Deciphering the role of alternative splicing in neoplastic diseases for immune-oncological therapies
M Bauer, CM Schöbel, C Wickenhauser… - Frontiers in …, 2024 - frontiersin.org
Alternative splicing (AS) is an important molecular biological mechanism regulated by
complex mechanisms involving a plethora of cis and trans-acting elements. Furthermore, AS …
complex mechanisms involving a plethora of cis and trans-acting elements. Furthermore, AS …
Non-viral delivery of nucleic acid for treatment of rare diseases of the muscle
Rare muscular disorders (RMDs) are disorders that affect a small percentage of the
population. The disorders which are attributed to genetic mutations often manifest in the form …
population. The disorders which are attributed to genetic mutations often manifest in the form …
CRISPR-mediated megabase-scale transgene de-duplication to generate a functional single-copy full-length humanized DMD mouse model
Background The development of sequence-specific precision treatments like CRISPR gene
editing therapies for Duchenne muscular dystrophy (DMD) requires sequence humanized …
editing therapies for Duchenne muscular dystrophy (DMD) requires sequence humanized …
Considering the promise of vamorolone for treating Duchenne muscular dystrophy
MD Grounds, EM Lloyd - Journal of Neuromuscular Diseases, 2023 - content.iospress.com
This commentary provides an independent consideration of data related to the drug
vamorolone (VBP15) as an alternative steroid proposed for treatment of Duchenne muscular …
vamorolone (VBP15) as an alternative steroid proposed for treatment of Duchenne muscular …
Challenges of Assessing Exon 53 Skipping of the Human DMD Transcript with Locked Nucleic Acid-Modified Antisense Oligonucleotides in a Mouse Model for …
S Engelbeen, D O'Reilly, D Van De Vijver… - nucleic acid …, 2023 - liebertpub.com
Antisense oligonucleotide (AON)-mediated exon skipping is a promising therapeutic
approach for Duchenne muscular dystrophy (DMD) patients to restore dystrophin expression …
approach for Duchenne muscular dystrophy (DMD) patients to restore dystrophin expression …
[HTML][HTML] An Updated Analysis of Exon-Skipping Applicability for Duchenne Muscular Dystrophy Using the UMD-DMD Database
J Leckie, A Zia, T Yokota - Genes, 2024 - mdpi.com
Background/Objectives: Antisense oligonucleotide (ASO)-mediated exon-skipping is an
effective approach to restore the disrupted reading frame of the dystrophin gene for the …
effective approach to restore the disrupted reading frame of the dystrophin gene for the …
Megabase-scale Transgene De-Duplication to Generate a Functional Single-Copy Full-Length Humanized DMD Mouse Model
The development of sequence-specific precision treatments like CRISPR gene editing
therapies for Duchenne Muscular Dystrophy (DMD) requires sequence humanised animal …
therapies for Duchenne Muscular Dystrophy (DMD) requires sequence humanised animal …
Design, synthesis, and purification of biopharmaceuticals, included highly modified oligonucleotides.
L Preti - 2024 - sfera.unife.it
Abstract Riassunto tesi dottorato, Lorenzo Preti Questo lavoro di tesi si concentra sulla
bioconiugazione, riflettendo sugli argomenti affrontati durante i miei studi di dottorato. La tesi …
bioconiugazione, riflettendo sugli argomenti affrontati durante i miei studi di dottorato. La tesi …
Exon Skipping Using Antisense Oligonucleotides as a Therapy for Duchenne Muscular Dystrophy
D Argyelan - jmsgr.tamhsc.edu
Background: Duchenne Muscular Dystrophy (DMD) is a fatal X-linked muscle wasting
disease affecting approximately 0.02% of male newborns. Characterized by progressive …
disease affecting approximately 0.02% of male newborns. Characterized by progressive …