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Tyrosine Hydroxylase Deficiency Impairs TH Axonal Transport, Brain Function, and Neuronal Plasticit
Author: Shi, Tie-Jun Sten; Jung-kc, Kunwar; Lyu, Gong-Wei; Han, Liang; Ying, Ming; Thony, Beat; Martinez, Aurora
Journal: JOURNAL OF INHERITED METABOLIC DISEASE. 2026; Vol. 49, Issue 2, pp. -. DOI: 10.1002/jimd.70169
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Signal Peptide Engineering and Codon Optimization to Enhance α-Gal A Activity for rAAV Gene Therapy of Fabry Diseas
Author: He, Siwu; Yang, Li; Wan, Ke; Zheng, Zhaoyue; Leng, Mi; Gan, Chunmei; Liu, Yu; Xiao, Lin; Ye, Jingya; Chen, Zhian; Zhou, Jiao; Valencia, C. Alexander; Abuammah, Aliah; Chow, Hoi Yee; Dong, Biao; Chen, Yucheng
Journal: JOURNAL OF INHERITED METABOLIC DISEASE. 2026; Vol. 49, Issue 2, pp. -. DOI: 10.1002/jimd.70168
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Pharmacodynamics, Efficacy, and Safety of Intraputaminal Eladocagene Exuparvovec Administered to Pediatric Patients With Aromatic L-Amino Acid Decarboxylase Deficiency Using an MR-Compatible Cannula: 48 Weeks of Follow-U
Author: Curry, Daniel J.; Pearl, Phillip L.; Stone, Scellig S. D.; Gilbert, Donald L.; Vadivelu, Sudhakar; Ben-zeev, Bruria; Vestal, Matthew; Zafar, Muhammad; Tai, Chun-Hwei; Chou, Sheng-Che; Zibly, Zion; Ungar, Lior; Parnes, Mered; Hull, Mariam; Emrick, Lisa; Werner, Christian; Krolick, Alexis; Penematsa, Vinay; Wang, Antonia; Rahman, Rezwanur; Golden, Lee; Chien, Yin-Hsiu; Hwu, Paul Wuh-Liang
Journal: JOURNAL OF INHERITED METABOLIC DISEASE. 2026; Vol. 49, Issue 2, pp. -. DOI: 10.1002/jimd.70151
