Results 131 to 140 of about 2,137 (206)

Infantile gangliosidoses: Mapping a timeline of clinical changes.

Molecular Genetics and Metabolism, 2017
Jeanine R. Jarnes Utz, Sarah Kim, K. King, Richard S. Ziegler, Lynn Schema, Evelyn Redtree, C. Whitley   +6 more
semanticscholar   +1 more source

Linking glycosphingolipid metabolism to disease-related changes in the plasma membrane proteome. [PDF]

Biochem Soc Trans
Monkhouse H, Deane JE.
europepmc   +1 more source

New multiplex LC-MS/MS method for lipid biomarker analysis of inherited neurodegenerative metabolic diseases. [PDF]

J Lipid Res
Sidorina A, Catesini G, Deodato F, Boenzi S, Martinelli D, Rizzo C, Dionisi-Vici C.   +6 more
europepmc   +1 more source

Advances in AAV-mediated gene replacement therapy for pediatric monogenic neurological disorders. [PDF]

Mol Ther Methods Clin Dev
Zhou L, Wang Y, Xu Y, Zhang Y, Zhu C.
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

Advancing CNS Therapeutics: Enhancing Neurological Disorders with Nanoparticle-Based Gene and Enzyme Replacement Therapies. [PDF]

Int J Nanomedicine
Liu S, Li H, Xi S, Zhang Y, Sun T.
europepmc   +1 more source

In Vitro Studies Of Liposome Carriers In Enzyme Replacement Therapy Of Feline Gangliosidoses. [PDF]

, 1978
Reynolds, Gary Dean
core   +1 more source

Animal models of GM2 gangliosidosis: utility and limitations [PDF]

core   +2 more sources

Morphological And Physiological Studies Of Neuronal Dysfunction In The Feline Gangliosidoses. [PDF]

, 1981
Walkley, Steven Upshaw
core   +1 more source

Conformational Dynamics and Membrane Insertion Mechanism of B4GALNT1 in Ganglioside Synthesis [PDF]

Nat Commun
Deane J, Welland J, Barrow H, Stansfeld P.   +3 more
europepmc   +2 more sources