Results 41 to 50 of about 172,662 (343)
Gene therapy for the neurological manifestations in lysosomal storage disorders
Journal of Lipid Research, 2014 Over the past several years, considerable progress has been made in the development of gene therapy as a therapeutic strategy for a variety of inherited metabolic diseases, including neuropathic lysosomal storage disorders (LSDs).
Seng H. Cheng
doaj +1 more source
eLife, 2016 Viruses manipulate host factors to enhance their replication and evade cellular restriction. We used multiplex tandem mass tag (TMT)-based whole cell proteomics to perform a comprehensive time course analysis of >6500 viral and cellular proteins ...
Edward JD Greenwood +6 more
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Intramuscular Delivery of Gene Therapy for Targeting the Nervous System
Frontiers in Molecular Neuroscience, 2020 Virus-mediated gene therapy has the potential to deliver exogenous genetic material into specific cell types to promote survival and counteract disease.
Andrew P. Tosolini +2 more
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Crosstalk between the ribosome quality control‐associated E3 ubiquitin ligases LTN1 and RNF10
FEBS Letters, EarlyView.Loss of the E3 ligase LTN1, the ubiquitin‐like modifier UFM1, or the deubiquitinating enzyme UFSP2 disrupts endoplasmic reticulum–ribosome quality control (ER‐RQC), a pathway that removes stalled ribosomes and faulty proteins. This disruption may trigger a compensatory response to ER‐RQC defects, including increased expression of the E3 ligase RNF10 ...
Yuxi Huang +8 more
wiley +1 more source
Function‐driven design of a surrogate interleukin‐2 receptor ligand
FEBS Letters, EarlyView.Interleukin (IL)‐2 signaling can be achieved and precisely fine‐tuned through the affinity, distance, and orientation of the heterodimeric receptors with their ligands. We designed a biased IL‐2 surrogate ligand that selectively promotes effector T and natural killer cell activation and differentiation. Interleukin (IL) receptors play a pivotal role in
Ziwei Tang +9 more
wiley +1 more source
Molecular Studies of HTLV-1 Replication: An Update
Viruses, 2016 Human T-cell leukemia virus type 1 (HTLV-1) was the first human retrovirus discovered. Studies on HTLV-1 have been instrumental for our understanding of the molecular pathology of virus-induced cancers.
Jessica L. Martin +4 more
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Multiple ETS family transcription factors bind mutant p53 via distinct interaction regions
FEBS Letters, EarlyView.Mutant p53 gain‐of‐function is thought to be mediated by interaction with other transcription factors. We identify multiple ETS transcription factors that can bind mutant p53 and found that this interaction can be promoted by a PXXPP motif. ETS proteins that strongly bound mutant p53 were upregulated in ovarian cancer compared to ETS proteins that ...
Stephanie A. Metcalf +6 more
wiley +1 more source
A Versatile Tool for Stable Inhibition of microRNA Activity
Biology, 2013 MicroRNAs (miRNAs) are a class of small RNAs (18–22 nt) that post transcriptionally regulate gene expression by binding to complementary sequences on target mRNAs, resulting in translational repression or target degradation and gene silencing.
Ileana Zucchi +9 more
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Turkish Journal of Hematology, 2023 Objective: Hemophilia A is an X-linked recessive bleeding disorder caused by a deficiency of plasma coagulation factor VIII (FVIII), and it accounts for about 80%-85% of all cases of hemophilia.
Cansu Hemşinlioğlu +14 more
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Lentivirus Production, Lentivirus Transduction, and Sorting Protocol v1
, 2023 Lentivirus Production, Lentivirus Transduction, and Sorting Protocol for making the transduced iPSCs and SH-SY5Ys
openaire +1 more source