Defects in SHH are the cause of microphthalmia isolated with coloboma type 5 (MCOPCB5) [MIM:611638].
Microphthalmia is a clinically heterogeneous disorder of eye formation, ranging from small size of
a single eye to complete bilateral absence of ocular tissues. Ocular abnormalities like opacities
of the cornea and lens, scaring of the retina and choroid, cataract and other abnormalities like
cataract may also be present. Ocular colobomas are a set of malformations resulting from abnormal
morphogenesis of the optic cup and stalk, and the fusion of the fetal fissure (optic fissure).
Defects in SHH are the cause of holoprosencephaly type 3 (HPE3) [MIM:142945]. Holoprosencephaly
(HPE) [MIM:236100] is the most common structural anomaly of the brain, in which the developing
forebrain fails to correctly separate into right and left hemispheres. Holoprosencephaly is
genetically heterogeneous and associated with several distinct facies and phenotypic variability.
The majority of HPE3 cases are apparently sporadic, although clear examples of autosomal dominant
inheritance have been described. Interestingly, up to 30% of obligate carriers of HPE3 gene in
autosomal dominant pedigrees are clinically unaffected.
Defects in SHH are a cause of solitary median maxillary central incisor (SMMCI) [MIM:147250].
SMMCI is a rare dental anomaly characterized by the congenital absence of one maxillary central incisor.
Defects in SHH are the cause of triphalangeal thumb-polysyndactyly syndrome (TPTPS) [MIM:174500].
TPTPS is an autosomal dominant syndrome characterized by a wide spectrum of pre- and post-axial
abnormalities due to altered SHH expression pattern during limb development. TPTPS mutations have
been mapped to the 7q36 locus in the LMBR1 gene which contains in its intron 5 a long-range
cis-regulatory element of SHH expression.
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