Search results (573)
« Back to PublicationsTDP-43 regulates LC3ylation in neural tissue through ATG4B cryptic splicing inhibition.
Journal article
Torres P. et al, (2024), Acta Neuropathol, 148
Novel Endogenous Engineering Platform for Robust Loading and Delivery of Functional mRNA by Extracellular Vesicles.
Journal article
Zickler AM. et al, (2024), Adv Sci (Weinh)
uORF-targeting steric block antisense oligonucleotides do not reproducibly activate RNASEH1 expression
Preprint
Ahlskog N. et al, (2024)
Phosphatidylserine-exposing extracellular vesicles in body fluids are an innate defence against apoptotic mimicry viral pathogens.
Journal article
Groß R. et al, (2024), Nat Microbiol
Multiple surgical resections for progressive IDH wildtype glioblastoma-is it beneficial?
Journal article
Honeyman SI. et al, (2024), Acta Neurochir (Wien), 166
A transcriptomics-based drug repositioning approach to identify drugs with similar activities for the treatment of muscle pathologies in spinal muscular atrophy (SMA) models.
Journal article
Hoolachan JM. et al, (2024), Hum Mol Genet, 33, 400 - 425
Why is early-onset atrial fibrillation uncommon in patients with Duchenne Muscular Dystrophy? Insights from the mdx mouse.
Journal article
Nguyen M-N. et al, (2024), Cardiovasc Res
Snorkel-tag Based Affinity Chromatography for Recombinant Extracellular Vesicle Purification
Preprint
Bobbili MR. et al, (2024)
Tissue-specific modulation of CRISPR activity by miRNA-sensing guide RNAs
Preprint
Garcia-Guerra A. et al, (2024)
Long-term dystrophin restoration supports development of splice correction therapy for DMD patients with exon 2 duplications
Journal article
Roberts TC., (2023), Molecular Therapy Methods and Clinical Development, 31
Peptide-conjugated antimiRs improve myotonic dystrophy type 1 phenotypes by promoting endogenous MBNL1 expression.
Journal article
González-Martínez I. et al, (2023), Mol Ther Nucleic Acids, 34
PRMT inhibitor promotes SMN2 exon 7 inclusion and synergizes with nusinersen to rescue SMA mice.
Journal article
Kordala AJ. et al, (2023), EMBO Mol Med, 15
An Induced Pluripotent Stem Cell-Derived Human Blood-Brain Barrier (BBB) Model to Test the Crossing by Adeno-Associated Virus (AAV) Vectors and Antisense Oligonucleotides.
Journal article
Selvakumaran J. et al, (2023), Biomedicines, 11
Modulation of Pro-Inflammatory IL-6 Trans-Signaling Axis by Splice Switching Oligonucleotides as a Therapeutic Modality in Inflammation.
Journal article
Gupta D. et al, (2023), Cells, 12
EV-mediated promotion of myogenic differentiation is dependent on dose, collection medium, and isolation method
Journal article
Hanson B. et al, (2023), Molecular Therapy - Nucleic Acids, 33, 511 - 528
Therapeutic approaches for Duchenne muscular dystrophy.
Journal article
Roberts TC. et al, (2023), Nat Rev Drug Discov
A Purposefully Designed pH/GSH-Responsive MnFe-Based Metal-Organic Frameworks as Cascade Nanoreactor for Enhanced Chemo-Chemodynamic-Starvation Synergistic Therapy.
Journal article
Chen WJ. et al, (2023), Small
An extracellular vesicle delivery platform based on the PTTG1IP protein
Preprint
Perez CM. et al, (2023)
Investigating Eukaryotic Elongation Factor 2 Kinase/Eukaryotic Translation Elongation Factor 2 Pathway Regulation and Its Role in Protein Synthesis Impairment during Disuse-Induced Skeletal Muscle Atrophy.
Journal article
Vilchinskaya N. et al, (2023), Am J Pathol, 193, 813 - 828
Tumor-targeted glycogen nanoparticles loaded with hemin and glucose oxidase to promote tumor synergistic therapy.
Journal article
Qiu L. et al, (2023), Int J Biol Macromol, 239