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SALL4 deletions are a common cause of Okihiro and acro-renal-ocular syndromes and confirm haploinsufficiency as the pathogenic mechanism

[DOI] [PMC free article] [PubMed] [Google Scholar] 163.Smith AD, Jernern F, Refsum H

Langmuir 40 (29), 1483814846

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These limitations highlight the need for next-generation agents engineered for enhanced CNS delivery, including prodrug strategies, receptor-mediated transcytosis and nanocarrier-based transport systems