Mutation of a new gene causes a unique form of Hermansky–Pudlak syndrome in a genetic isolate of central Puerto Rico
doi: 10.1038/ng576
pmid: 11455388
Mutation of a new gene causes a unique form of Hermansky–Pudlak syndrome in a genetic isolate of central Puerto Rico
Hermansky-Pudlak syndrome (HPS) is a rare autosomal recessive disorder characterized by oculocutaneous albinism and a storage pool deficiency due to an absence of platelet dense bodies. Lysosomal ceroid lipofuscinosis, pulmonary fibrosis and granulomatous colitis are occasional manifestations of the disease. HPS occurs with a frequency of one in 1800 in north-west Puerto Rico due to a founder effect. Several non-Puerto Rican patients also have mutations in HPS1, which produces a protein of unknown function. Another gene, ADTB3A, causes HPS in the pearl mouse and in two brothers with HPS-2 (refs. 11,12). ADTB3A encodes a coat protein involved in vesicle formation, implicating HPS as a disorder of membrane trafficking. We sought to identify other HPS-causing genes. Using homozygosity mapping on pooled DNA of 6 families from central Puerto Rico, we localized a new HPS susceptibility gene to a 1.6-cM interval on chromosome 3q24. The gene, HPS3, has 17 exons, and a putative 113.7-kD product expected to reveal how new vesicles form in specialized cells. The homozygous, disease-causing mutation is a large deletion and represents the second example of a founder mutation causing HPS on the small island of Puerto Rico. We also present an allele-specific assay for diagnosing individuals heterozygous or homozygous for this mutation.
- University of Minnesota Morris United States
- National Institute of Arthritis and Musculoskeletal and Skin Diseases United States
- National Cancer Institute United States
- National Institutes of Health United States
- Roswell Park Cancer Institute United States
Male, Genotype, Genetic Carrier Screening, DNA Mutational Analysis, Homozygote, Molecular Sequence Data, Intracellular Signaling Peptides and Proteins, Blotting, Northern, Founder Effect, Pedigree, Hermanski-Pudlak Syndrome, Organ Specificity, Mutation, Humans, Female, Genetic Predisposition to Disease, Amino Acid Sequence, Chromosomes, Human, Pair 3, Carrier Proteins, Alleles
Male, Genotype, Genetic Carrier Screening, DNA Mutational Analysis, Homozygote, Molecular Sequence Data, Intracellular Signaling Peptides and Proteins, Blotting, Northern, Founder Effect, Pedigree, Hermanski-Pudlak Syndrome, Organ Specificity, Mutation, Humans, Female, Genetic Predisposition to Disease, Amino Acid Sequence, Chromosomes, Human, Pair 3, Carrier Proteins, Alleles
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