cilium and affects its localization in an NPHP-4 dependent manner. Without this interaction calcium signaling and adaptation responses in the AWC neuron is impaired. Moreover, the data suggest that the human OSM-3 (Kif17) and CCA-1 (CACNA1I) homologs are candidate modifying locus affecting disease penetrance or expressivity in NPHP patients.

ProQuest Publication Number

Document on ProQuest

ISBN

978-1-339-31679-6

Comments

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