Results 251 to 260 of about 102,759 (307)

The Race to Salvage Glucocerebrosidase: Understanding Small‐Molecule Therapies for GBA1‐Associated Parkinsonism

Movement Disorders, EarlyView.
Variants in GBA1, the gene encoding the lysosomal enzyme glucocerebrosidase, cause Gaucher disease and confer an increased risk for parkinsonism. Strategies using small molecules can improve the function of glucocerebrosidase in lysosomes. A clear understanding of the mechanism‐of‐action of these compounds will facilitate development of GBA1‐modulating
Mark J. Henderson, Tiffany C. Chen, Logan M. Glasstetter, Yu Chen, Juan J. Marugan, Ellen Sidransky   +5 more
wiley   +1 more source

Protocol for the generation and xenotransplantation of human induced pluripotent stem cell-derived neural progenitor cells into the mouse forebrain. [PDF]

STAR Protoc
McInvale JJ, Ayers CE, Sarmah H, Kim K, Reinhardt P, Mahajan A, Humala N, Corneo B, Sterneckert J, Canoll P, Hargus G.   +10 more
europepmc   +1 more source

Parkinson's Disease Patient‐Specific Striatum Organoids Show Hallmarks of Increased Inflammation

Movement Disorders, EarlyView.
Abstract Background Dopaminergic neurons from the substantia nigra pars compacta project their axons into the dorsal striatum, forming the nigrostriatal pathway. In Parkinson's disease (PD), dopaminergic terminals degenerate in the striatum, leading to dopamine depletion, which in turn causes alterations in the basal ganglia circuits that are essential
Kyriaki Barmpa, Claudia Saraiva, Alise Zagare, Mona Tuzza, Joseph Longworth, Elisa Zuccoli, Katrin Hufnagel, Florian Skwirblies, Ronny Schmidt, Dirk Brenner, Christoph Schröder, Jens C. Schwamborn   +11 more
wiley   +1 more source

iPSC-derived ITGA6-positive cells restore aqueous humor outflow in glaucoma eyes. [PDF]

Nat Commun
Feng P, Yu C, Zhang X, Xu B, Wu S, Xin C, Littleton S, Kang J, Wang X, Liang S, Chen S, Wang W, Wang Y, Yuan Z, Xi G, Xing X, Ge X, Chang Z, Tan J, Zhang J, Zhang B, Cao Q, Xu W, Kuehn MH, Wang N, Zhu W.   +25 more
europepmc   +1 more source

The Promise of Exosomes in Cardiac Repair: When 'Best Things Come in Small Packages'. [PDF]

J Cell Mol Med
Yazbeck A, Wehbe Z, Menassa Y, Abdelhamid A, Eid AA, Sahebkar A, Eid AH.   +6 more
europepmc   +1 more source

Gene Editing of Pluripotent Stem Cell-Derived Hepatic Cells for Liver Disease Modeling and Therapeutic Development. [PDF]

Biomol Ther (Seoul)
Lim D, Kim HR.
europepmc   +1 more source

Beyond Pathology: α‐Synuclein Homeostasis and Three Principles to Guide Research

Movement Disorders Clinical Practice, EarlyView.
Alberto J. Espay, Andrew J. Lees
wiley   +1 more source