Results 101 to 110 of about 4,924,951 (177)

AAV gene therapy for Tay-Sachs disease. [PDF]

Nat Med, 2022
Flotte TR, Cataltepe O, Puri A, Batista AR, Moser R, McKenna-Yasek D, Douthwright C, Gernoux G, Blackwood M, Mueller C, Tai PWL, Jiang X, Bateman S, Spanakis SG, Parzych J, Keeler AM, Abayazeed A, Rohatgi S, Gibson L, Finberg R, Barton BA, Vardar Z, Shazeeb MS, Gounis M, Tifft CJ, Eichler FS, Brown RH, Martin DR, Gray-Edwards HL, Sena-Esteves M.   +29 more
europepmc   +1 more source

Gene expression changes in Tay-Sachs disease begin early in fetal brain development. [PDF]

J Inherit Metab Dis, 2023
Han ST, Hirt A, Nicoli ER, Kono M, Toro C, Proia RL, Tifft CJ.   +6 more
europepmc   +1 more source

Biochemical and mutational analyses of HEXA in a cohort of Egyptian patients with infantile Tay-Sachs disease. Expansion of the mutation spectrum. [PDF]

Orphanet J Rare Dis, 2023
Ibrahim DMA, Ali OSM, Nasr H, Fateen E, AbdelAleem A.   +4 more
europepmc   +1 more source

[Includes articles on euthanasia, suicide, and Tay-Sachs disease]

, 1981
Includes articles on euthanasia, suicide, and Tay-Sachs ...
Meserve, Harry C., ed.
core  

Diagnostic Tips from a Video Series and Literature Review of Patients with Late-Onset Tay-Sachs Disease. [PDF]

Tremor Other Hyperkinet Mov (N Y), 2022
Riboldi GM, Lau H.
europepmc   +1 more source

[Tay-Sachs disease].

Archivos de pediatria del Uruguay, 2003
C A, BAUZA, A, LYONNET
openaire   +3 more sources

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 Tay-Sachs Disease in an Irish Family. [PDF]

Mov Disord Clin Pract, 2021
Lefter S, O' Mahony O, Sweeney B, Ryan AM.   +3 more
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

Preconception risk assessment for thalassaemia, sickle cell disease, cystic fibrosis and Tay-Sachs disease. [PDF]

Cochrane Database Syst Rev, 2021
Hussein N, Henneman L, Kai J, Qureshi N.
europepmc   +1 more source