Results 91 to 100 of about 2,424 (142)

Real-time MR tracking of AAV gene therapy with βgal-responsive MR probe in a murine model of GM1-gangliosidosis. [PDF]

Mol Ther Methods Clin Dev, 2021
Taghian T, Batista AR, Kamper S, Caldwell M, Lilley L, Li H, Rodriguez P, Mesa K, Zheng S, King RM, Gounis MJ, Todeasa S, Maguire A, Martin DR, Sena-Esteves M, Meade TJ, Gray-Edwards HL.   +16 more
europepmc   +1 more source

Canine GM1-Gangliosidosis [PDF]

, 2020
openaire   +1 more source

Quantitative reliability assessment of brain MRI volumetric measurements in type II GM1 gangliosidosis patients. [PDF]

Front Neuroimaging
Zoppo C, Kolstad J, Johnston J, D'Souza P, Kühn AL, Vardar Z, Peker A, Lindsay C, Rentiya ZS, King R, Gray-Edwards H, Vachha B, Acosta MT, Tifft CJ, Shazeeb MS.   +14 more
europepmc   +1 more source

Insights into the Pathobiology of GM1 Gangliosidosis from Single-Nucleus Transcriptomic Analysis of CNS Cells in a Mouse Model. [PDF]

Int J Mol Sci
Liu S, Xie T, Huang Y.
europepmc   +1 more source

Corrigendum to "Natural history progression of MRI brain volumetrics in type II late-infantile and juvenile GM1 gangliosidosis patients" [Molecular Genetics and Metabolism 2025 Mar;144(3):109025]. [PDF]

Mol Genet Metab
Kolstad J, Zoppo C, Johnston JM, D'Souza P, Kühn AL, Vardar Z, Peker A, Hader A, Celik H, Lewis CJ, Lindsay C, Rentiya ZS, Lebel C, Vedantham S, Vachha B, Gray-Edwards HL, Acosta MT, Tifft CJ, Shazeeb MS.   +18 more
europepmc   +1 more source

Examination of a blood-brain barrier targeting β-galactosidase-monoclonal antibody fusion protein in a murine model of GM1-gangliosidosis. [PDF]

Mol Genet Metab Rep, 2021
Przybilla MJ, Stewart C, Carlson TW, Ou L, Koniar BL, Sidhu R, Kell PJ, Jiang X, Jarnes JR, O'Sullivan MG, Whitley CB.   +10 more
europepmc   +1 more source

Case report: Preimplantation genetic testing for infantile GM1 gangliosidosis. [PDF]

Front Genet
Zagaynova VA, Nasykhova YA, Tonyan ZN, Danilova MM, Dvoynova NM, Lazareva TE, Ivashchenko TE, Shabanova ES, Krikheli IO, Lesik EA, Bespalova ON, Kogan IY, Glotov AS.   +12 more
europepmc   +1 more source

Phenotypical changes of satellite glial cells in a murine model of G_M1 -gangliosidosis. [PDF]

J Cell Mol Med, 2022
Huang B, Zdora I, de Buhr N, Eikelberg D, Baumgärtner W, Leitzen E.   +5 more
europepmc   +1 more source

Novel insights into pathomechanisms of retinal neuronal degeneration and reactive gliosis in a murine model of G_M1-gangliosidosis. [PDF]

Sci Rep
Jubran L, Wannemacher R, Ulianytska A, Gerhauser I, Baumgärtner W, Leitzen E.   +5 more
europepmc   +1 more source

A natural history study of pediatric patients with early onset of GM1 gangliosidosis, GM2 gangliosidoses, or gaucher disease type 2 (RETRIEVE). [PDF]

Orphanet J Rare Dis
Héron B, Batzios S, Mengel E, Giugliani R, Patterson M, Gautschi M, Cornelisse P, Trokan L, Schwierin B, Rohrbach M.   +9 more
europepmc   +1 more source