Table 1.
Potential pathogenic variants were identified in 19 of the analysed families.
Family ID | Patient ID | Affected gene | Inherit –ance mode | Identified variant | Zygosity | Origin (as per segregation analysis) | ACMG classifi-cation | Reference |
---|---|---|---|---|---|---|---|---|
EYE42 | EYE42 | NMNAT1 | AR | c.1A > G;p.? | Het | Maternal | P | 13 |
c.709C > T;p.(R237C) | Het | Paternal | P | 12,13 | ||||
EYE68 | EYE68 | CRB1 | AR | c.668dupT;p.(L223Ffs*4) | Het | Maternal | P | Novel |
c.733dupG;p.(A245Gfs*16) | Het | Maternal | P | Novel | ||||
c.1567dupC;p.(L523Pfs*28) | Het | Paternal | P | Novel | ||||
EYE69 | EYE69 | CEP290 | AR | c.2390delA;p.(K797Sfs*2) | Het | Maternal | P | 43 |
c.6889A > T;p.(K2297*) | Het | Paternal | P | Novel | ||||
EYE70 | EYE70 | CRX | AD | c.124G > A;p.(E42K) | Het | de novo | P | 15 |
EYE115 | EYE115 | CRB1 | AR | c.1334_1740del;p.(C445Yfs*8) | Het | Maternal | P | Novel |
c.1576C > T;p.(R526*) | Het | Paternal | P | 42 | ||||
EYE121 | EYE121 | CRB1 | AR | c.2T > C;p.? | Het | NAc | P | Novel |
c.3068T > G;p.(L1023R) | Het | Maternal | L.P | Novel | ||||
EYE125 | EYE125 | IMPDH1 | AD | c.590A > C;p.(Q197P) | Het | de novo | L.P | Novel |
EYE139 | EYE139 | GUCY2D | AR | c.2765A > G;p.(Y922C) | Het | Maternal | L.P | Novel |
c.2983C > T;p.(R995W) | Het | Paternal | P | 14 | ||||
EYE149 | EYE149 | RPGRIP1 | AR | c.799C > T;p.(R267*) | Het | Paternal | P | 15 |
c.1687C > T;p.(R563*) | Het | Maternal | P | Novel | ||||
EYE156 | EYE156 | PRPH2 | AR | c.730_736delinsCAGCTCCTCCAGACGGGTGCACCAGAC;p.(N244Qfs*19) | Het | Maternal | P | Novel |
c.748T > G;p.(C250G) | Het | Paternal | L.P | Novel | ||||
EYE159 | EYE159 | NMNAT1 | AR | c.196C > T;p.(R66W) | Het | Maternal | P | 13 |
c.709C > T;p.(R237C) | Het | Paternal | P | 12,13 | ||||
JIKEI-145 | JU1039 | LRAT | AR | c.163C > T;p.(R55W) | Het | Paternal | P | 33 |
Exon 1–3 deletion | Het | Maternal | P | Novel | ||||
JU1040 | LRAT | AR | c.163C > T;p.(R55W) | Het | Paternal | P | 33 | |
Exon 1–3 deletion | Het | Maternal | P | Novel | ||||
S132 | S132 | NMNAT1 | AR | c.196C > T;p.(R66W) | Het | Paternal | P | 13 |
c.709C > T;p.(R237C) | Het | Maternal | P | 12,13 | ||||
EYE50 | EYE50 | RPGR | XL | c.977A > C;p.(K326T) | Hemi | Maternal | L.P | Novel |
EYE114 | EYE114 | RP2 | XL | c.769–2A > G | Hemi | Maternal | P | Novel |
EYE187 | EYE187 | BEST1 | AD | c.682G > T;p.(D228Y) | Het | Paternal (mosaic)b | L.P | Novel |
EYE20a | EYE20 | RPGRIP1 | AR | c.3565_3571delCGAAGGC;p.(R1189Gfs*7) | Hom | Biparental | P | 42 |
EYE64 | RPGRIP1 | AR | c.3565_3571delCGAAGGC;p.(R1189Gfs*7) | Hom | Biparental | P | 42 | |
EYE65 | RPGRIP1 | AR | c.3565_3571delCGAAGGC;p.(R1189Gfs*7) | Hom | Biparental | P | 42 | |
EYE55a | EYE55 | RPGRIP1 | AR | c.1467+1G > T | Het | Paternal | P | 11 |
Exon 17 deletion (c.2710 + 374_2895 + 74del) | Het | Maternal | P | 11,25 | ||||
LCA1Ha | Twin 1 | GUCY2D | AR | c.2113 + 2_2113 + 3insT | Het | Paternal | P | 10 |
c.2714 T > C;p.(L905P) | Het | Maternal | P | 10 | ||||
Twin 2 | GUCY2D | AR | c.2113 + 2_2113 + 3insT | Het | Paternal | P | 10 | |
c.2714T > C;p.(L905P) | Het | Maternal | P | 10 |
aMutation analyses for families EYE20, EYE55, and LCA1H were reported previously10,11.
bSee Supp. Figure 2 for sequencing electropherogram data.
cDNA was unavailable for the patient’s father, as he was deceased.
NA, not available; Hom, homozygous; Het, heterozygous; Hemi, hemizygous; P, pathogenic; L.P, likely pathogenic; AD, autosomal dominant; AR, autosomal recessive; XL, X-linked.