Emerging oligonucleotide therapeutics for rare neuromuscular diseases

Emerging oligonucleotide therapeutics for rare neuromuscular diseases

Research and drug development concerning rare diseases are at the cutting edge of scientific technology. To date, over 7,000 rare diseases have been identified. Despite their individual rarity, 1 in 10 individuals worldwide is affected by a rare condition. For the majority of these diseases, there i...

Disgrifiad llawn

Manylion Llyfryddiaeth
Prif Awduron:	Aoki, Y, Wood, MJA
Fformat:	Journal article
Iaith:	English
Cyhoeddwyd:	IOS Press 2021

Eitemau Tebyg

Oligonucleotide-based therapies for neuromuscular disease
gan: Douglas, A, et al.
Cyhoeddwyd: (2015)

Peptide-conjugate antisense based splice-correction for Duchenne muscular dystrophy and other neuromuscular diseases
gan: Tsoumpra, MK, et al.
Cyhoeddwyd: (2019)

Advances in oligonucleotide drug delivery
gan: Roberts, TC, et al.
Cyhoeddwyd: (2020)

Recent advances in antisense oligonucleotide therapy in genetic neuromuscular diseases
gan: Ashok Verma
Cyhoeddwyd: (2018-01-01)

Cell-penetrating peptide conjugates of steric blocking oligonucleotides as therapeutics for neuromuscular diseases from a historical perspective to current prospects of treatment
gan: Gait, M, et al.
Cyhoeddwyd: (2018)

Antisense Oligonucleotide Gene Therapy for Neuromuscular Disorders
gan: Ryan Gotesman
Cyhoeddwyd: (2018-11-01)

Development of LNA gapmer oligonucleotide-based therapy for ALS/FTD caused by the C9orf72 repeat expansion
gan: Sathyaprakash, C, et al.
Cyhoeddwyd: (2020)

Antisense oligonucleotides and their applications in rare neurological diseases
gan: Simon McDowall, et al.
Cyhoeddwyd: (2024-09-01)

Oligonucleotides-Based Therapeutics
gan: Bernard Lebleu
Cyhoeddwyd: (2021-09-01)

Therapeutic Oligonucleotides for Patients with Inflammatory Bowel Diseases
gan: Marafini I, et al.
Cyhoeddwyd: (2020-06-01)

Challenges for antisense oligonucleotide-based therapeutics, in particular for exon 51-skipping in Duchenne muscular dystrophy
gan: Aoki, Y, et al.
Cyhoeddwyd: (2011)

Therapeutic Oligonucleotides Targeting Liver Disease: TTR Amyloidosis
gan: Christoph Niemietz, et al.
Cyhoeddwyd: (2015-09-01)

Delivery of therapeutic oligonucleotides in nanoscale
gan: Lei Wu, et al.
Cyhoeddwyd: (2022-01-01)

Fine tuning of phosphorothioate inclusion in 2'-O-methyl oligonucleotides contributes to specific cell targeting for splice-switching modulation
gan: Aoki, Y, et al.
Cyhoeddwyd: (2021)

Immortalized canine dystrophic myoblast cell lines for development of peptide-conjugated splice-switching oligonucleotides
gan: Tone, Y, et al.
Cyhoeddwyd: (2021)

The endosomal escape vehicle platform enhances delivery of oligonucleotides in preclinical models of neuromuscular disorders
gan: Xiang Li, et al.
Cyhoeddwyd: (2023-09-01)

Mesyl phosphoramidate backbone modified antisense oligonucleotides targeting miR-21 with enhanced in vivo therapeutic potency
gan: Patutina, OA, et al.
Cyhoeddwyd: (2020)

Splicing therapy for neuromuscular disease.
gan: Douglas, A, et al.
Cyhoeddwyd: (2013)

The role of chemistry in the success of oligonucleotides as therapeutics
gan: Pawan Kumar, et al.
Cyhoeddwyd: (2022-02-01)

TRIM32 and Malin in Neurological and Neuromuscular Rare Diseases
gan: Lorena Kumarasinghe, et al.
Cyhoeddwyd: (2021-04-01)

Selective Neuromuscular Denervation in Taiwanese Severe SMA Mouse Can Be Reversed by Morpholino Antisense Oligonucleotides.
gan: Te-Lin Lin, et al.
Cyhoeddwyd: (2016-01-01)

Antisense oligonucleotide therapeutics in clinical trials for the treatment of inherited retinal diseases
gan: Xue, K, et al.
Cyhoeddwyd: (2020)

Short G-rich oligonucleotides as a potential therapeutic for Huntington's Disease
gan: Parekh-Olmedo Hetal, et al.
Cyhoeddwyd: (2006-10-01)

Antisense Oligonucleotide: Basic Concepts and Therapeutic Application in Inflammatory Bowel Disease
gan: Davide Di Fusco, et al.
Cyhoeddwyd: (2019-03-01)

Covalently attached intercalators restore duplex stability and splice-switching activity to triazole-modified oligonucleotides
gan: Dysko, A, et al.
Cyhoeddwyd: (2022)

Targeting RNA to treat neuromuscular disease.
gan: Muntoni, F, et al.
Cyhoeddwyd: (2011)

Spinal muscular atrophy: antisense oligonucleotide therapy opens the door to an integrated therapeutic landscape
gan: Wood, M, et al.
Cyhoeddwyd: (2017)

Neuromuscular disorders: therapeutic advances.
gan: Talbot, K
Cyhoeddwyd: (2007)

Delivery of oligonucleotide‐based therapeutics: challenges and opportunities
gan: Hammond, SM, et al.
Cyhoeddwyd: (2021)

Amphiphilic Oligonucleotide Derivatives—Promising Tools for Therapeutics
gan: Irina A. Bauer, et al.
Cyhoeddwyd: (2024-11-01)

Delivery of oligonucleotide‐based therapeutics: challenges and opportunities
gan: Suzan M Hammond, et al.
Cyhoeddwyd: (2021-04-01)

Peptide–Oligonucleotide Conjugation: Chemistry and Therapeutic Applications
gan: Anna L. Malinowska, et al.
Cyhoeddwyd: (2024-09-01)

Nonviral delivery systems for antisense oligonucleotide therapeutics
gan: Si Huang, et al.
Cyhoeddwyd: (2022-09-01)

Chemistry of Therapeutic Oligonucleotides That Drives Interactions with Biomolecules
gan: Chisato Terada, et al.
Cyhoeddwyd: (2022-11-01)

Oligonucleotide Therapeutics: From Discovery and Development to Patentability
gan: Lara Moumné, et al.
Cyhoeddwyd: (2022-01-01)

Mesyl phosphoramidate oligonucleotides as potential splice-switching agents: impact of backbone structure on activity and intracellular localization
gan: Hammond, SM, et al.
Cyhoeddwyd: (2021)

Improving translational studies: lessons from rare neuromuscular diseases
gan: Dominic Wells
Cyhoeddwyd: (2015-10-01)

Therapeutic approaches for Duchenne muscular dystrophy
gan: Roberts, TC, et al.
Cyhoeddwyd: (2023)

Regeneration mechanisms and therapeutic strategies for neuromuscular junctions in aging and diseases
gan: Masashi Fujitani, et al.
Cyhoeddwyd: (2025-01-01)

Editorial: Diagnosis, animal models and therapeutic interventions for neuromuscular diseases
gan: Muthukumar Karuppasamy, et al.
Cyhoeddwyd: (2024-09-01)