相关参考文献
注意：仅列出部分参考文献，下载原文获取全部文献信息。Potential therapeutic applications of antisense morpholino oligonucleotides in modulation of splicing in primary immunodeficiency diseases
Liutao Du et al.
JOURNAL OF IMMUNOLOGICAL METHODS (2011)
Naturally- and experimentally-designed restorations of the Parkin gene deficit in autosomal recessive juvenile parkinsonism
Hirohide Asai et al.
BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS (2010)
Delivery of nucleic acids with a stearylated (RxR)4 peptide using a non-covalent co-incubation strategy
Taavi Lehto et al.
JOURNAL OF CONTROLLED RELEASE (2010)
Cellular Uptake of Neutral Phosphorodiamidate Morpholino Oligomers
Patrick L. Iversen et al.
CURRENT PHARMACEUTICAL BIOTECHNOLOGY (2009)
Theoretic Applicability of Antisense-Mediated Exon Skipping for Duchenne Muscular Dystrophy Mutations
Annemieke Aartsma-Rus et al.
HUMAN MUTATION (2009)
Nonaminoglycoside compounds induce readthrough of nonsense mutations
Liutao Du et al.
JOURNAL OF EXPERIMENTAL MEDICINE (2009)
Cell penetrating peptide conjugates of steric block oligonucleotides
Bernard Lebleu et al.
ADVANCED DRUG DELIVERY REVIEWS (2008)
Cell-penetrating peptide-conjugated antisense oligonucleotides restore systemic muscle and cardiac dystrophin expression and function
HaiFang Yin et al.
HUMAN MOLECULAR GENETICS (2008)
Arginine-rich cell penetrating peptides: Design, structure-activity, and applications to alter pre-mRNA splicing by steric-block oligonucleotides
R. Abes et al.
JOURNAL OF PEPTIDE SCIENCE (2008)
Sustained dystrophin expression induced by peptide-conjugated morpholino oligomers in the muscles of mdx mice
Natee Jearawiriyapaisarn et al.
MOLECULAR THERAPY (2008)
Ataxia-telangiectasia: from a rare disorder to a paradigm for cell signalling and cancer
Martin F. Lavin
NATURE REVIEWS MOLECULAR CELL BIOLOGY (2008)
Effective rescue of dystrophin improves cardiac function in dystrophin-deficient mice by a modified morpholino oligomer
Bo Wu et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2008)
Cell-penetrating peptides for drug delivery across membrane barriers
Camilla Foged et al.
EXPERT OPINION ON DRUG DELIVERY (2008)
Role for KAP1 serine 824 phosphorylation and sumoylation/desumoylation switch in regulating KAP1-mediated transcriptional repression
Xu Li et al.
JOURNAL OF BIOLOGICAL CHEMISTRY (2007)
Activation and regulation of ATM kinase activity in response to DNA double-strand breaks
J-H Lee et al.
ONCOGENE (2007)
A novel cell-penetrating peptide, M918, for efficient delivery of proteins and peptide nucleic acids
Samir El-Andaloussi et al.
MOLECULAR THERAPY (2007)
Morpholino oligomer -: Mediated Exon skipping averts the onset of dystrophic pathology in the mdx mouse
Sue Fletcher et al.
MOLECULAR THERAPY (2007)
Arginine-rich cell-penetrating peptides facilitate delivery of antisense oligomers into murine leukocytes and alter pre-mRNA splicing
N. B. Marshall et al.
JOURNAL OF IMMUNOLOGICAL METHODS (2007)
Pharmacokinetics, biodistribution, stability and toxicity of a cell-penetrating peptide-morpholino oligomer conjugate
Adams Amantana et al.
BIOCONJUGATE CHEMISTRY (2007)
Achieving targeted and quantifiable alteration of mRNA splicing with Morpholino oligos
Paul A. Morcos
BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS (2007)
Correction of prototypic ATM splicing mutations and aberrant ATM function with antisense morpholino oligonucleotides
Liutao Du et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2007)
Stability of cell-penetrating peptide-morpholino oligomer conjugates in human serum and in cells
Derek S. Youngblood et al.
BIOCONJUGATE CHEMISTRY (2007)
Current and potential therapeutic strategies for the treatment of ataxia-telangiectasia
Martin F. Lavin et al.
BRITISH MEDICAL BULLETIN (2007)
Modulating the expression of disease genes with RNA-Based therapy
Matthew Wood et al.
PLOS GENETICS (2007)
Vectorization of morpholino oligomers by the (R-Ahx-R)4 peptide allows efficient splicing correction in the absence of endosomolytic agents
Said Abes et al.
JOURNAL OF CONTROLLED RELEASE (2006)
Pharmacokinetics and biodistribution of phosphorodiamidate morpholino antisense oligomers
A Amantana et al.
CURRENT OPINION IN PHARMACOLOGY (2005)
Functional significance of deep intronic mutation in the ATM gene and evidence for an alternative Exon 28a
G Coutinho et al.
HUMAN MUTATION (2005)
Alternative splicing in disease and therapy
MA Garcia-Blanco et al.
NATURE BIOTECHNOLOGY (2004)
Neutrally charged phosphorodiamidate morpholino antisense oligomers: Uptake, efficacy and pharmacokinetics
V Arora et al.
CURRENT PHARMACEUTICAL BIOTECHNOLOGY (2004)
ATM protein purified from vaccinia virus expression system: DNA binding requirements for kinase activation
HH Chun et al.
BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS (2004)
Chances and pitfalls of cell penetrating peptides for cellular drug delivery
R Tréhin et al.
EUROPEAN JOURNAL OF PHARMACEUTICS AND BIOPHARMACEUTICS (2004)
Immunoassay to measure ataxia-telangiectasia mutated protein in cellular lysates
AW Butch et al.
CLINICAL CHEMISTRY (2004)
Nonclassical splicing mutations in the coding and noncoding regions of the ATM gene: Maximum entropy estimates of splice junction strengths
L Eng et al.
HUMAN MUTATION (2004)
Independent mutational events are rare in the ATM gene: Haplotype prescreening enhances mutation detection rate
M Mitui et al.
HUMAN MUTATION (2003)
Distribution of calcium-binding proteins in the cerebellum
E Bastianelli
CEREBELLUM (2003)
Early diagnosis of ataxia-telangiectasia using radiosensitivity testing
X Sun et al.
JOURNAL OF PEDIATRICS (2002)
Involvement of the cohesin protein, Smc1, in Atm-dependent and independent responses to DNA damage
ST Kim et al.
GENES & DEVELOPMENT (2002)
The pathogenesis of ataxia-telangiectasia - Learning from a Rosetta Stone
RA Gatti et al.
CLINICAL REVIEWS IN ALLERGY & IMMUNOLOGY (2001)
分享论文
