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Search results (89)

« Back to Publications

Targeted BDNF upregulation via upstream open reading frame disruption.

Feng N. et al, (2026), Mol Ther, 34, 1652 - 1671

Timing matters: exon skipping therapy is most effective when initiated early in a mouse model of Duchenne muscular dystrophy

Stenler S. et al, (2025)

Progress and prospects in antisense oligonucleotide-mediated exon skipping therapies for Duchenne muscular dystrophy.

Chwalenia K. et al, (2025), J Muscle Res Cell Motil, 46, 293 - 300

AAV microdystrophin gene replacement therapy for Duchenne muscular dystrophy: progress and prospects.

Chwalenia K. et al, (2025), Gene Ther, 32, 447 - 461

Myonuclear domain-associated and central nucleation-dependent spatial restriction of dystrophin protein expression in a novel DMD mouse model

Chwalenia K. et al, (2025)

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), Mol Ther Nucleic Acids, 36

Targeted BDNF upregulation via upstream open reading frame disruption

Feng N. et al, (2025)

An extracellular vesicle delivery platform based on the PTTG1IP protein.

Martin Perez C. et al, (2024), Extracell Vesicle, 4

uORF-targeting steric block antisense oligonucleotides do not reproducibly activate RNASEH1 expression

Ahlskog N. et al, (2024)

Long-term dystrophin restoration supports development of splice correction therapy for DMD patients with exon 2 duplications.

Roberts TC., (2023), Mol Ther Methods Clin Dev, 31

Therapeutic approaches for Duchenne muscular dystrophy.

Roberts TC. et al, (2023), Nat Rev Drug Discov, 22, 917 - 934

EV-mediated promotion of myogenic differentiation is dependent on dose, collection medium, and isolation method.

Hanson B. et al, (2023), Mol Ther Nucleic Acids, 33, 511 - 528

An extracellular vesicle delivery platform based on the PTTG1IP protein

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.

Vilchinskaya N. et al, (2023), Am J Pathol, 193, 813 - 828

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