Search Results - "Moreno, Pedro M. D"
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Nuclease resistant oligonucleotides with cell penetrating properties
Published in Chemical communications (Cambridge, England) (01-01-2015)“…2'-O-AECM modified oligonucleotides provide an unusual combination of remarkable properties. This includes the combination of high resistance towards enzymatic…”
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Therapeutic antisense oligonucleotides against cancer: hurdling to the clinic
Published in Frontiers in chemistry (01-01-2014)“…Under clinical development since the early 90's and with two successfully approved drugs (Fomivirsen and Mipomersen), oligonucleotide-based therapeutics has…”
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PepFect 14, a novel cell-penetrating peptide for oligonucleotide delivery in solution and as solid formulation
Published in Nucleic acids research (01-07-2011)“…Numerous human genetic diseases are caused by mutations that give rise to aberrant alternative splicing. Recently, several of these debilitating disorders have…”
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Nanotechnology approaches for gene transfer
Published in Genetica (01-09-2009)“…In both basic research as well as experimental gene therapy the need to transfer genetic material into a cell is of vital importance. The cellular compartment,…”
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Delivery of Splice Switching Oligonucleotides by Amphiphilic Chitosan-Based Nanoparticles
Published in Molecular pharmaceutics (01-02-2016)“…Splice switching oligonucleotides (SSOs) are a class of single-stranded antisense oligonucleotides (ssONs) being used as gene therapeutics and demonstrating…”
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Next-generation bis-locked nucleic acids with stacking linker and 2'-glycylamino-LNA show enhanced DNA invasion into supercoiled duplexes
Published in Nucleic acids research (18-03-2016)“…Targeting and invading double-stranded DNA with synthetic oligonucleotides under physiological conditions remain a challenge. Bis-locked nucleic acids…”
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A Peptide-based Vector for Efficient Gene Transfer In Vitro and In Vivo
Published in Molecular therapy (01-08-2011)“…Finding suitable nonviral delivery vehicles for nucleic acid–based therapeutics is a landmark goal in gene therapy. Cell-penetrating peptides (CPPs) are one…”
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Temperature-Assisted Cyclic Hybridization (TACH): An Improved Method for Supercoiled DNA Hybridization
Published in Molecular biotechnology (01-06-2010)“…Accurate hybridization is dependent on the ratio between sequence-specific and unspecific binding. Dissociation of unspecifically bound, while maintaining…”
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Functionalized chitosan derivatives as nonviral vectors: physicochemical properties of acylated N,N,N-trimethyl chitosan/oligonucleotide nanopolyplexes
Published in Soft matter (01-01-2015)“…Cationic polymers have recently attracted attention due to their proven potential for nonviral gene delivery. In this study, we report novel biocompatible…”
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A high-throughput bioimaging study to assess the impact of chitosan-based nanoparticle degradation on DNA delivery performance
Published in Acta biomaterialia (01-12-2016)“…[Display omitted] By using imaging flow cytometry as a powerful statistical high-throughput technique we investigated the impact of degradation on the…”
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11
Antisense Oligonucleotides Modulating Activation of a Nonsense-Mediated RNA Decay Switch Exon in the ATM Gene
Published in Nucleic acid therapeutics (01-12-2016)“…ATM (ataxia-telangiectasia, mutated) is an important cancer susceptibility gene that encodes a key apical kinase in the DNA damage response pathway. ATM…”
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Hydrogel-Assisted Antisense LNA Gapmer Delivery for In Situ Gene Silencing in Spinal Cord Injury
Published in Molecular therapy. Nucleic acids (01-06-2018)“…After spinal cord injury (SCI), nerve regeneration is severely hampered due to the establishment of a highly inhibitory microenvironment at the injury site,…”
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Optimizing anti-gene oligonucleotide 'Zorro-LNA' for improved strand invasion into duplex DNA
Published in Nucleic acids research (01-02-2011)“…Zorro-LNA (Zorro) is a newly developed, oligonucleotide (ON)-based, Z-shaped construct with the potential of specific binding to each strand of duplex DNA. The…”
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14
synthetic snRNA m₃G-CAP enhances nuclear delivery of exogenous proteins and nucleic acids
Published in Nucleic acids research (01-04-2009)“…Accessing the nucleus through the surrounding membrane poses one of the major obstacles for therapeutic molecules large enough to be excluded due to nuclear…”
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Enhancing Neuronal Cell Uptake of Therapeutic Nucleic Acids with Tetrahedral DNA Nanostructures
Published in Small (Weinheim an der Bergstrasse, Germany) (01-07-2024)“…The successful translation of therapeutic nucleic acids (NAs) for the treatment of neurological disorders depends on their safe and efficient delivery to…”
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Engineering a Novel Self-Assembled Multi-siRNA Nanocaged Architecture with Controlled Enzyme-Mediated siRNA Release
Published in ACS applied materials & interfaces (28-12-2022)“…The RNA interference (RNAi) chemical and structural design space has evolved since its original definitions. Although this has led to the development of RNAi…”
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Repeatable, Inducible Micro-RNA-Based Technology Tightly Controls Liver Transgene Expression
Published in Molecular therapy. Nucleic acids (01-07-2014)“…Inducible systems for gene expression emerge as a new class of artificial vectors offering temporal and spatial exogenous control of gene expression. However,…”
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Design of a peptide-based vector, PepFect6, for efficient delivery of siRNA in cell culture and systemically in vivo
Published in Nucleic acids research (01-05-2011)“…While small interfering RNAs (siRNAs) have been rapidly appreciated to silence genes, efficient and non-toxic vectors for primary cells and for systemic in…”
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Design and application of bispecific splice-switching oligonucleotides
Published in Nucleic acid therapeutics (01-02-2014)“…Targeting of pre-mRNA by short splice-switching oligonucleotides (SSOs) is increasingly being used as a therapeutic modality, one rationale being to disrupt…”
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Delivery of Antisense Oligonucleotides Mediated by a Hydrogel System: In Vitro and In Vivo Application in the Context of Spinal Cord Injury
Published in Methods in molecular biology (Clifton, N.J.) (2019)“…Biomaterials-based hydrogels are attractive drug-eluting vehicles in the context of RNA therapeutics, such as those utilizing antisense oligonucleotide or RNA…”
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