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PUBLICATIONS

Biochemical Correction of GM2 Ganglioside Accumulation in AB-Variant GM2 Gangliosidosis

Deschenes NM, Cheng C, Ryckman AE, Quinville BM, Khanal P, Mitchell M, Chen Z, Sangrar W, Gray SJ, Walia JS

Gene Expression Profile in the Sandhoff Mouse Brain with Progression of Age.

Singh K, Quinville BM, Mitchell M, Chen Z, Walia JS.

A Comprehensive Review: Sphingolipid Metabolism and Implications of Disruption in Sphingolipid Homeostasis.

Quinville BM, Deschenes NM, Ryckman AE, Walia JS.

Metabolism of Glycosphingolipids and Their Role in the Pathophysiology of Lysosomal Storage Disorders.

Ryckman AE, Brockhausen I, Walia JS.

Efficacy of a Bicistronic Vector for Correction of Sandhoff Disease in a Mouse Model

Woodley E, Osmon KJL, Thompson P, Richmond C, Chen Z, Gray SJ, Walia JS.

Battery of Behavioral Tests Assessing General Locomotion, Muscular Strength, and Coordination in Mice.

Osmon KJ, Vyas M, Woodley E, Thompson P, Walia JS.

Systemic Gene Transfer of a Hexosaminidase Variant Using an scAAV9.47 Vector Corrects GM2 Gangliosidosis in Sandhoff Mice.

Osmon KJ, Woodley E, Thompson P, Ong K, Karumuthil-Melethil S, Keimel JG, Mark BL, Mahuran D, Gray SJ, Walia JS.

PARP Inhibitor Drugs in the Treatment of Breast, Ovarian, Prostate and Pancreatic Cancers: An Update of Clinical Trials.

Kamel D, Gray C, Walia JS, Kumar V.

Novel Vector Design and Hexosaminidase Variant Enabling Self-Complementary Adeno-Associated Virus for the Treatment of Tay-Sachs Disease.

Karumuthil-Melethil S, Nagabhushan Kalburgi S, Thompson P, Tropak M, Kaytor MD, Keimel JG, Mark BL, Mahuran D, Walia JS, Gray SJ.

Construction of a hybrid β-hexosaminidase subunit capable of forming stable homodimers that hydrolyze GM2 ganglioside in vivo.

Tropak MB, Yonekawa S, Karumuthil-Melethil S, Thompson P, Wakarchuk W, Gray SJ, Walia JS, Mark BL, Mahuran D.

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.

Autologous transplantation of lentivector/acid ceramidase-transduced hematopoietic cells in nonhuman primates.

Walia JS, Neschadim A, Lopez-Perez O, Alayoubi A, Fan X, Carpentier S, Madden M, Lee CJ, Cheung F, Jaffray DA, Levade T, McCart JA, Medin JA.

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