A novel compound heterozygous variant of ECHS1 identified in a Japanese patient with Leigh syndromeHuman Genome Variation - Tập 6 Số 1
Shumpei Uchino, Aritoshi Iida, Atsushi Sato, Keiko Ishikawa, Masakazu Mimaki, Ichizo Nishino, Yu‐ichi Goto
AbstractLeigh syndrome (LS) is a heterogeneous neurodegenerative disorder caused by mitochondrial dysfunction. Certain LS cases have mutations in ECHS1, which encodes a short-chain enoyl-CoA hydratase involved in the metabolism of fatty acids and branched-chain amino acids in mitochondria. Using exome sequencing, we diagnosed a Japanese p...... hiện toàn bộ