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Usher syndrome type 1(USH1)

MedGen UID:
292820
Concept ID:
C1568247
Disease or Syndrome
Synonyms: Retinitis pigmentosa and congenital deafness; USH1; Usher syndrome, type I, French variety
SNOMED CT: Usher syndrome type 1 (232057003)
Modes of inheritance:
Autosomal recessive inheritance
MedGen UID:
141025
Concept ID:
C0441748
Intellectual Product
Source: Orphanet
A mode of inheritance that is observed for traits related to a gene encoded on one of the autosomes (i.e., the human chromosomes 1-22) in which a trait manifests in individuals with two pathogenic alleles, either homozygotes (two copies of the same mutant allele) or compound heterozygotes (whereby each copy of a gene has a distinct mutant allele).
 
Genes (locations): MYO7A (11q13.5); USH1C (11p15.1)
Related genes: USH1G, PCDH15, CDH23
 
Monarch Initiative: MONDO:0010168
OMIM®: 276900
Orphanet: ORPHA231169

Disease characteristics

Excerpted from the GeneReview: Usher Syndrome Type I
Usher syndrome type I (USH1) is characterized by congenital, bilateral, profound sensorineural hearing loss, vestibular areflexia, and adolescent-onset retinitis pigmentosa (RP). Unless fitted with a cochlear implant, individuals do not typically develop speech. RP, a progressive, bilateral, symmetric degeneration of rod and cone functions of the retina, develops in adolescence, resulting in progressively constricted visual fields and impaired visual acuity. [from GeneReviews]
Authors:
Robert K Koenekoop  |  Moises A Arriaga  |  Karmen M Trzupek, et. al.   view full author information

Additional descriptions

From OMIM
Usher syndrome type I is an autosomal recessive condition characterized by profound congenital hearing impairment with unintelligible speech, early retinitis pigmentosa (usually evident within the first decade), and constant vestibular dysfunction. Type I is distinguished from type II (276901) on the basis of severity of hearing loss and the extent of vestibular involvement. Type I patients are profoundly deaf, whereas type II patients are 'hard of hearing.' Vestibular function is defective in type I patients, whereas type II patients have normal vestibular function (Moller et al., 1989). Patients with type III (USH3; 276902) have progressive hearing loss. Patients with type IV (USH4; 618144) have late onset of both retinitis pigmentosa and progressive, moderate to severe sensorineural hearing loss without vestibular involvement (Khateb et al., 2018). Genetic Heterogeneity of Usher Syndrome Type I USH type I is genetically heterogeneous. USH1C (276904), the 'Acadian variety,' is caused by mutation in harmonin (605242), on 11p15. USH1D (601067) is caused by mutation in the cadherin-23 (CDH23; 605516) on 10q21. USH1F (602083) is caused by mutation in the protocadherin-15 (PCDH15; 605514) on 10q22. USH1G (606943) is caused by mutation in the SANS gene (607696), on 17q25. USH1E (602097) maps to 21q21, and USH1H (612632) maps to 15q22-q23. USH1J (614869) is caused by mutation in the CIB2 gene (605564) on 15q24. USH1K (614990) maps to chromosome 10p11.21-q21.1. A form of USH type I in which affected members carried heterozygous mutations in both CDH23 and PCDH15 has been reported (USH1D/F; see 601067), thus supporting a digenic model for some individuals with this phenotype. Gerber et al. (2006) presented evidence that the form of USH1 previously called USH1A, or the 'French variety,' and mapped to chromosome 14 does not in fact exist; mutations in the MYO7A gene were found in most of these families, and in others the phenotype was found to map to other loci. Ahmed et al. (2003) reviewed the molecular genetics of Usher syndrome and indicated that at least 12 loci had been identified as underlying the 3 different clinical subtypes.  http://www.omim.org/entry/276900
From MedlinePlus Genetics
Usher syndrome is a condition characterized by partial or total hearing loss and vision loss that worsens over time. The hearing loss is classified as sensorineural, which means that it is caused by abnormalities of the inner ear. The loss of vision is caused by an eye disease called retinitis pigmentosa (RP), which affects the layer of light-sensitive tissue at the back of the eye (the retina). Vision loss occurs as the light-sensing cells of the retina gradually break down. Loss of night vision begins first, followed by blind spots that develop in the side (peripheral) vision. Over time, these blind spots enlarge and merge to produce tunnel vision. In some cases, vision is further impaired by clouding of the lens of the eye (cataracts). However, many people with retinitis pigmentosa retain some central vision throughout their lives.

Researchers have identified three major types of Usher syndrome, designated as types I, II, and III. These types are distinguished by the severity of hearing loss, the presence or absence of balance problems, and the age at which signs and symptoms appear. The types are further divided into subtypes based on their genetic cause.

Most individuals with Usher syndrome type I are born with severe to profound hearing loss. Worsening vision loss caused by retinitis pigmentosa becomes apparent in childhood. This type of Usher syndrome also causes abnormalities of the vestibular system, which is the part of the inner ear that helps maintain the body's balance and orientation in space. As a result of the vestibular abnormalities, children with the condition have trouble with balance. They begin sitting independently and walking later than usual, and they may have difficulty riding a bicycle and playing certain sports.

Usher syndrome type II is characterized by hearing loss from birth and progressive vision loss that begins in adolescence or adulthood. The hearing loss associated with this form of Usher syndrome ranges from mild to severe and mainly affects the ability to hear high-frequency sounds. For example, it is difficult for affected individuals to hear high, soft speech sounds, such as those of the letters d and t. The degree of hearing loss varies within and among families with this condition, and it may become more severe over time. Unlike the other forms of Usher syndrome, type II is not associated with vestibular abnormalities that cause difficulties with balance.

People with Usher syndrome type III experience hearing loss and vision loss beginning somewhat later in life. Unlike the other forms of Usher syndrome, type III is usually associated with normal hearing at birth. Hearing loss typically begins during late childhood or adolescence, after the development of speech, and becomes more severe over time. By middle age, most affected individuals have profound hearing loss. Vision loss caused by retinitis pigmentosa also develops in late childhood or adolescence. Some people with Usher syndrome type III develop vestibular abnormalities that cause problems with balance.  https://medlineplus.gov/genetics/condition/usher-syndrome

Clinical features

From HPO
Sensorineural hearing loss disorder
MedGen UID:
9164
Concept ID:
C0018784
Disease or Syndrome
A type of hearing impairment in one or both ears related to an abnormal functionality of the cochlear nerve.
Absent vestibular function
MedGen UID:
870219
Concept ID:
C4024656
Finding
Complete lack of functioning of the vestibular apparatus.
Motor delay
MedGen UID:
381392
Concept ID:
C1854301
Finding
A type of Developmental delay characterized by a delay in acquiring motor skills.
Nystagmus
MedGen UID:
45166
Concept ID:
C0028738
Disease or Syndrome
Rhythmic, involuntary oscillations of one or both eyes related to abnormality in fixation, conjugate gaze, or vestibular mechanisms.
Abnormal electroretinogram
MedGen UID:
96908
Concept ID:
C0476397
Finding
Any abnormality of the electrical responses of various cell types in the retina as measured by electroretinography.
Undetectable electroretinogram
MedGen UID:
383742
Concept ID:
C1855685
Finding
Lack of any response to stimulation upon electroretinography.
Visual loss
MedGen UID:
784038
Concept ID:
C3665386
Finding
Loss of visual acuity (implying that vision was better at a certain time point in life). Otherwise the term reduced visual acuity should be used (or a subclass of that).
Rod-cone dystrophy
MedGen UID:
1632921
Concept ID:
C4551714
Disease or Syndrome
An inherited retinal disease subtype in which the rod photoreceptors appear to be more severely affected than the cone photoreceptors. Typical presentation is with nyctalopia (due to rod dysfunction) followed by loss of mid-peripheral field of vision, which gradually extends and leaves many patients with a small central island of vision due to the preservation of macular cones.

Term Hierarchy

CClinical test,  RResearch test,  OOMIM,  GGeneReviews,  VClinVar  

Professional guidelines

PubMed

Friedman TB, Schultz JM, Ahmed ZM
Retina 2005 Dec;25(8 Suppl):S40-S42. doi: 10.1097/00006982-200512001-00016. PMID: 16374329

Curated

Bolz HJ, Roux AF
Eur J Hum Genet 2011 Aug;19(8) Epub 2011 Mar 9 doi: 10.1038/ejhg.2011.15. PMID: 21697857Free PMC Article

Recent clinical studies

Etiology

Ehn M, Wahlqvist M, Danermark B, Dahlström Ö, Möller C
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Rong W, Chen X, Zhao K, Liu Y, Liu X, Ha S, Liu W, Kang X, Sheng X, Zhao C
PLoS One 2014;9(5):e97808. Epub 2014 May 15 doi: 10.1371/journal.pone.0097808. PMID: 24831256Free PMC Article
Henricson C, Wass M, Lidestam B, Möller C, Lyxell B
Int J Pediatr Otorhinolaryngol 2012 Oct;76(10):1449-57. Epub 2012 Jul 12 doi: 10.1016/j.ijporl.2012.06.020. PMID: 22795738
Flores-Guevara R, Renault F, Loundon N, Marlin S, Pelosse B, Momtchilova M, Auzoux-Chevé M, Vermersch AI, Richard P
Eur J Paediatr Neurol 2009 Nov;13(6):505-7. Epub 2008 Nov 9 doi: 10.1016/j.ejpn.2008.10.002. PMID: 19006676
Sadeghi AM, Eriksson K, Kimberling WJ, Sjöström A, Möller C
Acta Ophthalmol Scand 2006 Aug;84(4):537-44. doi: 10.1111/j.1600-0420.2006.00675.x. PMID: 16879578

Diagnosis

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Prognosis

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Khan AO, Becirovic E, Betz C, Neuhaus C, Altmüller J, Maria Riedmayr L, Motameny S, Nürnberg G, Nürnberg P, Bolz HJ
Sci Rep 2017 May 3;7(1):1411. doi: 10.1038/s41598-017-01577-8. PMID: 28469144Free PMC Article
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Clinical prediction guides

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Lenassi E, Saihan Z, Cipriani V, Le Quesne Stabej P, Moore AT, Luxon LM, Bitner-Glindzicz M, Webster AR
Ophthalmology 2014 Feb;121(2):580-7. Epub 2013 Nov 5 doi: 10.1016/j.ophtha.2013.09.017. PMID: 24199935
Larget-Piet D, Gerber S, Bonneau D, Rozet JM, Marc S, Ghazi I, Dufier JL, David A, Bitoun P, Weissenbach J
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