RNA-based therapeutics for neurological diseases
K Anthony - RNA biology, 2022 - Taylor & Francis
ABSTRACT RNA-based therapeutics have entered the mainstream with seemingly limitless
possibilities to treat all categories of neurological disease. Here, common RNA-based drug …
possibilities to treat all categories of neurological disease. Here, common RNA-based drug …
[HTML][HTML] Antisense oligonucleotide-based therapy for neuromuscular disease
Neuromuscular disorders such as Duchenne Muscular Dystrophy and Spinal Muscular
Atrophy are neurodegenerative genetic diseases characterized primarily by muscle …
Atrophy are neurodegenerative genetic diseases characterized primarily by muscle …
[HTML][HTML] Antisense oligonucleotides as a potential treatment for brain deficits observed in myotonic dystrophy type 1
S Ait Benichou, D Jauvin, T de Serres-Bérard, M Pierre… - Gene Therapy, 2022 - nature.com
Myotonic dystrophy, or dystrophia myotonica type 1 (DM1), is a multi-systemic disorder and
is the most common adult form of muscular dystrophy. It affects not only muscles but also …
is the most common adult form of muscular dystrophy. It affects not only muscles but also …
[HTML][HTML] Myotonic dystrophies: state of the art of new therapeutic developments for the CNS
Myotonic dystrophies are multisystemic diseases characterized not only by muscle and heart
dysfunction but also by CNS alteration. They are now recognized as brain diseases affecting …
dysfunction but also by CNS alteration. They are now recognized as brain diseases affecting …
Efficient CRISPR/Cas9-mediated editing of trinucleotide repeat expansion in myotonic dystrophy patient-derived iPS and myogenic cells
S Dastidar, S Ardui, K Singh, D Majumdar… - Nucleic acids …, 2018 - academic.oup.com
CRISPR/Cas9 is an attractive platform to potentially correct dominant genetic diseases by
gene editing with unprecedented precision. In the current proof-of-principle study, we …
gene editing with unprecedented precision. In the current proof-of-principle study, we …
Limb-girdle muscular dystrophies—international collaborations for translational research
R Thompson, V Straub - Nature Reviews Neurology, 2016 - nature.com
The limb-girdle muscular dystrophies (LGMDs) are a diverse group of genetic
neuromuscular conditions that usually manifest in the proximal muscles of the hip and …
neuromuscular conditions that usually manifest in the proximal muscles of the hip and …
Immortalized human myotonic dystrophy muscle cell lines to assess therapeutic compounds
L Arandel, M Polay Espinoza… - Disease models & …, 2017 - journals.biologists.com
ABSTRACT Myotonic dystrophy type 1 (DM1) and type 2 (DM2) are autosomal dominant
neuromuscular diseases caused by microsatellite expansions and belong to the family of …
neuromuscular diseases caused by microsatellite expansions and belong to the family of …
Genome engineering: a new approach to gene therapy for neuromuscular disorders
CE Nelson, JN Robinson-Hamm… - Nature reviews …, 2017 - nature.com
For many neuromuscular disorders, including Duchenne muscular dystrophy, spinal
muscular atrophy and myotonic dystrophy, the genetic causes are well known. Gene therapy …
muscular atrophy and myotonic dystrophy, the genetic causes are well known. Gene therapy …
[HTML][HTML] Genome editing of expanded CTG repeats within the human DMPK gene reduces nuclear RNA foci in the muscle of DM1 mice
Myotonic dystrophy type 1 (DM1) is caused by a CTG repeat expansion located in the 3′
UTR of the DMPK gene. Expanded DMPK transcripts aggregate into nuclear foci and alter …
UTR of the DMPK gene. Expanded DMPK transcripts aggregate into nuclear foci and alter …
An overview of recent advances and clinical applications of exon skipping and splice modulation for muscular dystrophy and various genetic diseases
M Rodrigues, T Yokota - Exon Skipping and Inclusion Therapies: Methods …, 2018 - Springer
Exon skipping is a therapeutic approach that is feasible for various genetic diseases and
has been studied and developed for over two decades. This approach uses antisense …
has been studied and developed for over two decades. This approach uses antisense …