Three novel SLC37A4 variants in glycogen storage disease type 1b and a literature review.
J Int Med Res
; 51(12): 3000605231216633, 2023 Dec.
Article
en En
| MEDLINE
| ID: mdl-38087503
Glycogen storage disease type 1b (GSD1b) is a rare genetic disorder, resulting from mutations in the SLC37A4 gene located on chromosome 11q23.3. Although the SLC37A4 gene has been identified as the pathogenic gene for GSD1b, the complete variant spectrum of this gene remains to be fully elucidated. In this study, we present three patients diagnosed with GSD1b through genetic testing. We detected five variants of the SLC37A4 gene in these three patients, with three of these mutations (p. L382Pfs*15, p. G117fs*28, and p. T312Sfs*13) being novel variants not previously reported in the literature. We also present a literature review and general overview of the currently reported SLC37A4 gene variants. Our study expands the mutation spectrum of SLC37A4, which may help enable genetic testing to facilitate prompt diagnosis, appropriate intervention, and genetic counseling for affected families.
Palabras clave
Texto completo:
1
Colección:
01-internacional
Banco de datos:
MEDLINE
Asunto principal:
Enfermedad del Almacenamiento de Glucógeno
/
Enfermedad del Almacenamiento de Glucógeno Tipo I
Límite:
Humans
Idioma:
En
Revista:
J Int Med Res
Año:
2023
Tipo del documento:
Article
País de afiliación:
China