Results 101 to 110 of about 2,093,614 (359)
Intron retention is regulated by altered MeCP2-mediated splicing factor recruitment
Nature Communications, 2017 Intron retention is a conserved mechanism that controls gene expression but its regulation is poorly understood. Here, the authors provide evidence that DNA methylation regulates intron retention and find reduced MeCP2 occupancy and splicing factor ...
Justin J. -L. Wong +11 more
doaj +1 more source
Antiangiogenic gene therapy [PDF]
Proceedings of the National Academy of Sciences, 1998 The authors (1) laid the foundation for this experiment by previously showing that a single injection of soluble Tie2 receptor protein into a rat cutaneous window reduced tumor vascular length by 40% …
openaire +2 more sources
Sequence determinants of RNA G‐quadruplex unfolding by Arg‐rich regions
FEBS Letters, EarlyView.We show that Arg‐rich peptides selectively unfold RNA G‐quadruplexes, but not RNA stem‐loops or DNA/RNA duplexes. This length‐dependent activity is inhibited by acidic residues and is conserved among SR and SR‐related proteins (SRSF1, SRSF3, SRSF9, U1‐70K, and U2AF1).
Naiduwadura Ivon Upekala De Silva +10 more
wiley +1 more source
FEBS Letters, EarlyView.The Ile181Asn variant of human UDP‐xylose synthase (hUXS1), associated with a short‐stature genetic syndrome, has previously been reported as inactive. Our findings demonstrate that Ile181Asn‐hUXS1 retains catalytic activity similar to the wild‐type but exhibits reduced stability, a looser oligomeric state, and an increased tendency to precipitate ...
Tuo Li +2 more
wiley +1 more source
Adeno‐associated virus serotype 1‐based gene therapy for FTD caused by GRN mutations
Annals of Clinical and Translational Neurology, 2020 Objective Dominant loss‐of‐function mutations in the gene encoding the lysosomal protein, progranulin, cause 5‐10% of frontotemporal dementia cases. As progranulin undergoes secretion and endocytosis, a small number of progranulin‐expressing cells can ...
Christian Hinderer +6 more
doaj +1 more source
Gene therapy for cystic fibrosis: new tools for precision medicine [PDF]
, 2021 Jin A Lee +6 more
openalex +1 more source
, 2018 Ewing’s sarcoma is the second most common malignant tumor of the bone occurring in children and adolescents. Typically, patients present between the ages of 10 and 20, with the disease having a slight predilection for males.1 Tumors often arise in the ...
Fenderson, PhD, Bruce A. +1 more
core +1 more source
Improved Network Performance via Antagonism: From Synthetic Rescues to Multi-drug Combinations [PDF]
, 2010 Recent research shows that a faulty or sub-optimally operating metabolic network can often be rescued by the targeted removal of enzyme-coding genes--the exact opposite of what traditional gene therapy would suggest.
Adilson E. Motter +58 more
core +3 more sources
PICALM::MLLT10 translocated leukemia
FEBS Letters, EarlyView.This comprehensive review of PICALM::MLLT10 translocated acute leukemia provides an in‐depth review of the structure and function of CALM, AF10, and the fusion oncoprotein (1). The multifaceted molecular mechanisms of oncogenesis, including nucleocytoplasmic shuttling (2), epigenetic modifications (3), and disruption of endocytosis (4), are then ...
John M. Cullen +7 more
wiley +1 more source
AAV gene replacement therapy for treating MPS IIIC: Facilitating bystander effects via EV‐mRNA cargo
Journal of Extracellular VesiclesMPS IIIC is a lysosomal storage disease caused by mutations in heparan‐α‐glucosaminide N‐acetyltransferase (HGSNAT), for which no treatment is available.
Tierra A. Bobo +6 more
doaj +1 more source