Novel homozygous and compound heterozygous COL17A1 mutations associated with junctional epidermolysis bullosa. 1998

M Floeth, and J Fiedorowicz, and H Schäcke, and N Hammami-Hausli, and K Owaribe, and R M Trüeb, and L Bruckner-Tuderman
Department of Dermatology, University of Münster, Germany.

Junctional epidermolysis bullosa is a heritable, heterogeneous blistering skin disease with mechanically induced dermal-epidermal separation, mild skin atrophy, nail dystrophy, and alopecia. Four unrelated junctional epidermolysis bullosa families with different phenotypes were investigated here and four novel mutations associated with the disease were identified. Patients 1, 2, and 3 had generalized atrophic benign epidermolysis bullosa, with nonscarring blistering and varying degree of alopecia. Patient 4 had the localisata variant of junctional epidermolysis bullosa, with predominantly acral blistering and normal hair. All patients had mutations in the COL17A1 gene encoding collagen XVII, a hemidesmosomal transmembrane protein. Patients 1 and 2 carried homozygous deletions 520delAG and 2965delG, respectively. Patient 3 was compound heterozygous for a missense and a deletion mutation (G539E and 2666delTT), and patient 4 was heterozygous for a known mutation R1226X. The deletions led to premature termination codons and to drastically reduced collagen XVII mRNA and protein levels, consistent with the absence of the collagen in generalized atrophic benign epidermolysis bullosa skin. The missense mutation G539E allowed synthesis of immunoreactive collagen XVII in keratinocytes, but prevented its secretion, thus causing lack of the protein in the skin. The data suggest that different COL17A1 mutations and their combinations can result in a spectrum of biologic and clinical phenotypes of not only generalized atrophic benign epidermolysis bullosa, but also localized junctional epidermolysis bullosa.

UI MeSH Term Description Entries
D008297 Male Males
D002872 Chromosome Deletion Actual loss of portion of a chromosome. Monosomy, Partial,Partial Monosomy,Deletion, Chromosome,Deletions, Chromosome,Monosomies, Partial,Partial Monosomies
D003062 Codon A set of three nucleotides in a protein coding sequence that specifies individual amino acids or a termination signal (CODON, TERMINATOR). Most codons are universal, but some organisms do not produce the transfer RNAs (RNA, TRANSFER) complementary to all codons. These codons are referred to as unassigned codons (CODONS, NONSENSE). Codon, Sense,Sense Codon,Codons,Codons, Sense,Sense Codons
D003094 Collagen A polypeptide substance comprising about one third of the total protein in mammalian organisms. It is the main constituent of SKIN; CONNECTIVE TISSUE; and the organic substance of bones (BONE AND BONES) and teeth (TOOTH). Avicon,Avitene,Collagen Felt,Collagen Fleece,Collagenfleece,Collastat,Dermodress,Microfibril Collagen Hemostat,Pangen,Zyderm,alpha-Collagen,Collagen Hemostat, Microfibril,alpha Collagen
D006579 Heterozygote An individual having different alleles at one or more loci regarding a specific character. Carriers, Genetic,Genetic Carriers,Carrier, Genetic,Genetic Carrier,Heterozygotes
D006720 Homozygote An individual in which both alleles at a given locus are identical. Homozygotes
D006801 Humans Members of the species Homo sapiens. Homo sapiens,Man (Taxonomy),Human,Man, Modern,Modern Man
D000368 Aged A person 65 years of age or older. For a person older than 79 years, AGED, 80 AND OVER is available. Elderly
D016109 Epidermolysis Bullosa, Junctional Form of epidermolysis bullosa having onset at birth or during the neonatal period and transmitted through autosomal recessive inheritance. It is characterized by generalized blister formation, extensive denudation, and separation and cleavage of the basal cell plasma membranes from the basement membrane. Epidermolysis Bullosa Letalis,Herlitz Disease,Junctional Epidermolysis Bullosa,Lethal Junctional Epidermolysis Bullosa,Epidermolysis Bullosa Junctionalis, Disentis Type,Epidermolysis Bullosa Junctionalis, Herlitz Type,Epidermolysis Bullosa Junctionalis, Progressive,Epidermolysis Bullosa Junctionalis, Severe Nonlethal,Epidermolysis Bullosa Progressiva,Epidermolysis Bullosa, Generalized Atrophic Benign,Epidermolysis Bullosa, Junctional, Herlitz Type,Epidermolysis Bullosa, Junctional, Herlitz-Pearson Type,Herlitz's Disease,Herlitz-Pearson Type Epidermolysis Bullosa,Herlitz-Pearson-Type Epidermolysis Bullosa,Bullosa Letali, Epidermolysis,Bullosa Letalis, Epidermolysis,Bullosa, Herlitz-Pearson-Type Epidermolysis,Disease, Herlitz,Disease, Herlitz's,Epidermolysis Bullosa Letali,Epidermolysis Bullosa, Herlitz-Pearson-Type,Herlitz Pearson Type Epidermolysis Bullosa,Herlitzs Disease,Letali, Epidermolysis Bullosa,Letalis, Epidermolysis Bullosa
D017354 Point Mutation A mutation caused by the substitution of one nucleotide for another. This results in the DNA molecule having a change in a single base pair. Mutation, Point,Mutations, Point,Point Mutations

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