Entry - #620438 - CILIARY DYSKINESIA, PRIMARY, 51; CILD51 - OMIM - (OMIM.ORG)

# 620438

CILIARY DYSKINESIA, PRIMARY, 51; CILD51


Phenotype-Gene Relationships

Location Phenotype Phenotype
MIM number
Inheritance Phenotype
mapping key
Gene/Locus Gene/Locus
MIM number
21q22.2 Ciliary dyskinesia, primary, 51 620438 AR 3 BRWD1 617824
Clinical Synopsis
 
Phenotypic Series
 

INHERITANCE
- Autosomal recessive
CARDIOVASCULAR
Heart
- Dextrocardia
RESPIRATORY
Nasopharynx
- Nasal obstruction
- Chronic rhinosinusitis
Airways
- Bronchiectasis
- Chronic bronchitis
- Defects of inner and outer dynein arms of respiratory cilia
- Frequent absence of outer doublet microtubules of respiratory cilia
Lung
- Chronic cough
- Recurrent pneumonia
ABDOMEN
- Situs inversus (in some patients)
GENITOURINARY
Internal Genitalia (Male)
- Infertility
- Reduced sperm count (in some patients)
- Reduced progressive motility of sperm
- Multiple morphologic abnormalities of the sperm flagella
- Absent sperm flagella
- Short sperm flagella
- Irregular-caliber sperm flagella
- Absence of inner and outer dynein arms of sperm flagella
LABORATORY ABNORMALITIES
- Decreased nasal nitric oxide (nNO, in some patients)
MISCELLANEOUS
- Based on report of 3 unrelated Chinese men (last curated June 2023)
MOLECULAR BASIS
- Caused by mutation in the bromodomain and WD repeat domain-containing protein-1 gene (BRWD1, 617824.0001)
Primary ciliary dyskinesia - PS244400 - 54 Entries
Location Phenotype Inheritance Phenotype
mapping key
Phenotype
MIM number
Gene/Locus Gene/Locus
MIM number
1p36.33 Ciliary dyskinesia, primary, 49, without situs inversus AR 3 620197 CFAP74 620187
1p36.32 Ciliary dyskinesia, primary, 47, and lissencephaly AR 3 619466 TP73 601990
2p23.3 Ciliary dyskinesia, primary, 21 AR 3 615294 DRC1 615288
2q14.2 Ciliary dyskinesia, primary, 55 AR 3 279000 CFAP221 618704
2q32.3 Ciliary dyskinesia, primary, 50 AR 3 620356 DNAH7 610061
2q35 ?Ciliary dyskinesia, primary, 46 AR 3 619436 STK36 607652
2q36.3 Ciliary dyskinesia, primary, 52 AR 3 620570 DAW1 620279
3p24.1 Ciliary dyskinesia, primary, 44 AR 3 618781 NEK10 618726
3p21.31 Ciliary dyskinesia, primary, 22 AR 3 615444 ZMYND10 607070
3p21.1 Ciliary dyskinesia, primary, 37 AR 3 617577 DNAH1 603332
3q26.33 Ciliary dyskinesia, primary, 14 AR 3 613807 CCDC39 613798
5p15.2 Ciliary dyskinesia, primary, 3, with or without situs inversus AR 3 608644 DNAH5 603335
5q11.2 Ciliary dyskinesia, primary, 42 AR 3 618695 MCIDAS 614086
5q11.2 Ciliary dyskinesia, primary, 29 AR 3 615872 CCNO 607752
5q31.2 Ciliary dyskinesia, primary, 48, without situs inversus AR 3 620032 NME5 603575
6p21.1 Ciliary dyskinesia, primary, 12 AR 3 612650 RSPH9 612648
6q22.1 Ciliary dyskinesia, primary, 11 AR 3 612649 RSPH4A 612647
6q25.3 Ciliary dyskinesia, primary, 32 AR 3 616481 RSPH3 615876
7p22.3 Ciliary dyskinesia, primary, 18 AR 3 614874 DNAAF5 614864
7p15.3 Ciliary dyskinesia, primary, 7, with or without situs inversus AR 3 611884 DNAH11 603339
7p14.1 ?Ciliary dyskinesia, primary, 6 AR 3 610852 NME8 607421
8q11.21 Ciliary dyskinesia, primary, 53 AR 3 620642 CLXN 619564
8q22.2 Ciliary dyskinesia, primary, 28 AR 3 615505 SPAG1 603395
8q24.22 Ciliary dyskinesia, primary, 19 AR 3 614935 DNAAF11 614930
9p13.3 Ciliary dyskinesia, primary, 1, with or without situs inversus AR 3 244400 DNAI1 604366
10p12.1 Ciliary dyskinesia, primary, 23 AR 3 615451 ODAD2 615408
11p15.5 Ciliary dyskinesia, primary, 39 AR 3 618254 LRRC56 618227
11q13.4 Ciliary dyskinesia, primary, 34 AR 3 617091 DNAJB13 610263
11q22.1 Ciliary dyskinesia, primary, 38 AR 3 618063 CFAP300 618058
11q23.2 Ciliary dyskinesia, primary, 45 AR 3 618801 TTC12 610732
12q13.12 Ciliary dyskinesia, primary, 27 AR 3 615504 DRC2 611088
12q23.1 Ciliary dyskinesia, primary, 54 AR 3 621125 CFAP54 621121
14q21.3 Ciliary dyskinesia, primary, 10 AR 3 612518 DNAAF2 612517
14q24.3 Ciliary dyskinesia, primary, 16 AR 3 614017 DNAL1 610062
15q13.1-q15.1 Ciliary dyskinesia, primary, 4 AR 2 608646 CILD4 608646
15q21.3 Ciliary dyskinesia, primary, 25 AR 3 615482 DNAAF4 608706
15q24-q25 Ciliary dyskinesia, primary, 8 AR 2 612274 CILD8 612274
16q22.2 Ciliary dyskinesia, primary, 5 AR 3 608647 HYDIN 610812
16q24.1 Ciliary dyskinesia, primary, 13 AR 3 613193 DNAAF1 613190
16q24.3 Ciliary dyskinesia, primary, 33 AR 3 616726 DRC4 605178
17p12 Ciliary dyskinesia, primary, 40 AR 3 618300 DNAH9 603330
17q12 ?Ciliary dyskinesia, primary, 41 AR 3 618449 GAS2L2 611398
17q21.2 Ciliary dyskinesia, primary, 35 AR 3 617092 ODAD4 617095
17q21.31 Ciliary dyskinesia, primary, 17 AR 3 614679 DNAAF19 614677
17q25.1 Ciliary dyskinesia, primary, 9, with or without situs inversus AR 3 612444 DNAI2 605483
17q25.1 Ciliary dyskinesia, primary, 43 AD 3 618699 FOXJ1 602291
17q25.3 Ciliary dyskinesia, primary, 15 AR 3 613808 CCDC40 613799
19p13.2 Ciliary dyskinesia, primary, 30 AR 3 616037 ODAD3 615956
19q13.33 Ciliary dyskinesia, primary, 20 AR 3 615067 ODAD1 615038
19q13.42 Ciliary dyskinesia, primary, 2 AR 3 606763 DNAAF3 614566
21q22.11 Ciliary dyskinesia, primary, 26 AR 3 615500 CFAP298 615494
21q22.2 Ciliary dyskinesia, primary, 51 AR 3 620438 BRWD1 617824
21q22.3 Ciliary dyskinesia, primary, 24 AR 3 615481 RSPH1 609314
Xq22.3 Ciliary dyskinesia, primary, 36, X-linked XLR 3 300991 DNAAF6 300933

TEXT

A number sign (#) is used with this entry because of evidence that primary ciliary dyskinesia-51 (CILD51) is caused by homozygous or compound heterozygous mutation in the BRWD1 gene (617824) on chromosome 21q22.


Description

Primary ciliary dyskinesia-51 (CILD51) is characterized by male infertility due to multiple morphologic abnormalities of the sperm flagella (MMAF), resulting in severely reduced progressive motility. Some men also have a low sperm count. In addition, affected individuals experience chronic rhinosinusitis and bronchitis, and recurrent upper and lower respiratory infections, and some exhibit dextrocardia and/or situs inversus (Guo et al., 2021).

For a discussion of genetic heterogeneity of primary ciliary dyskinesia, see CILD1 (244400).


Clinical Features

Guo et al. (2021) reported 3 unrelated Chinese men (F1, F2, and F3) with infertility due to MMAF as well as chronic respiratory illnesses, and mutation in the BRWD1 gene. Semen analysis in all 3 men revealed severely reduced progressive sperm motility, with increased percentages of abnormal flagella, including absent, short, and irregular-caliber flagella. Probands F2 and F3 also showed markedly reduced sperm counts. Transmission electron microscopy (TEM) of patient sperm (from F1 and F2) showed absence of the inner and outer dynein arms (IDAs and ODAs), and this was confirmed by immunostaining; however, the outer doublet microtubule (DMT) number and central pair complex were normal. All 3 men also had a history of chronic wet cough, nasal obstruction, and pneumonia early in life, which developed into chronic rhinosinusitis and bronchitis and recurrent upper and lower respiratory tract infections in adulthood. CT of the paranasal sinuses in F1 and F2 confirmed chronic rhinosinusitis with a narrowed sinus cavity. Chest CT revealed bronchiectasis and dextrocardia in both, and F1 also had situs inversus. Nasal nitric oxide testing in F1, who had relatively more severe respiratory symptoms than the other 2 probands, showed a level below the diagnostic cutoff for primary ciliary dyskinesia, whereas nasal nitric oxide in F2 was normal; F3 was not tested. TEM of respiratory cilia from patient F1 revealed defects in the IDAs and ODAs as well as a frequent absence of outer DMTs (ranging from 8+2 to 5+2). Staining for markers of the ODAs confirmed their absence in respiratory epithelial cells from F1. In addition, the overall number of cilia was decreased in F1, and cilia on the free surface of respiratory epithelial cells were notably shorter and fewer than those of healthy controls.


Inheritance

The transmission pattern of CILD51 in the families reported by Guo et al. (2021) was consistent with autosomal recessive inheritance.


Molecular Genetics

In a cohort of 53 infertile Chinese men with MMAF and respiratory symptoms consistent with primary ciliary dyskinesia, who were negative for mutation in known male infertility-associated genes, Guo et al. (2021) performed exome sequencing and identified biallelic missense mutations in 3 unrelated men (see, e.g., 617824.0001 and 617824.0002). The mutations segregated with disease in all 3 families and were not found in 442 Han Chinese controls, including 219 men with isolated oligoasthenozoospermia and 223 fertile controls. The mutations either were not found or were present at very low minor allele frequency in public variant databases.


Animal Model

Pattabiraman et al. (2015) generated Brwd1 -/- mice, noting that both mutant males and mutant females are infertile. The authors found that loss of Brwd1 causes a sexually dimorphic phenotype in male and female germ cells due to very different underlying mechanisms: in males, meiotic division is unaffected, but spermiogenesis is abnormal, whereas in females, the oocyte-to-embryo transition is blocked. Microarray analysis of wildtype and mutant testes revealed that approximately 300 genes were misregulated in Brwd1 -/- mutants; the ontologies and functions of those genes included spermatogenesis, cytoskeletal dynamics, flagellar formation, chromatin organization, sperm motility, protein dynamics, and metabolic pathways, consistent with the Brwd1 mutant phenotype. FLAG-tagged BRWD1 associated with chromatin when expressed in HEK cells; the authors suggested that BRWD1 might be part of unique postmeiotic transcriptional-activator complexes that interact with acetylated histones around postmeiotic genes.


REFERENCES

  1. Guo, T., Tu, C.-F., Yang, D.-H., Ding, S.-Z., Lei, C., Wang, R.-C., Liu, L., Kang, X., Shen, X.-Q., Yang, Y.-F., Tan, Z.-P., Tan, Y.-Q., Luo, H. Bi-allelic BRWD1 variants cause male infertility with asthenoteratozoospermia and likely primary ciliary dyskinesia. Hum. Genet. 140: 761-773, 2021. [PubMed: 33389130, related citations] [Full Text]

  2. Pattabiraman, S., Baumann, C., Guisado, D., Eppig, J. J., Schimenti, J. C., De La Fuente, R. Mouse BRWD1 is critical for spermatid postmeiotic transcription and female meiotic chromosome stability. J. Cell Biol. 208: 53-69, 2015. [PubMed: 25547156, images, related citations] [Full Text]


Creation Date:
Marla J. F. O'Neill : 06/27/2023
Edit History:
alopez : 06/27/2023

# 620438

CILIARY DYSKINESIA, PRIMARY, 51; CILD51


ORPHA: 244;   MONDO: 0957396;  


Phenotype-Gene Relationships

Location Phenotype Phenotype
MIM number
Inheritance Phenotype
mapping key
Gene/Locus Gene/Locus
MIM number
21q22.2 Ciliary dyskinesia, primary, 51 620438 Autosomal recessive 3 BRWD1 617824

TEXT

A number sign (#) is used with this entry because of evidence that primary ciliary dyskinesia-51 (CILD51) is caused by homozygous or compound heterozygous mutation in the BRWD1 gene (617824) on chromosome 21q22.


Description

Primary ciliary dyskinesia-51 (CILD51) is characterized by male infertility due to multiple morphologic abnormalities of the sperm flagella (MMAF), resulting in severely reduced progressive motility. Some men also have a low sperm count. In addition, affected individuals experience chronic rhinosinusitis and bronchitis, and recurrent upper and lower respiratory infections, and some exhibit dextrocardia and/or situs inversus (Guo et al., 2021).

For a discussion of genetic heterogeneity of primary ciliary dyskinesia, see CILD1 (244400).


Clinical Features

Guo et al. (2021) reported 3 unrelated Chinese men (F1, F2, and F3) with infertility due to MMAF as well as chronic respiratory illnesses, and mutation in the BRWD1 gene. Semen analysis in all 3 men revealed severely reduced progressive sperm motility, with increased percentages of abnormal flagella, including absent, short, and irregular-caliber flagella. Probands F2 and F3 also showed markedly reduced sperm counts. Transmission electron microscopy (TEM) of patient sperm (from F1 and F2) showed absence of the inner and outer dynein arms (IDAs and ODAs), and this was confirmed by immunostaining; however, the outer doublet microtubule (DMT) number and central pair complex were normal. All 3 men also had a history of chronic wet cough, nasal obstruction, and pneumonia early in life, which developed into chronic rhinosinusitis and bronchitis and recurrent upper and lower respiratory tract infections in adulthood. CT of the paranasal sinuses in F1 and F2 confirmed chronic rhinosinusitis with a narrowed sinus cavity. Chest CT revealed bronchiectasis and dextrocardia in both, and F1 also had situs inversus. Nasal nitric oxide testing in F1, who had relatively more severe respiratory symptoms than the other 2 probands, showed a level below the diagnostic cutoff for primary ciliary dyskinesia, whereas nasal nitric oxide in F2 was normal; F3 was not tested. TEM of respiratory cilia from patient F1 revealed defects in the IDAs and ODAs as well as a frequent absence of outer DMTs (ranging from 8+2 to 5+2). Staining for markers of the ODAs confirmed their absence in respiratory epithelial cells from F1. In addition, the overall number of cilia was decreased in F1, and cilia on the free surface of respiratory epithelial cells were notably shorter and fewer than those of healthy controls.


Inheritance

The transmission pattern of CILD51 in the families reported by Guo et al. (2021) was consistent with autosomal recessive inheritance.


Molecular Genetics

In a cohort of 53 infertile Chinese men with MMAF and respiratory symptoms consistent with primary ciliary dyskinesia, who were negative for mutation in known male infertility-associated genes, Guo et al. (2021) performed exome sequencing and identified biallelic missense mutations in 3 unrelated men (see, e.g., 617824.0001 and 617824.0002). The mutations segregated with disease in all 3 families and were not found in 442 Han Chinese controls, including 219 men with isolated oligoasthenozoospermia and 223 fertile controls. The mutations either were not found or were present at very low minor allele frequency in public variant databases.


Animal Model

Pattabiraman et al. (2015) generated Brwd1 -/- mice, noting that both mutant males and mutant females are infertile. The authors found that loss of Brwd1 causes a sexually dimorphic phenotype in male and female germ cells due to very different underlying mechanisms: in males, meiotic division is unaffected, but spermiogenesis is abnormal, whereas in females, the oocyte-to-embryo transition is blocked. Microarray analysis of wildtype and mutant testes revealed that approximately 300 genes were misregulated in Brwd1 -/- mutants; the ontologies and functions of those genes included spermatogenesis, cytoskeletal dynamics, flagellar formation, chromatin organization, sperm motility, protein dynamics, and metabolic pathways, consistent with the Brwd1 mutant phenotype. FLAG-tagged BRWD1 associated with chromatin when expressed in HEK cells; the authors suggested that BRWD1 might be part of unique postmeiotic transcriptional-activator complexes that interact with acetylated histones around postmeiotic genes.


REFERENCES

  1. Guo, T., Tu, C.-F., Yang, D.-H., Ding, S.-Z., Lei, C., Wang, R.-C., Liu, L., Kang, X., Shen, X.-Q., Yang, Y.-F., Tan, Z.-P., Tan, Y.-Q., Luo, H. Bi-allelic BRWD1 variants cause male infertility with asthenoteratozoospermia and likely primary ciliary dyskinesia. Hum. Genet. 140: 761-773, 2021. [PubMed: 33389130] [Full Text: https://doi.org/10.1007/s00439-020-02241-4]

  2. Pattabiraman, S., Baumann, C., Guisado, D., Eppig, J. J., Schimenti, J. C., De La Fuente, R. Mouse BRWD1 is critical for spermatid postmeiotic transcription and female meiotic chromosome stability. J. Cell Biol. 208: 53-69, 2015. [PubMed: 25547156] [Full Text: https://doi.org/10.1083/jcb.201404109]


Creation Date:
Marla J. F. O'Neill : 06/27/2023

Edit History:
alopez : 06/27/2023