Arginine-rich cell-penetrating peptide dramatically enhances AMO-mediated ATM aberrant splicing correction and enables delivery to brain and cerebellum.
about
Antisense mediated splicing modulation for inherited metabolic diseases: challenges for deliveryAttachment of cell-binding ligands to arginine-rich cell-penetrating peptides enables cytosolic translocation of complexed siRNA.Delivery is key: lessons learnt from developing splice-switching antisense therapiesSplice-correcting oligonucleotides restore BTK function in X-linked agammaglobulinemia modelPhosphorodiamidate morpholino oligomers suppress mutant huntingtin expression and attenuate neurotoxicity.Systemic peptide-mediated oligonucleotide therapy improves long-term survival in spinal muscular atrophy.A novel peptide delivers plasmids across blood-brain barrier into neuronal cells as a single-component transfer vector.SMRT compounds abrogate cellular phenotypes of ataxia telangiectasia in neural derivatives of patient-specific hiPSCs.A new series of small molecular weight compounds induce read through of all three types of nonsense mutations in the ATM gene.Splice-correction strategies for treatment of X-linked agammaglobulinemia.Functional characterization and targeted correction of ATM mutations identified in Japanese patients with ataxia-telangiectasia.Development and Application of an Ultrasensitive Hybridization-Based ELISA Method for the Determination of Peptide-Conjugated Phosphorodiamidate Morpholino Oligonucleotides.The delivery of therapeutic oligonucleotidesUpon the tightrope in prostate cancer: two acrobats on the same tightrope to cross the finishline.Cell-penetrating Peptide-mediated therapeutic molecule delivery into the central nervous system.Exon-skipping antisense oligonucleotides to correct missplicing in neurogenetic diseases.Peptide-based vectors: recent developments.Antisense Oligonucleotide-Based Therapy for Neuromuscular Disease.Alternative splicing: the pledge, the turn, and the prestige : The key role of alternative splicing in human biological systems.Pharmacomodulation of microRNA Expression in Neurocognitive Diseases: Obstacles and Future Opportunities.Splice-Switching Therapy for Spinal Muscular Atrophy.MicroRNAs as Molecular Targets for Cancer Therapy: On the Modulation of MicroRNA Expression.Peptide-mediated Cell and In Vivo Delivery of Antisense Oligonucleotides and siRNA.Making sense of antisense oligonucleotides: A narrative review.Nucleic Acid-Based Theranostics for Tackling Alzheimer's Disease.Comparison of neuroprotective efficacy of poly-arginine R18 and R18D (D-enantiomer) peptides following permanent middle cerebral artery occlusion in the Wistar rat and in vitro toxicity studies.Antisense oligonucleotides in neurological disorders.Synthetic molecular evolution of hybrid cell penetrating peptides.Cell-Penetrating Peptides to Enhance Delivery of Oligonucleotide-Based Therapeutics.
P2860
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P2860
Arginine-rich cell-penetrating peptide dramatically enhances AMO-mediated ATM aberrant splicing correction and enables delivery to brain and cerebellum.
description
2011 nî lūn-bûn
@nan
2011 թուականի Մայիսին հրատարակուած գիտական յօդուած
@hyw
2011 թվականի մայիսին հրատարակված գիտական հոդված
@hy
2011年の論文
@ja
2011年論文
@yue
2011年論文
@zh-hant
2011年論文
@zh-hk
2011年論文
@zh-mo
2011年論文
@zh-tw
2011年论文
@wuu
name
Arginine-rich cell-penetrating ...... ivery to brain and cerebellum.
@ast
Arginine-rich cell-penetrating ...... ivery to brain and cerebellum.
@en
type
label
Arginine-rich cell-penetrating ...... ivery to brain and cerebellum.
@ast
Arginine-rich cell-penetrating ...... ivery to brain and cerebellum.
@en
prefLabel
Arginine-rich cell-penetrating ...... ivery to brain and cerebellum.
@ast
Arginine-rich cell-penetrating ...... ivery to brain and cerebellum.
@en
P2093
P2860
P356
P1476
Arginine-rich cell-penetrating ...... livery to brain and cerebellum
@en
P2093
Carmen Bertoni
Francesca Fike
Hailiang Hu
Refik Kayali
Richard A Gatti
P2860
P304
P356
10.1093/HMG/DDR217
P577
2011-05-16T00:00:00Z