Arginine-rich cell-penetrating peptide dramatically enhances AMO-mediated ATM aberrant splicing correction and enables delivery to brain and cerebellum. (Q35119681)

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English	Arginine-rich cell-penetrating peptide dramatically enhances AMO-mediated ATM aberrant splicing correction and enables delivery to brain and cerebellum.	scientific article

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1 February 2020

Arginine-rich cell-penetrating peptide dramatically enhances AMO-mediated ATM aberrant splicing correction and enables delivery to brain and cerebellum (English)

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1 February 2020

Patrick L Iversen

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1 February 2020

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Liutao Du

1

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1 February 2020

Refik Kayali

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1 February 2020

Carmen Bertoni

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1 February 2020

Francesca Fike

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1 February 2020

Hailiang Hu

5

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1 February 2020

Richard A Gatti

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1 February 2020

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16 May 2011

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1 February 2020

Human Molecular Genetics

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1 February 2020

20

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1 February 2020

16

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1 February 2020

3151-3160

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1 February 2020

Potential therapeutic applications of antisense morpholino oligonucleotides in modulation of splicing in primary immunodeficiency diseases

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9 July 2018

Nonaminoglycoside compounds induce readthrough of nonsense mutations

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9 July 2018

Delivery of nucleic acids with a stearylated (RxR)4 peptide using a non-covalent co-incubation strategy

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Cellular uptake of neutral phosphorodiamidate morpholino oligomers.

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9 July 2018

Progress toward therapy with antisense-mediated splicing modulation

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9 July 2018

Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations

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9 July 2018

Ataxia-telangiectasia: from a rare disorder to a paradigm for cell signalling and cancer

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9 July 2018

Effective rescue of dystrophin improves cardiac function in dystrophin-deficient mice by a modified morpholino oligomer

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9 July 2018

Atm and cellular response to DNA damage

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9 July 2018

Sustained dystrophin expression induced by peptide-conjugated morpholino oligomers in the muscles of mdx mice.

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9 July 2018

Arginine-rich cell penetrating peptides: design, structure-activity, and applications to alter pre-mRNA splicing by steric-block oligonucleotides.

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9 July 2018

Cell-penetrating peptides for drug delivery across membrane barriers

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9 July 2018

Activation and regulation of ATM kinase activity in response to DNA double-strand breaks

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9 July 2018

Cell penetrating peptide conjugates of steric block oligonucleotides

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9 July 2018

Role for KAP1 serine 824 phosphorylation and sumoylation/desumoylation switch in regulating KAP1-mediated transcriptional repression

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9 July 2018

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9 July 2018

Current and potential therapeutic strategies for the treatment of ataxia-telangiectasia.

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9 July 2018

Pharmacokinetics, biodistribution, stability and toxicity of a cell-penetrating peptide-morpholino oligomer conjugate.

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9 July 2018

Achieving targeted and quantifiable alteration of mRNA splicing with Morpholino oligos

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9 July 2018

Correction of prototypic ATM splicing mutations and aberrant ATM function with antisense morpholino oligonucleotides

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9 July 2018

Stability of cell-penetrating peptide-morpholino oligomer conjugates in human serum and in cells.

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9 July 2018

Vectorization of morpholino oligomers by the (R-Ahx-R)4 peptide allows efficient splicing correction in the absence of endosomolytic agents.

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9 July 2018

Pharmacokinetics and biodistribution of phosphorodiamidate morpholino antisense oligomers.

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9 July 2018

Functional significance of a deep intronic mutation in the ATM gene and evidence for an alternative exon 28a.

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9 July 2018

Neutrally charged phosphorodiamidate morpholino antisense oligomers: uptake, efficacy and pharmacokinetics.

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Chances and pitfalls of cell penetrating peptides for cellular drug delivery

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Distribution of calcium-binding proteins in the cerebellum

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9 July 2018

Nonclassical splicing mutations in the coding and noncoding regions of the ATM Gene: maximum entropy estimates of splice junction strengths

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9 July 2018

Early diagnosis of ataxia-telangiectasia using radiosensitivity testing

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9 July 2018

Involvement of the cohesin protein, Smc1, in Atm-dependent and independent responses to DNA damage

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12 December 2020

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