Results 71 to 80 of about 4,845,721 (162)

Investigating Immune Responses to the scAAV9-HEXM Gene Therapy Treatment in Tay-Sachs Disease and Sandhoff Disease Mouse Models. [PDF]

Int J Mol Sci, 2021
Kot S, Karumuthil-Melethil S, Woodley E, Zaric V, Thompson P, Chen Z, Lykken E, Keimel JG, Kaemmerer WF, Gray SJ, Walia JS.   +10 more
europepmc   +1 more source

Effect of Yuzu (Citrus junos) Seed Limonoids and Spermine on Intestinal Microbiota and Hypothalamic Tissue in the Sandhoff Disease Mouse Model. [PDF]

Med Sci (Basel), 2021
Minamisawa M, Suzumura T, Bose S, Taniai T, Kawai G, Suzuki K, Yamaguchi A, Yamanaka S.   +7 more
europepmc   +1 more source

N-butyldeoxygalactonojirimycin reduces brain ganglioside and GM2 content in neonatal Sandhoff disease mice.

, 2008
Sandhoff disease involves the CNS accumulation of ganglioside GM2 and asialo-GM2 (GA2) due to inherited defects in the beta-subunit gene of beta-hexosaminidase A and B (Hexb gene).
Baek, RC, Platt, Frances, Seyfried, TN, Kasperzyk, JL, Platt, FM   +4 more
core   +1 more source

Therapeutic benefit after intracranial gene therapy delivered during the symptomatic stage in a feline model of Sandhoff disease. [PDF]

Gene Ther, 2021
McCurdy VJ, Johnson AK, Gray-Edwards HL, Randle AN, Bradbury AM, Morrison NE, Hwang M, Baker HJ, Cox NR, Sena-Esteves M, Martin DR.   +10 more
europepmc   +1 more source

Investigating Sandhoff Disease in Saskatchewan

, 2013
A Thesis Submitted to the Faculty of Graduate Studies and Research in Partial Fulfillment of the Requirements for the Degree of Doctor of Philosophy in Biology, University of Regina. xii, 142p.
openaire   +2 more sources

Sphingosine kinase 1/S1P receptor signaling axis controls glial proliferation in mice with Sandhoff disease

, 2008
Sphingosine-1-phosphate (S1P) is a lipid-signaling molecule produced by sphingosine kinase in response to a wide number of stimuli. By acting through a family of widely expressed G protein-coupled receptors, S1P regulates diverse physiological processes.
Kiyomi Mizugishi, Meryem Bektas, Yun-ping Wu, Roger S, Richard L. Proia   +4 more
core  

Abnormal epiphyseal development in a feline model of Sandhoff disease. [PDF]

J Orthop Res, 2020
McNulty MA, Prevatt PB, Nussbaum ER, Randle AN, Johnson AK, Hudson JA, Gray-Edwards HL, Sena-Esteves M, Martin DR, Carlson CS.   +9 more
europepmc   +1 more source

Lysosomal storage and pathologenesis in a novel in vitro cellular model of Sandhoff disease

, 2009
Sandhoff disease is a devastating autosomal recessive GM2 gangliosidosis in which a deficiency of β-hexosaminidase results in lysosomal storage of the enzyme's substrates, including GM2 and GA2 glycolipid as well as glycoprotein-derived oligosaccharides

core  

Bioprinting neural tissue to decode Sandhoff disease: promise and barriers. [PDF]

Ann Med Surg (Lond)
Ullah SH, Waqas M, Fatima M, Talha M, Rehman UU.   +4 more
europepmc   +1 more source

Adult-onset Sandhoff disease presenting with a motor neuron disease phenotype: clinical and mechanistic insights from patient-derived models. [PDF]

Acta Neuropathol Commun
Tang Y, Shan D, Wang H, Ji X, Jiao Y, Li J, Zhan Z, Liu S, Sun X, Lin P, Xu J, Wang D, Sun X, Yan C, Zhao Y, Liu F.   +15 more
europepmc   +1 more source