Results 41 to 50 of about 47,774 (288)
Fast-track applications : the potential for direct delivery of proteins and nucleic acids to plant cells for the discovery of the gene function. [PDF]
, 2005 In animal systems, several methods exist for the direct delivery of nucleic acids and proteins into cells for functional analysis. Until recently, these methods have not been applied to plant systems.
Roberts, Michael R. +2 more
core +1 more source
A mechanistic pharmacokinetic model for intrathecal administration of antisense oligonucleotides
Frontiers in Physiology, 2023 Intrathecal administration is an important mode for delivering biological agents targeting central nervous system (CNS) diseases. However, current clinical practices lack a sound theorical basis for a quantitative understanding of the variables and ...
Andreas A. Linninger +4 more
doaj +1 more source
Antisense Oligonucleotide-Based Therapy for Neuromuscular Disease
Molecules, 2017 Neuromuscular disorders such as Duchenne Muscular Dystrophy and Spinal Muscular Atrophy are neurodegenerative genetic diseases characterized primarily by muscle weakness and wasting.
Valentina Sardone +4 more
doaj +1 more source
Silencing disease genes in the laboratory and the clinic
, 2011 Synthetic nucleic acids are commonly used laboratory tools for modulating gene expression and have the potential to be widely used in the clinic. Progress towards nucleic acid drugs, however, has been slow and many challenges remain to be overcome before
Jonathan K Watts +3 more
core +1 more source
Hippo pathway at the crossroads of stemness and therapeutic resistance in breast cancer
Molecular Oncology, EarlyView.Dysregulation of the Hippo pathway drives nuclear accumulation of YAP/TAZ, activating stemness‐related transcriptional programs that sustain breast cancer stemness and fuel therapeutic resistance across subtypes, underscoring Hippo signaling as a targetable vulnerability. Figure created and edited with BioRender.com.
Giulia Schiavoni +11 more
wiley +1 more source
Efficient Modulation of Exon Skipping via Antisense Circular RNAs
Research, 2023 Splice-switching antisense oligonucleotides (ASOs) and engineered U7 small nuclear ribonucleoprotein (U7 Sm OPT) are the most commonly used methods for exon skipping. However, challenges remain, such as limited organ delivery and repeated dosing for ASOs
Shuaiwei Ren +8 more
doaj +1 more source
Antisense therapeutics in oncology: current status
, 2014 There is increasing progress in translational oncology and tremendous breakthroughs have been made as evidenced by preclinical and clinical trials. Data obtained from high-throughput technologies are deepening our understanding about the molecular and ...
Muntané Relat, Jordi +2 more
core +1 more source
Antisense delivery using protamine-oligonucleotide particles [PDF]
Nucleic Acids Research, 2000 Protamine, a polycationic peptide (mol. wt 4000-4500), was evaluated as a potential penetration enhancer for phosphodiester antisense oligonucleotides (ODNs). Unique complexes in the form of nanoparticles were spontaneously formed, which we call 'proticles'.
M, Junghans, J, Kreuter, A, Zimmer
openaire +2 more sources
Functional and Structural Evidence of Neurofluid Circuit Aberrations in Huntington Disease
Annals of Clinical and Translational Neurology, EarlyView.ABSTRACT Objective Disrupted neurofluid regulation may contribute to neurodegeneration in Huntington disease (HD). Because neurofluid pathways influence waste clearance, inflammation, and the distribution of central nervous system (CNS)–delivered therapeutics, understanding their dysfunction is increasingly important as targeted treatments emerge.
Kilian Hett +8 more
wiley +1 more source
Molecular Therapy: Nucleic Acids, 2017 Antisense-mediated exon skipping is a promising approach for the treatment of Duchenne muscular dystrophy (DMD), a rare life-threatening genetic disease due to dystrophin deficiency.
Ngoc Lu-Nguyen +5 more
doaj +1 more source