Search Results - "Boehler, Jessica"
-
1
Effect of endurance exercise on microRNAs in myositis skeletal muscle-A randomized controlled study
Published in PloS one (22-08-2017)“…To identify changes in skeletal muscle microRNA expression after endurance exercise and associate the identified microRNAs with mRNA and protein expression to…”
Get full text
Journal Article -
2
Myoblasts and macrophages are required for therapeutic morpholino antisense oligonucleotide delivery to dystrophic muscle
Published in Nature communications (16-10-2017)“…Exon skipping is a promising therapeutic strategy for Duchenne muscular dystrophy (DMD), employing morpholino antisense oligonucleotides (PMO-AO) to exclude…”
Get full text
Journal Article -
3
N-terminal titin fragment: a non-invasive, pharmacodynamic biomarker for microdystrophin efficacy
Published in Skeletal muscle (16-01-2024)“…Multiple clinical trials to assess the efficacy of AAV-directed gene transfer in participants with Duchenne muscular dystrophy (DMD) are ongoing. The success…”
Get full text
Journal Article -
4
A novel estrogen receptor 1: sphingomyelin phosphodiesterase acid-like 3B pathway mediates rituximab response in myositis patients
Published in Rheumatology (Oxford, England) (01-08-2023)“…Abstract Objectives The B-cell depleting biologic, rituximab, is used to treat refractory autoimmune myositis. However, the beneficial effects of rituximab…”
Get full text
Journal Article -
5
Mitochondrial dysfunction and role of harakiri in the pathogenesis of myositis
Published in The Journal of pathology (01-10-2019)“…The etiology of myositis is unknown. Although attempts to identify viruses in myositis skeletal muscle have failed, several studies have identified the…”
Get full text
Journal Article -
6
Cc2d1a Loss of Function Disrupts Functional and Morphological Development in Forebrain Neurons Leading to Cognitive and Social Deficits
Published in Cerebral cortex (New York, N.Y. 1991) (01-02-2017)“…Loss-of-function (LOF) mutations in CC2D1A cause a spectrum of neurodevelopmental disorders, including intellectual disability, autism spectrum disorder, and…”
Get full text
Journal Article -
7
Clinical potential of microdystrophin as a surrogate endpoint
Published in Neuromuscular disorders : NMD (01-01-2023)“…•We directly address the similarities and difference between rationally designed microdystrophin proteins and ‘natural’ skipped dystrophins from both a…”
Get full text
Journal Article -
8
Morpholino‐induced exon skipping stimulates cell‐mediated and humoral responses to dystrophin in mdx mice
Published in The Journal of pathology (01-07-2019)“…Exon skipping is a promising genetic therapeutic strategy for restoring dystrophin expression in the treatment of Duchenne muscular dystrophy (DMD). The…”
Get full text
Journal Article -
9
Membrane recruitment of nNOSµ in microdystrophin gene transfer to enhance durability
Published in Neuromuscular disorders : NMD (01-10-2019)“…Several gene transfer clinical trials are currently ongoing with the common aim of delivering a shortened version of dystrophin, termed a microdystrophin, for…”
Get full text
Journal Article -
10
Creation and characterization of an immortalized canine myoblast cell line: Myok9
Published in Mammalian genome (01-04-2020)“…The availability of an in vitro canine cell line would reduce the need for dogs for primary in vitro cell culture and reduce overall cost in pre-clinical…”
Get full text
Journal Article -
11
Author Correction: Myoblasts and macrophages are required for therapeutic morpholino antisense oligonucleotide delivery to dystrophic muscle
Published in Nature communications (23-03-2018)“…The originally published version of this Article contained an error in Figure 6. In panel b, the top graph (BrdU 21–24d) and the bottom graph (BrdU 28–31d)…”
Get full text
Journal Article -
12
Author Correction: Myoblasts and macrophages are required for therapeutic morpholino antisense oligonucleotide delivery to dystrophic muscle
Published in Nature communications (15-01-2018)“…In the original version of this Article, financial support was not fully acknowledged. The PDF and HTML versions of the Article have now been corrected to…”
Get full text
Journal Article -
13
Mitochondrial dysfunction and consequences in calpain-3-deficient muscle
Published in Skeletal muscle (11-12-2020)“…Nonsense or loss-of-function mutations in the non-lysosomal cysteine protease calpain-3 result in limb-girdle muscular dystrophy type 2A (LGMD2A). While…”
Get full text
Journal Article -
14
Synthesis, Characterization, and Function of an RNA-Based Transfection Reagent
Published in Current protocols in nucleic acid chemistry (01-03-2018)“…A synthetic 8-mer, amphipathic, trans-acting poly-2'-O-methyluridylic thiophosphate triester RNA element (2'-OMeUtaPS) can be prepared using solid-phase…”
Get more information
Journal Article -
15
Elusive sources of variability of dystrophin rescue by exon skipping
Published in Skeletal muscle (01-12-2015)“…Systemic delivery of anti-sense oligonucleotides to Duchenne muscular dystrophy (DMD) patients to induce de novo dystrophin protein expression in muscle (exon…”
Get full text
Journal Article -
16
Annexin A2 links poor myofiber repair with inflammation and adipogenic replacement of the injured muscle
Published in Human molecular genetics (01-06-2017)“…Repair of skeletal muscle after sarcolemmal damage involves dysferlin and dysferlin-interacting proteins such as annexins. Mice and patient lacking dysferlin…”
Get full text
Journal Article -
17
584. Myoblast Fusion Mediates Morpholino Entry and Exon Skipping In Dystrophic Muscle
Published in Molecular therapy (01-05-2016)Get full text
Journal Article -
18
Role of Epigenetic Mechanisms in the Pathogenesis of Idiopathic Inflammatory Myopathies
Published 01-01-2017“…The idiopathic inflammatory myopathies (IIM) (polymyositis [PM], dermatomyositis [DM], and inclusion body myositis [IBM]) are a heterogeneous group of…”
Get full text
Dissertation -
19
Effects of Chronic, Maximal Phosphorodiamidate Morpholino Oligomer (PMO) Dosing on Muscle Function and Dystrophin Restoration in a Mouse Model of Duchenne Muscular Dystrophy
Published in Journal of neuromuscular diseases (01-01-2021)“…Phosphorodiamidate morpholino oligomer (PMO)-mediated exon skipping is currently used in clinical development to treat Duchenne muscular dystrophy (DMD), with…”
Get more information
Journal Article