Long-Term Correction of Sandhoff Disease Following Intravenous Delivery of rAAV9 to Mouse Neonates von Walia, Jagdeep S, Altaleb, Naderah, Bello, Alexander, Kruck, Christa, LaFave, Matthew C, Varshney, Gaurav K, Burgess, Shawn M, Chowdhury, Biswajit, Hurlbut, David, Hemming, Richard, Kobinger, Gary P, Triggs-Raine, Barbara

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Bialleleic PKD1 mutations underlie early-onset autosomal dominant polycystic kidney disease in Saudi Arabian families von Al-Hamed, Mohamed H., Alsahan, Nada, Rice, Sarah J., Edwards, Noel, Nooreddeen, Eman, Alotaibi, Maha, Kurdi, Wesam, Alnemer, Maha, Altaleb, Naderah, Ali, Wafa, Al-Numair, Nouf, Almejaish, Najd, Sayer, John A., Imtiaz, Faiqa

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