Predominant cellular mitochondrial dysfunction in the TOP3A gene-caused Bloom syndrome-like disorder

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Cellular defects caused by hypomorphic variants of the Bloom syndrome helicase gene BLM.

BACKGROUND Bloom syndrome is an autosomal recessive disorder characterized by extraordinary cancer incidence early in life and an average life expectancy of ~27 years. Premature stop codons in BLM, which encodes a DNA helicase that functions in DNA double-strand-break repair, make up the vast majority of Bloom syndrome mutations, with only 13 single amino acid changes identified in the syndrome...

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ژورنال

عنوان ژورنال: Biochimica et Biophysica Acta (BBA) - Molecular Basis of Disease

سال: 2021

ISSN: 0925-4439

DOI: 10.1016/j.bbadis.2021.166106