Results 131 to 140 of about 12,643 (191)

Juvenile Tay Sachs Disease Due to Compound Heterozygous Mutation in Hex-A Gene, with Early Sign of Bilateral Tremors. [PDF]

Ann Indian Acad Neurol, 2022
Sheth J, Mohapatra I, Patra G, Bhavsar R, Patel C, Shah S, Nair A.   +6 more
europepmc   +1 more source

Intrathecal delivery of AAVrh10-mHexa combined with anti-inflammatory treatment reduces neuropathological markers and extends the lifespan of mice with early-onset Tay-Sachs disease. [PDF]

Metab Brain Dis
Can M, Ausseil J, Seyrantepe V.
europepmc   +1 more source

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

Yeast-Produced Human Recombinant Lysosomal β-Hexosaminidase Efficiently Rescues GM2 Ganglioside Accumulation in Tay-Sachs Disease. [PDF]

J Pers Med
Inci OK, Leal AF, Ates N, Súarez DA, Espejo-Mojica AJ, Alméciga-Diaz CJ, Seyrantepe V.   +6 more
europepmc   +1 more source

[Tay-Sachs disease].

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

Toward a Framework of Mutualism: The Jewish Community in Genetics Research [PDF]

, 1998
Rothenberg, Karen H., Rutkin, Amy B.
core   +1 more source

Combined treatment of Ketogenic diet and propagermanium reduces neuroinflammation in Tay-Sachs disease mouse model. [PDF]

Metab Brain Dis
Inci OK, Seyrantepe V.
europepmc   +1 more source

Serum Cytokine Profile, Beta-Hexosaminidase A Enzymatic Activity and GM₂ Ganglioside Levels in the Plasma of a Tay-Sachs Disease Patient after Cord Blood Cell Transplantation and Curcumin Administration: A Case Report. [PDF]

Life (Basel), 2021
Shaimardanova AA, Chulpanova DS, Solovyeva VV, Garanina EE, Salafutdinov II, Laikov AV, Kursenko VV, Chakrabarti L, Zakharova EY, Bukina TM, Baydakova GV, Rizvanov AA.   +11 more
europepmc   +1 more source

Evaluation of the PP6D5 Polymer as a Novel Non-Viral Vector in the Development of a CRISPR/nCas9-Based Gene Therapy for Tay-Sachs Disease. [PDF]

Pharmaceutics
Guerrero-Vargas JM, Suarez-Garcia DA, Leal AF, Diaz-Ariza IL, Pérez-Pérez LD, Espejo-Mojica AJ, Alméciga-Díaz CJ.   +6 more
europepmc   +1 more source

Precise template-free correction restores gene function in Tay-Sachs disease while reframing is ineffective. [PDF]

Mol Ther Nucleic Acids
Hung JE, Brewer RA, Elbakr L, Mollica A, Forguson G, Chan WS, Ivakine EA.   +6 more
europepmc   +1 more source