Postnatal gene restoration in succinic semialdehyde dehydrogenase deficiency (SSADHD) reveals phenotype reversibility
琥珀酸半醛脱氢酶缺乏症 (SSADHD) 的出生后基因恢复揭示了表型可逆性
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doi:10.64898/2026.03.24.713250
Lee, Henry H C; McGinty, Gabrielle; Liebhardt, Amanda; Zhang, Zihe; Welzel, Björn; Vermudez, Sheryl Anne D; Arning, Erland; Lin, Rui; Nguyen, Minh; Cakici, Didem; Yu, Timothy; Woolf, Clifford J; Pearl, Phillip L; Gao, Guangping; Sahin, Mustafa; Rotenberg, Alexander