Results 81 to 90 of about 2,912 (167)

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

Modeling Tay-Sachs Disease in Astrocyte-like Cells Reveals Significant Changes in the Transcriptomic Profile. [PDF]

Int J Mol Sci
Suárez-García DA, Espejo-Mojica AJ, Alméciga-Díaz CJ.   +2 more
europepmc   +1 more source

[Late-onset manifestation of Tay-Sachs disease-A disease of the cerebellum and motor neurons with psychiatric sequelae]. [PDF]

Nervenarzt
Mengel KE, Mendoza G, Mani L, Deckert J, Arash-Kaps L.   +4 more
europepmc   +1 more source

Application of Mesenchymal Stromal Cells and Their Exosomes in Neurodegenerative Diseases and Lysosomal Storage Diseases. [PDF]

Cells
Ayupova AI, Sidorova AS, Luzina EA, Sufianov AA, Sufianova GZ, Zaynutdinov AM, Rizvanov AA, Solovyeva VV.   +7 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

Trends in the Engineering of Adeno-Associated Virus (AAV) for Precision Gene Delivery to the Central Nervous System (CNS). [PDF]

Int J Mol Sci
Oloruntimehin S, Malogolovkin A.
europepmc   +1 more source

Cerebrospinal delivery of a bidirectional AAV9 vector improves optic nerve and retinal pathology in a sheep model of Tay-Sachs disease. [PDF]

Mol Ther
Story BD, Thomasy SM, Adelman SA, Taghian T, Lopez Y, Ardon M, Shiraishi H, Khan S, Khan M, Do M, Benatti HR, Bertrand S, Prestigiacomo R, Gately R, Martin DR, Sena-Esteves M, Xie J, Gao G, Teixeira LBC, Shaw GC, Marsh-Armstrong N, Gray-Edwards HL.   +21 more
europepmc   +1 more source

Sphingolipidoses: expanding the spectrum of α-synucleinopathies. [PDF]

J Neural Transm (Vienna)
Erskine D, Bronowska AK, Outeiro TF, Attems J.   +3 more
europepmc   +1 more source

Epigenetic landscape in lysosomal storage disorders: mechanisms and modulation. [PDF]

Front Genet
Leal AF, Pachajoa H, Tomatsu S.
europepmc   +1 more source

Long-term follow-up of a Tay-Sachs disease patient with cherry-red spot. [PDF]

Am J Ophthalmol Case Rep
Tsutsumi N, Sakata S, Ikari R, Ebihara N.   +3 more
europepmc   +1 more source