Results 191 to 200 of about 49,802 (297)

Targeting a pathogenic cryptic exon that drives HLRCC to induce exon skipping. [PDF]

open access: yesMol Ther Nucleic Acids
Maligireddy SS   +10 more
europepmc   +1 more source

mCherry on Top: A Positive Read-Out Cellular Platform for Screening DMD Exon Skipping Xenopeptide-PMO Conjugates. [PDF]

open access: yesBioconjug Chem, 2023
Lessl AL   +7 more
europepmc   +1 more source

Efficacy of exon-skipping therapy for DMD cardiomyopathy with mutations in actin binding domain 1. [PDF]

open access: yesMol Ther Nucleic Acids, 2023
Shiba N   +18 more
europepmc   +1 more source

Severe Clinical Phenotype in Alport Syndrome Due to 2 <i>COL4A4</i> Exon-Skipping Events. [PDF]

open access: yesKidney Med
Pleško J   +6 more
europepmc   +1 more source

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