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。Potent and Selective Inhibition of A-to-I RNA Editing with 2′-O-Methyl/Locked Nucleic Acid-Containing Antisense Oligoribonucleotides
Rena A. Mizrahi et al.
ACS CHEMICAL BIOLOGY (2013)
Agammaglobulinemia: causative mutations and their implications for novel therapies
Anna Berglof et al.
EXPERT REVIEW OF CLINICAL IMMUNOLOGY (2013)
Clinical Trials Using Antisense Oligonucleotides in Duchenne Muscular Dystrophy
Taeyoung Koo et al.
HUMAN GENE THERAPY (2013)
Dual Phosphorylation of Btk by Akt/Protein Kinase B Provides Docking for 14-3-3ζ, Regulates Shuttling, and Attenuates both Tonic and Induced Signaling in B Cells
Dara K. Mohammad et al.
MOLECULAR AND CELLULAR BIOLOGY (2013)
Improved Performance of Anti-miRNA Oligonucleotides Using a Novel Non-Nucleotide Modifier
Kim A. Lennox et al.
MOLECULAR THERAPY-NUCLEIC ACIDS (2013)
Splice Modulating Therapies for Human Disease
Pietro Spitali et al.
CELL (2012)
Use of Cell-Penetrating-Peptides in Oligonucleotide Splice Switching Therapy
Samir A. El Andaloussi et al.
CURRENT GENE THERAPY (2012)
PCSK9 LNA Antisense Oligonucleotides Induce Sustained Reduction of LDL Cholesterol in Nonhuman Primates
Marie W. Lindholm et al.
MOLECULAR THERAPY (2012)
Lymphoid priming in human bone marrow begins before expression of CD10 with upregulation of L-selectin
Lisa A. Kohn et al.
NATURE IMMUNOLOGY (2012)
Locked vs. unlocked nucleic acids (LNA vs. UNA): contrasting structures work towards common therapeutic goals
Meghan A. Campbell et al.
CHEMICAL SOCIETY REVIEWS (2011)
miR-31 modulates dystrophin expression: new implications for Duchenne muscular dystrophy therapy
Davide Cacchiarelli et al.
EMBO REPORTS (2011)
Arginine-rich cell-penetrating peptide dramatically enhances AMO-mediated ATM aberrant splicing correction and enables delivery to brain and cerebellum
Liutao Du et al.
HUMAN MOLECULAR GENETICS (2011)
Compensatory signals associated with the activation of human GC 5' splice sites
Jana Kralovicova et al.
NUCLEIC ACIDS RESEARCH (2011)
Amino acids attached to 2 '-amino-LNA: synthesis and excellent duplex stability
Marie W. Johannsen et al.
ORGANIC & BIOMOLECULAR CHEMISTRY (2011)
Long-term improvement in mdx cardiomyopathy after therapy with peptide-conjugated morpholino oligomers
Natee Jearawiriyapaisarn et al.
CARDIOVASCULAR RESEARCH (2010)
Locked Nucleic Acids: Promising Nucleic Acid Analogs for Therapeutic Applications
Rakesh N. Veedu et al.
CHEMISTRY & BIODIVERSITY (2010)
The life situations of patients with primary antibody deficiency untreated or treated with subcutaneous gammaglobulin infusions
A. GARDULF et al.
CLINICAL AND EXPERIMENTAL IMMUNOLOGY (2010)
MicroRNAs Prevent the Generation of Autoreactive Antibodies
Laura Belver et al.
IMMUNITY (2010)
Efficient gene silencing by delivery of locked nucleic acid antisense oligonucleotides, unassisted by transfection reagents
C. A. Stein et al.
NUCLEIC ACIDS RESEARCH (2010)
Short locked nucleic acid antisense oligonucleotides potently reduce apolipoprotein B mRNA and serum cholesterol in mice and non-human primates
Ellen Marie Straarup et al.
NUCLEIC ACIDS RESEARCH (2010)
Primary B Cell Immunodeficiencies: Comparisons and Contrasts
Mary Ellen Conley et al.
ANNUAL REVIEW OF IMMUNOLOGY (2009)
Bruton's tyrosine kinase (Btk): function, regulation, and transformation with special emphasis on the PH domain
Abdalla J. Mohamed et al.
IMMUNOLOGICAL REVIEWS (2009)
Design of Phosphorodiamidate Morpholino Oligomers ( PMOs) for the Induction of Exon Skipping of the Human DMD Gene
Linda J. Popplewell et al.
MOLECULAR THERAPY (2009)
RNA repair restores hemoglobin expression in IVS2-654 thalassemic mice
Saovaros Svasti et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2009)
Splice-switching efficiency and specificity for oligonucleotides with locked nucleic acid monomers
Peter Guterstam et al.
BIOCHEMICAL JOURNAL (2008)
Cell-penetrating peptide-conjugated antisense oligonucleotides restore systemic muscle and cardiac dystrophin expression and function
HaiFang Yin et al.
HUMAN MOLECULAR GENETICS (2008)
An endogenous TNF-α antagonist induced by splice-switching oligonucleotides reduces inflammation in hepatitis and arthritis mouse models
Maria A. Graziewicz et al.
MOLECULAR THERAPY (2008)
Sustained dystrophin expression induced by peptide-conjugated morpholino oligomers in the muscles of mdx mice
Natee Jearawiriyapaisarn et al.
MOLECULAR THERAPY (2008)
Adults with X-linked agammaglobulinemia - Impact of disease on daily lives, quality of life, educational and socioeconomic status, knowledge of inheritance, and reproductive attitudes
Jerry A. Winkelstein et al.
MEDICINE (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)
Splicing regulation: From a parts list of regulatory elements to an integrated splicing code
Zefeng Wang et al.
RNA (2008)
miR-122 targeting with LNA/2′-O-methyl oligonucleotide mixmers, peptide nucleic acids (PNA), and PNA-peptide conjugates
Martin M. Fabani et al.
RNA (2008)
Perspectives on chemistry and therapeutic applications of Locked Nucleic Acid (LNA)
Harleen Kaur et al.
CHEMICAL REVIEWS (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)
Antisense inhibition of proprotein convertase subtilisin/kexin type 9 reduces serum LDL in hyperlipidemic mice
Mark J. Graham et al.
JOURNAL OF LIPID RESEARCH (2007)
Enhancement of SMN2 Exon 7 inclusion by antisense oligonucleotides targeting the exon
Yimin Hua et al.
PLOS BIOLOGY (2007)
BDTKbase:: The mutation database for X-linked agammaglobullinemia
Jouni Valiaho et al.
HUMAN MUTATION (2006)
Efficient and persistent splice switching by systemically delivered LNA oligonucleotides in mice
Jennifer Roberts et al.
MOLECULAR THERAPY (2006)
Are splicing mutations the most frequent cause of hereditary disease?
N López-Bigas et al.
FEBS LETTERS (2005)
Pleiotropic consequences of Bruton tyrosine kinase deficiency in myeloid lineages lead to poor inflammatory responses
A Mangla et al.
BLOOD (2004)
Computational definition of sequence motifs governing constitutive exon splicing
XHF Zhang et al.
GENES & DEVELOPMENT (2004)
ESEfinder: a web resource to identify exonic splicing enhancers
L Cartegni et al.
NUCLEIC ACIDS RESEARCH (2003)
Clinical, immunological, and molecular analysis in a large cohort of patients with X-linked agammaglobulinemia: An Italian multicenter study
A Plebani et al.
CLINICAL IMMUNOLOGY (2002)
Predictive identification of exonic splicing enhancers in human genes
WG Fairbrother et al.
SCIENCE (2002)
Implications of high-affinity hybridization by locked nucleic acid oligomers for inhibition of human telomerase
AN Elayadi et al.
BIOCHEMISTRY (2002)
Composition of precursor B-cell compartment in bone marrow from patients with X-linked agammaglobulinemia compared with healthy children
JG Noordzij et al.
PEDIATRIC RESEARCH (2002)
Inhibition of HIV-1 Tat-dependent trans activation by steric block chimeric 2′-O-methyl/LNA oligoribonucleotides
A Arzumanov et al.
BIOCHEMISTRY (2001)
The Tec family of cytoplasmic tyrosine kinases: mammalian Btk, Bmx, Itk, Tec, Txk and homologs in other species
CIE Smith et al.
BIOESSAYS (2001)
Regulation of B lymphocyte development and activation by Bruton's tyrosine kinase
WN Khan
IMMUNOLOGIC RESEARCH (2001)
Severe B cell deficiency in mice lacking the Tec kinase family members Tec and Btk
W Ellmeier et al.
JOURNAL OF EXPERIMENTAL MEDICINE (2000)
Potent and nontoxic antisense oligonucleotides containing locked nucleic acids
C Wahlestedt et al.
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2000)