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

Por Tropak, Michael B, Yonekawa, Sayuri, Karumuthil-Melethil, Subha, Thompson, Patrick, Wakarchuk, Warren, Gray, Steven J, Walia, Jagdeep S, Mark, Brian L, Mahuran, Don
Publicado no Mol Ther Methods Clin Dev (2016)

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

Por Karumuthil-Melethil, Subha, Kalburgi, Sahana Nagabhushan, Thompson, Patrick, Tropak, Michael, Kaytor, Michael D., Keimel, John G., Mark, Brian L., Mahuran, Don, Walia, Jagdeep S., Gray, Steven J.
Publicado no Hum Gene Ther (2016)

Bioluminescent imaging of a marking transgene and correction of Fabry mice by neonatal injection of recombinant lentiviral vectors

Por Yoshimitsu, Makoto, Sato, Takeya, Tao, Kesheng, Walia, Jagdeep S., Rasaiah, Vanessa I., Sleep, Gillian T., Murray, Gary J., Poeppl, Armando G., Underwood, John, West, Lori, Brady, Roscoe O., Medin, Jeffrey A.
Publicado em 2004

Long-Term Correction of Sandhoff Disease Following Intravenous Delivery of rAAV9 to Mouse Neonates

Por 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
Publicado no Mol Ther (2015)

Autologous Transplantation of Lentivector/Acid Ceramidase–Transduced Hematopoietic Cells in Nonhuman Primates

Por Walia, Jagdeep S., Neschadim, Anton, Lopez-Perez, Orlay, Alayoubi, Abdulfatah, Fan, Xin, Carpentier, Stéphane, Madden, Melissa, Lee, Chyan-Jang, Cheung, Fred, Jaffray, David A., Levade, Thierry, McCart, J. Andrea, Medin, Jeffrey A.
Publicado em 2011

Experiences of caregivers of children with inherited metabolic diseases: a qualitative study

Por Siddiq, Shabnaz, Wilson, Brenda J., Graham, Ian D., Lamoureux, Monica, Khangura, Sara D., Tingley, Kylie, Tessier, Laure, Chakraborty, Pranesh, Coyle, Doug, Dyack, Sarah, Gillis, Jane, Greenberg, Cheryl, Hayeems, Robin Z., Jain-Ghai, Shailly, Kronick, Jonathan B., Laberge, Anne-Marie, Little, Julian, Mitchell, John J., Prasad, Chitra, Siriwardena, Komudi, Sparkes, Rebecca, Speechley, Kathy N., Stockler, Sylvia, Trakadis, Yannis, Wafa, Sarah, Walia, Jagdeep, Wilson, Kumanan, Yuskiv, Nataliya, Potter, Beth K.
Publicado no Orphanet J Rare Dis (2016)