Figure 1. Schematic of the GATA1 gene and known mutations in patients with congenital erythroid disorders.
GATA1 is composed of six exons which encode two transcripts that produce the full-length isoform (upper) or the N-terminally truncated GATA1s isoform (lower). The locations of GATA1 mutations found in patients with congenital erythroid disorders are indicated along GATA1 gene. Mutations at the boundaries of exon 2 (green), mutations within the sequences that encode the N-terminal zinc finger (purple), and those within intron 5 (red) are depicted. The mutations at the boundaries of exon 2 cause splicing defects that result in the exclusive generation of GATA1s, while mutations within exon 4 create variants that attenuate the GATA1-FOG1 interaction or impair DNA binding. The g.7230C>T alteration causes a five-amino acid (PPFWQ) insertion at the C-terminus of the C-terminal zinc finger. GATA1 cofactors and their interacting domains are shown on top. N-TAD, amino-terminal trans-activation domain; N-ZF, amino-terminal zinc finger; C-ZF, carboxyl-terminal zinc finger.