Generation of Choline for Acetylcholine Synthesis by Phospholipase D Isoforms [PDF]
, 2001 DEDICATION: This article is dedicated to the memory of Sue Kim Hanson, a graduate student in the department of Pathology and Laboratory Medicine at Boston University School of Medicine, who perished in the terrorist attacks of September 11, 2001 ...
Blusztajn, Jan Krzysztof +2 more
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Correction to the pathogenic alternative splicing, caused by the common GNB3 c.825C>T allele, using a novel, antisense morpholino [PDF]
, 2016 The very common GNB3 c.825C>T polymorphism (rs5443), is present in approximately half of all human chromosomes. Significantly the presence of the GNB3 825T allele has been strongly associated, with predisposition to essential hypertension ...
Lester, Douglas H. +2 more
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Targeting STAT3 in Cancer with Nucleotide Therapeutics. [PDF]
, 2019 Signal transducer and activator of transcription 3 (STAT3) plays a critical role in promoting the proliferation and survival of tumor cells. As a ubiquitously-expressed transcription factor, STAT3 has commonly been considered an "undruggable" target for ...
Grandis, Jennifer R +3 more
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Dual targeting of MIR-208 and MIR-499 in the treatment of cardiac disorders [PDF]
, 2014 The present invention provides a method of treating or preventing cardiac disorders in a subject in need thereof by inhibiting the expression or function of both miR-499 and miR-208 in the heart cells of the subject. In particular, specific protocols for
Olson, Eric S., van Rooij, Eva
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Site Selective Antibody-Oligonucleotide Conjugation via Microbial Transglutaminase. [PDF]
, 2019 Nucleic Acid Therapeutics (NATs), including siRNAs and AntiSense Oligonucleotides (ASOs), have great potential to drug the undruggable genome. Targeting siRNAs and ASOs to specific cell types of interest has driven dramatic improvement in efficacy and ...
Cui, Xianshu +6 more
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Silencing disease genes in the laboratory and the clinic
, 2011 Synthetic nucleic acids are commonly used laboratory tools for modulating gene expression and have the potential to be widely used in the clinic. Progress towards nucleic acid drugs, however, has been slow and many challenges remain to be overcome before
Corey, David R., Watts, Jonathan K.
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Isolation of pigment cell specific genes in the sea urchin embryo by differential macroarray screening [PDF]
, 2003 New secondary mesenchyme specific genes, expressed exclusively in pigment cells, were isolated from sea urchin embryos using a differential screening of a macroarray cDNA library. The comparison was performed between mRNA populations of embryos having an
Calestani, Cristina +2 more
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Cell-penetrating peptide conjugates of peptide nucleic acids (PNA) as inhibitors of HIV-1 Tat-dependent trans-activation in cells [PDF]
, 2005 The trans-activation response (TAR) RNA stem–loop that occurs at the 5′ end of HIV RNA transcripts is an important antiviral target and is the site of interaction of the HIV-1 Tat protein together with host cellular factors.
Abes, Saïd +7 more
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Polymerase-endonuclease amplification reaction for large-scale enzymatic production of antisense oligonucleotide [PDF]
, 2009 Synthetic oligonucleotides are contaminated with highly homologous failure sequences. Oligonucleotide synthesis is difficult to scale up because it requires expensive equipments, hazardous chemicals, and tedious purification process.
Deming Gou, Xiaolong Wang
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Targeting RyR Activity Boosts Antisense Exon 44 and 45 Skipping in Human DMD Skeletal or Cardiac Muscle Culture Models. [PDF]
, 2019 Systemic delivery of antisense oligonucleotides (AO) for DMD exon skipping has proven effective for reframing DMD mRNA, rescuing dystrophin expression, and slowing disease progression in animal models.
Barthélémy, Florian +6 more
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