Investigating Immune Responses to the scAAV9-HEXM Gene Therapy Treatment in Tay–Sachs Disease and Sandhoff Disease Mouse Models
Kot, Shalini, Karumuthil-Melethil, Subha, Woodley, Evan, Zaric, Violeta, Thompson, Patrick, Chen, Zhilin, Lykken, Erik, Keimel, John G., Kaemmerer, William F., Gray, Steven J., Walia, Jagdeep S.
Published in International journal of molecular sciences (01.07.2021)
Published in International journal of molecular sciences (01.07.2021)
Get full text
Journal Article
Intracerebroventricular amyloid-β antibodies reduce cerebral amyloid angiopathy and associated micro-hemorrhages in aged Tg2576 mice
Thakker, Deepak R, Weatherspoon, Marcy R, Harrison, Jonathan, Keene, Thomas E, Lane, Deanna S, Kaemmerer, William F, Stewart, Gregory R, Shafer, Lisa L
Published in Proceedings of the National Academy of Sciences - PNAS (17.03.2009)
Published in Proceedings of the National Academy of Sciences - PNAS (17.03.2009)
Get full text
Journal Article
Onset Time and Durability of Huntingtin Suppression in Rhesus Putamen After Direct Infusion of Antihuntingtin siRNA
Grondin, Richard, Ge, Pei, Chen, Qingmin, Sutherland, Jessica E, Zhang, Zhiming, Gash, Don M, Stiles, David K, Stewart, Gregory R, Sah, Dinah W Y, Kaemmerer, William F
Published in Molecular therapy. Nucleic acids (01.06.2015)
Published in Molecular therapy. Nucleic acids (01.06.2015)
Get full text
Journal Article
The effects of huntingtin-lowering: what do we know so far?
Kaemmerer, William F, Grondin, Richard C
Published in Degenerative neurological and neuromuscular disease (01.03.2019)
Published in Degenerative neurological and neuromuscular disease (01.03.2019)
Get full text
Journal Article
Six-month partial suppression of Huntingtin is well tolerated in the adult rhesus striatum
GRONDIN, Richard, KAYTOR, Michael D, KAEMMERER, William F, YI AI, NELSON, Peter T, THAKKER, Deepak R, HEISEL, Jennifer, WEATHERSPOON, Marcy R, BLUM, Janelle L, BURRIGHT, Eric N, ZHIMING ZHANG
Published in Brain (London, England : 1878) (01.04.2012)
Published in Brain (London, England : 1878) (01.04.2012)
Get full text
Journal Article
Treatment of GM2 Gangliosidosis in Adult Sandhoff Mice Using an Intravenous Self-Complementary Hexosaminidase Vector
Osmon, Karlaina Jl, Thompson, Patrick, Woodley, Evan, Karumuthil-Melethil, Subha, Heindel, Cliff, Keimel, John G, Kaemmerer, William F, Gray, Steven J, Walia, Jagdeep S
Published in Current gene therapy (01.06.2022)
Published in Current gene therapy (01.06.2022)
Get more information
Journal Article
A majority of Huntington's disease patients may be treatable by individualized allele-specific RNA interference
Lombardi, Maria Stella, Jaspers, Leonie, Spronkmans, Christine, Gellera, Cinzia, Taroni, Franco, Di Maria, Emilio, Donato, Stefano Di, Kaemmerer, William F.
Published in Experimental neurology (01.06.2009)
Published in Experimental neurology (01.06.2009)
Get full text
Journal Article
Widespread suppression of huntingtin with convection-enhanced delivery of siRNA
Stiles, David K., Zhang, Zhiming, Ge, Pei, Nelson, Brian, Grondin, Richard, Ai, Yi, Hardy, Peter, Nelson, Peter T., Guzaev, Andrei P., Butt, Mark T., Charisse, Klaus, Kosovrasti, Verbena, Tchangov, Lubomir, Meys, Michael, Maier, Martin, Nechev, Lubomir, Manoharan, Muthiah, Kaemmerer, William F., Gwost, Douglas, Stewart, Gregory R., Gash, Don M., Sah, Dinah W.Y.
Published in Experimental neurology (01.01.2012)
Published in Experimental neurology (01.01.2012)
Get full text
Journal Article
Adeno-associated virus type 2 vectors: transduction and long-term expression in cerebellar Purkinje cells in vivo is mediated by the fibroblast growth factor receptor 1: bFGFR-1 mediates AAV2 transduction of Purkinje cells
Belur, Lalitha R., Kaemmerer, William F., McIvor, R. Scott, Low, Walter C.
Published in Archives of virology (01.11.2008)
Published in Archives of virology (01.11.2008)
Get full text
Journal Article
87 Intrathecal continuous or intermittent bolus delivery of laronidase results in elevated levels in canine CNS tissue
Keimel, John, Keimel, John G., Passage, Merry, Billstrom, Tina, Lentz, Linnea, Rose, Rebecca, Kaemmerer, William F.
Published in Molecular genetics and metabolism (01.12.2007)
Published in Molecular genetics and metabolism (01.12.2007)
Get full text
Journal Article
Adeno-associated virus type 2 vectors: transduction and long-term expression in cerebellar Purkinje cells in vivo is mediated by the fibroblast growth factor receptor 1
Belur, Lalitha R, Kaemmerer, William F, McIvor, R. Scott, Low, Walter C
Published in Archives of virology (01.11.2008)
Published in Archives of virology (01.11.2008)
Get full text
Journal Article
Intracerebroventricular amyloid-[beta] antibodies reduce cerebral amyloid angiopathy and associated micro-hemorrhages in aged Tg2576 mice
Thakker, Deepak R, Weatherspoon, Marcy R, Harrison, Jonathan, Keene, Thomas E, Lane, Deanna S, Kaemmerer, William F, Stewart, Gregory R, Shafer, Lisa L
Published in Proceedings of the National Academy of Sciences - PNAS (17.03.2009)
Get full text
Published in Proceedings of the National Academy of Sciences - PNAS (17.03.2009)
Journal Article
Intracerebroventricular amyloid-β antibodies reduce cerebral amyloid angiopathy and associated micro-hemorrhages in aged Tg2576 mice
Thakker, Deepak R, Weatherspoon, Marcy R, Harrison, Jonathan, Keene, Thomas E, Lane, Deanna S, Kaemmerer, William F, Stewart, Gregory R, Shafer, Lisa L
Published in Proceedings of the National Academy of Sciences - PNAS (2009)
Get full text
Published in Proceedings of the National Academy of Sciences - PNAS (2009)
Journal Article
WITHDRAWN: 32 Intrathecal continuous or intermittent bolus delivery of laronidase results in elevated levels in canine CNS tissue
Keimel, John, Keimel, John G., Lentz, Linnea, Passage, Merry, Rose, Rebecca, Billstrom, Tina, Kaemmerer, William F.
Published in Molecular genetics and metabolism (01.10.2007)
Published in Molecular genetics and metabolism (01.10.2007)
Get full text
Journal Article