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Harada H, Itasaka S, Kizaka-Kondoh S, Shibuya K, Morinibu A, Shinomiya K, et al

This strategy targets single-gene disorders and has strong preclinical and early-phase clinical rationale but remains experimental, as its long-term efficacy and safety are still under evaluation [99].Ex vivo Genome editing to directly repair pathogenic variants in autologous HSCs or to modulate regulatory elements can potentially avoid random vector insertions and achieve better physiological gene regulation, but it raises concerns about off-target edits, mosaicism, and the requirement for careful safety validation prior to wider clinical use [104]

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In addition to 1112 brains per group at P5 and P9, brains from seven preterm newborn pigs were collected at P1 for immunohistochemical analysis

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[DOI] [PubMed] [Google Scholar] 122.Health Canada Canadian nutrient file (tryptophan) (Accessed on January 26, 2010)