A Case of Aymé-Gripp Syndromic Congenital Cataracts and Pigmentary Retinopathy Caused by a Novel MAF Variant in the N-Terminal Transactivation Domain—A Case Report and Literature Review
MAF encodes a transcription factor involved in T-helper-2 (Th2) cell differentiation. Heterozygous pathogenic variants in MAF have been observed in both isolated and syndromic congenital cataract cases; genotype–phenotype correlations are based on the location of the variant within the gene. Variants in the N-terminus domain of MAF are associated with cataracts as part of Aymé-Gripp syndrome. The purpose of this report is to expand the ocular phenotypic spectrum of Aymé-Gripp syndrome by describing a patient with MAF variant c.185C>G, p.Thr62Arg, and the traditional systemic findings and congenital cataracts as well as an unusual feature of pigmentary retinopathy, which has not been previously reported in Aymé-Gripp syndrome. Additionally, a comprehensive review of the literature was completed to report ocular genotype–phenotype data on previously reported patients with MAF-associated Aymé-Gripp syndrome.
Authors
- Pamela Rathbun (ORCID: https://orcid.org/0000-0001-5830-5075)
- Jennifer L. Rossen (ORCID: https://orcid.org/0000-0002-9742-8868)
- Alexander Ing (ORCID: https://orcid.org/0000-0001-5233-2937)
- Brenda L. Bohnsack (ORCID: https://orcid.org/0000-0001-8961-6343)
- Andrew D. Skol (ORCID: https://orcid.org/0009-0005-7530-8127)
- Patrick D. McMullen (ORCID: https://orcid.org/0000-0002-9635-932X)
- Andy Drackley
- Allison Goetsch Weisman
- Kailee Yap
- Sudhi P. Kurup
- Kaersti L. Rickels
- Max Chauhan
- Valerie Allegreti
- Hantamala Ralay Ranaivo
Institutions
- Northwestern University (US)
- Lurie Children's Hospital (US)
- Lurie Children's Hospital (US)
Publication Details
- Journal
- Genes
- Published
- 2026-03-27
- DOI
- https://doi.org/10.3390/genes17040380
- Primary Topic
- Developmental Biology and Gene Regulation
- Type
- article
- Field-Weighted Citation Impact
- 0.00