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Bialleleic PKD1 mutations underlie early-onset autosomal dominant polycystic kidney disease in Saudi Arabian families.
Pediatr Nephrol. 2019 Sep;34(9):1615-1623. doi: 10.1007/s00467-019-04267-x. Epub 2019 May 11.
Pediatr Nephrol. 2019.
PMID: 31079206
Long-term correction of Sandhoff disease following intravenous delivery of rAAV9 to mouse neonates.
Walia JS, Altaleb N, Bello A, Kruck C, LaFave MC, Varshney GK, Burgess SM, Chowdhury B, Hurlbut D, Hemming R, Kobinger GP, Triggs-Raine B.
Walia JS, et al. Among authors: altaleb n.
Mol Ther. 2015 Mar;23(3):414-22. doi: 10.1038/mt.2014.240. Epub 2014 Dec 17.
Mol Ther. 2015.
PMID: 25515709
Free PMC article.
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