Targeting a pathogenic cryptic exon that drives HLRCC to induce exon skipping. [PDF]
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]
Lessl AL +7 more
europepmc +1 more source
Identification of cryptic splice site, exon skipping, and novel point mutations in type I CD36 deficiency [PDF]
Haruo Hanawa
openalex +1 more source
CRISPR-directed exon skipping; friend or foe (opportunity or warning sign)? [PDF]
Banas KH, Kmiec EB.
europepmc +1 more source
Efficacy of exon-skipping therapy for DMD cardiomyopathy with mutations in actin binding domain 1. [PDF]
Shiba N +18 more
europepmc +1 more source
Procollagen with Skipping of α1(I) Exon 41 Has Lower Binding Affinity for α1(I) C-telopeptide, Impaired in Vitro Fibrillogenesis, and Altered Fibril Morphology [PDF]
Wayne A. Cabral +5 more
openalex +1 more source
Morpholino antisense oligonucleotide induced dystrophin exon 23 skipping in mdx mouse muscle [PDF]
B L Gebski +3 more
openalex +1 more source
Severe Clinical Phenotype in Alport Syndrome Due to 2 <i>COL4A4</i> Exon-Skipping Events. [PDF]
Pleško J +6 more
europepmc +1 more source

