Search results (606)
« Back to PublicationsmiR-107 represses DMPK and is sequestered by CUG repeats triggering the MSI2/miR-7 pathogenesis axis in myotonic dystrophy
Moreno N. et al, (2025), Molecular Therapy Nucleic Acids, 36
AAV microdystrophin gene replacement therapy for Duchenne muscular dystrophy: progress and prospects.
Chwalenia K. et al, (2025), Gene Ther
Exon skipping peptide-conjugated morpholinos downregulate dynamin 2 to rescue centronuclear myopathy
Moschovaki-Filippidou F. et al, (2025), Brain
Engineering of extracellular vesicles for efficient intracellular delivery of multimodal therapeutics including genome editors.
Liang X. et al, (2025), Nat Commun, 16
uORF-targeting steric block antisense oligonucleotides do not reproducibly increase RNASEH1 expression
Ahlskog N. et al, (2025), Molecular Therapy - Nucleic Acids, 36, 102406 - 102406
Activation-induced thrombospondin-4 works with thrombospondin-1 to build cytotoxic supramolecular attack particles.
Cassioli C. et al, (2025), Proc Natl Acad Sci U S A, 122
Progress and prospects in antisense oligonucleotide-mediated exon skipping therapies for Duchenne muscular dystrophy.
Chwalenia K. et al, (2025), J Muscle Res Cell Motil
Inflammatory disease microbiomes share a functional pathogenicity predicted by C-reactive protein.
Britton GJ. et al, (2025), bioRxiv
Tissue-specific modulation of CRISPR activity by miRNA-sensing guide RNAs.
Garcia-Guerra A. et al, (2025), Nucleic Acids Res, 53
An extracellular vesicle delivery platform based on the PTTG1IP protein.
Martin Perez C. et al, (2024), Extracell Vesicle, 4
Antibody-displaying extracellular vesicles for targeted cancer therapy.
Wiklander OPB. et al, (2024), Nat Biomed Eng, 8, 1453 - 1468
Synthesis, Biophysical and Biological Evaluation of Splice-Switching Oligonucleotides with Multiple LNA-Phosphothiotriester Backbones.
Dhara D. et al, (2024), J Am Chem Soc, 146, 29773 - 29781
Snorkel-tag based affinity chromatography for recombinant extracellular vesicle purification.
Bobbili MR. et al, (2024), J Extracell Vesicles, 13
TDP-43 regulates LC3ylation in neural tissue through ATG4B cryptic splicing inhibition.
Torres P. et al, (2024), Acta Neuropathol, 148
Urine-derived stem cells serve as a robust platform for generating native or engineered extracellular vesicles.
Boysen AT. et al, (2024), Stem Cell Res Ther, 15
Novel Endogenous Engineering Platform for Robust Loading and Delivery of Functional mRNA by Extracellular Vesicles.
Zickler AM. et al, (2024), Adv Sci (Weinh)