Focuses on substrate deprivation as treatment for Sandhoof disease, a neurodegenerative disorder. Animals and treatment procedures; Behavioral tests; Horizontal bar test; Biochemical analysis; Histology; Electron microscopy; Symptom onset and life expectancy.Butters...
The present disclosure provides methods for the treatment of lysosomal storage disorders using gene replacement therapy. In particular, methods are provided for the treatment of Tay-Sachs disease, Sandhoff Disease, and GM1-gangliosidosis using enzyme replacement therapy. Expression constructs encoding enzymes...
Cats treated in the early symptomatic phase showed delayed disease progression and a significant survival increase versus untreated cats. Treatment was less effective when administered later in the disease course, although therapeutic benefit was still possible. Results are encouraging for the treatment of...
Sandhoff Disease (SD) involves the CNS accumulation of ganglioside GM2 and asialo-GM2 (GA2) due to inherited defects in the β-subunit gene of β-h
Multi-target-directed triazole derivatives as promising agents for the treatment of Alzheimer's disease. Bioorg Chem. 2019 Jun;87:572-584. doi: https://doi.org/10.1016/j.bioorg.2019.03.058. Epub 2019 Mar 23. PMID: 30928879. 31. Chen SG, Gu RX, Dai H, Wei DQ. Virtual screening for ...
B-DGJ treatment, administered from postnatal day 2 (p-2) to p-5 (600 mg/kg/day)), significantly reduced total brain ganglioside and GM2 content in the Sandhoff disease (−/−) mice, but did not reduce the content of GA2. We also found that B-DGJ treatment caused a slight, but...
Novel vector design and hexosaminidase variant enabling self-complementary adeno-associated virus for the treatment of Tay–Sachs disease. Hum Gene Ther. 2016;27:509–21. CAS PubMed PubMed Central Google Scholar Osmon KJ, Woodley E, Thompson P, Ong K, Karumuthil-Melethil S, Keimel JG, et...
AframeshiftmutationinthecanineHEXBgeneintoypoodleswithGM2gangliosidosisvariant0(Sandhoffdisease) MohammadM.Rahmana(b)Hye-SookChanga,KeijiroMizukamia,MohammadA.Hossaina(b)kiraYabukia,ShinjiTamurac,MasatoKitagawad,SawaneMitania(e)TakashiHigoe,ohammadM.Uddina(b)KazuyukiUchidaf,OsamuYamatoa,⇑ ...
The present disclosure provides methods for the treatment of lysosomal storage disorders using gene replacement therapy. In particular, methods are provided for the treatment of Tay-Sachs disease, Sandhoff Disease, and GM1-gangliosidosis using enzyme replacement therapy. Expression constructs encoding ...
Treatment was less effective when administered later in the disease course, although therapeutic benefit was still possible. Results are encouraging for the treatment of human patients and provide support for the development AAV-gene therapy for human SD....