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Development of exon skip** therapies for Duchenne muscular dystrophy: a critical review and a perspective on the outstanding issues
Duchenne muscular dystrophy (DMD) is a rare, severe, progressive muscle-wasting disease
leading to disability and premature death. Patients lack the muscle membrane-stabilizing …
leading to disability and premature death. Patients lack the muscle membrane-stabilizing …
[ΒΙΒΛΙΟ][B] RNA, the epicenter of genetic information
The origin story and emergence of molecular biology is muddled. The early triumphs in
bacterial genetics and the complexity of animal and plant genomes complicate an intricate …
bacterial genetics and the complexity of animal and plant genomes complicate an intricate …
Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy
DE Frank, FJ Schnell, C Akana, SH El-Husayni… - Neurology, 2020 - neurology.org
Objective To report safety, pharmacokinetics, exon 53 skip**, and dystrophin expression
in golodirsen-treated patients with Duchenne muscular dystrophy (DMD) amenable to exon …
in golodirsen-treated patients with Duchenne muscular dystrophy (DMD) amenable to exon …
ALS-Associated FUS Mutations Result in Compromised FUS Alternative Splicing and Autoregulation
Y Zhou, S Liu, G Liu, A Öztürk, GG Hicks - PLoS genetics, 2013 - journals.plos.org
The gene encoding a DNA/RNA binding protein FUS/TLS is frequently mutated in
amyotrophic lateral sclerosis (ALS). Mutations commonly affect its carboxy-terminal nuclear …
amyotrophic lateral sclerosis (ALS). Mutations commonly affect its carboxy-terminal nuclear …
Optimization of antisense-mediated exon skip** for Duchenne muscular dystrophy
K Dzierlega, T Yokota - Gene therapy, 2020 - nature.com
Duchenne muscular dystrophy (DMD) is one of the most common lethal muscle-wasting
disorders affecting young boys caused by mutations in the DMD gene. Exon skip** has …
disorders affecting young boys caused by mutations in the DMD gene. Exon skip** has …
Antisense-mediated modulation of splicing is one of the few fields where antisense
oligonucleotides (AONs) have been able to live up to their expectations. In this approach …
oligonucleotides (AONs) have been able to live up to their expectations. In this approach …
The FSHD atrophic myotube phenotype is caused by DUX4 expression
C Vanderplanck, E Ansseau, S Charron, N Stricwant… - PloS one, 2011 - journals.plos.org
Background Facioscapulohumeral muscular dystrophy (FSHD) is linked to deletions in 4q35
within the D4Z4 repeat array in which we identified the dou ble homeobo x 4 (DUX4) gene …
within the D4Z4 repeat array in which we identified the dou ble homeobo x 4 (DUX4) gene …
Comparative analysis of antisense oligonucleotide sequences for targeted skip** of exon 51 during dystrophin pre-mRNA splicing in human muscle
V Arechavala-Gomeza, IR Graham… - Human Gene …, 2007 - liebertpub.com
Duchenne muscular dystrophy (DMD) is caused by mutations in the dystrophin gene that
result in the absence of functional protein. In the majority of cases these are out-of-frame …
result in the absence of functional protein. In the majority of cases these are out-of-frame …
Guidelines for antisense oligonucleotide design and insight into splice-modulating mechanisms
Antisense oligonucleotides (AONs) can interfere with mRNA processing through RNase H–
mediated degradation, translational arrest, or modulation of splicing. The antisense …
mediated degradation, translational arrest, or modulation of splicing. The antisense …