Challenges to oligonucleotides-based therapeutics for Duchenne muscular dystrophy
Skeletal Muscle, 2011 Antisense oligonucleotides are short nucleic acids designed to bind to specific messenger RNAs in order to modulate splicing patterns or inhibit protein translation.
Goyenvalle Aurélie, Davies Kay E
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Cfp1 is required for gene expression dependent H3K4me3 and H3K9 acetylation in embryonic stem cells [PDF]
, 2014 BackgroundTrimethylation of histone H3 lysine 4 (H3K4me3) accumulates at promoters in a gene activity dependent manner. The Set1 complex is responsible for most H3K4me3 in somatic cells and contains the conserved subunit Cfp1, which is implicated in ...
Bird, Adrian +2 more
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Mucin Glycoprotein Nanoparticles Enable a Selective Antisense Therapy for Oncogenic MicroRNAs
Advanced NanoBiomed Research, EarlyView.Mucin glycoproteins are turned into nanoparticles by employing synthetic DNA strands, which have a dual function: they stabilize the nanoparticles and act as binding sites for intracellular miRNA‐21. Thus, upon internalization into tumor cells, these mucin nanoparticles can deplete miRNA‐21 from the cytosol, which induces apoptosis in vitro and in vivo.
Ceren Kimna +9 more
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Gene Therapy Applications in Gastroenterology and Hepatology
Canadian Journal of Gastroenterology, 2000 Advantages and disadvantages of viral vectors and nonviral vectors for gene delivery to digestive organs are reviewed. Advances in systems for the introduction of new gene expression are described, including self-deleting retroviral transfer vectors ...
Catherine H Wu, Lanlan Shen, George Y Wu
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Secondary structure of the segment 5 genomic RNA of influenza A virus and its application for designing antisense oligonucleotides [PDF]
, 2019 Paula Michalak +8 more
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Antisense oligonucleotides targeting the miR-29b binding site in the GRN mRNA increase progranulin translation [PDF]
, 2022 Geetika Aggarwal +19 more
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Genetic diagnosis as a tool for personalized treatment of Duchenne muscular dystrophy [PDF]
, 2016 Accurate definition of genetic mutations causing Duchenne muscular dystrophy (DMD) has always been relevant in order to provide genetic counseling to patients and families, and helps to establish the prognosis in the case where the distinction between ...
Bello, Luca, Pegoraro, Elena
core
Antisense Oligonucleotide Therapy for Calmodulinopathy
CirculationBACKGROUND: Calmodulinopathies are rare inherited arrhythmia syndromes caused by dominant heterozygous variants in CALM1 , CALM2 , or CALM3 , which each encode the identical CaM (calmodulin) protein. We hypothesized that antisense
Raul H. Bortolin +29 more
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CEt KRAS Antisense Oligonucleotide AZD4785 [PDF]
, 2020 National Cancer Institute
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