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...

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Bibliografiske detaljer
Main Authors:	Aoki, Y, Wood, MJA
Format:	Journal article
Sprog:	English
Udgivet:	IOS Press 2021

Lignende værker

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

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

Advances in oligonucleotide drug delivery
af: Roberts, TC, et al.
Udgivet: (2020)

Recent advances in antisense oligonucleotide therapy in genetic neuromuscular diseases
af: Ashok Verma
Udgivet: (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
af: Gait, M, et al.
Udgivet: (2018)

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

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

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

Oligonucleotides-Based Therapeutics
af: Bernard Lebleu
Udgivet: (2021-09-01)

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

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

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

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

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

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

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

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

Splicing therapy for neuromuscular disease.
af: Douglas, A, et al.
Udgivet: (2013)

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

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

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

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

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

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

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

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

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

Neuromuscular disorders: therapeutic advances.
af: Talbot, K
Udgivet: (2007)

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

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

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

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

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

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

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

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

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

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

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

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