Results 171 to 180 of about 21,042 (237)
New Indications for Hematopoietic Stem Cell Gene Therapy in Lysosomal Storage Disorders
Linda Rossini +3 more
openalex +1 more source
The relationship between the attributes of verteporfin‐lipid nanoparticles and the exposure of immunogenic cell death markers (HSP‐70, HMGB1, and calreticulin) following 690 nm light activation in murine pancreatic cancer cells. Immunogenic cell death (ICD) is an immunostimulatory process that can be induced by light‐activated photosensitizers, but its
Nimit Shah +13 more
wiley +1 more source
Reply: Lysosomal storage disorder gene variants in multiple system atrophy [PDF]
Joshua M. Shulman
openalex +1 more source
Genetic Pigmentary Disorders: From Molecular Mechanisms to Clinical Manifestations
ABSTRACT Genetic pigmentary disorders represent a diverse group of genetic conditions characterized by alterations in melanin production and transport and melanocyte development, resulting from single‐gene pathological variants. These disorders encompass both hypopigmentary and hyperpigmentary phenotypes, affecting not only skin pigmentation but also ...
Ken Okamura, Tamio Suzuki
wiley +1 more source
Intracellular Delivery of β-Galactosidase Enzyme Using Arginase-Responsive Dextran Sulfate/Poly-l-arginine Capsule for Lysosomal Storage Disorder. [PDF]
Gupta M, Pandey H, Sivakumar S.
europepmc +1 more source
Age Deceleration and Reversal Gene Patterns in Dauer Diapause
The dauer diapause is a naturally occurring extraordinarily long‐lived alternative C. elegans larval stage that, upon dauer exit, lives a normal adult life with full reproductive capacity. Here, we define the processes underlying the slowed aging during dauer and the rejuvenation during exit.
Khrystyna Totska +4 more
wiley +1 more source
Pressure for drug development in lysosomal storage disorders – a quantitative analysis thirty years beyond the US orphan drug act [PDF]
Konstantin Mechler +3 more
openalex +1 more source
Sphingolipids are vital components of cell membranes. Metabolic disruptions of sphingolipids, including ceramide and sphingosine‐1‐phosphate, are linked to neurological disorders. This article summarizes the classification, structure, and metabolic processes of sphingolipids, and the physiological and pathological effects of sphingolipid metabolism and
Tian Li +7 more
wiley +1 more source
Fig4 deficiency: a newly emerged lysosomal storage disorder? [PDF]
Martyn C, Li J.
europepmc +1 more source

