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...

Bibliografski detalji
Glavni autori:	Aoki, Y, Wood, MJA
Format:	Journal article
Jezik:	English
Izdano:	IOS Press 2021

Slični predmeti

Oligonucleotide-based therapies for neuromuscular disease
od: Douglas, A, i dr.
Izdano: (2015)

Peptide-conjugate antisense based splice-correction for Duchenne muscular dystrophy and other neuromuscular diseases
od: Tsoumpra, MK, i dr.
Izdano: (2019)

Advances in oligonucleotide drug delivery
od: Roberts, TC, i dr.
Izdano: (2020)

Recent advances in antisense oligonucleotide therapy in genetic neuromuscular diseases
od: Ashok Verma
Izdano: (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
od: Gait, M, i dr.
Izdano: (2018)

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

Development of LNA gapmer oligonucleotide-based therapy for ALS/FTD caused by the C9orf72 repeat expansion
od: Sathyaprakash, C, i dr.
Izdano: (2020)

Antisense oligonucleotides and their applications in rare neurological diseases
od: Simon McDowall, i dr.
Izdano: (2024-09-01)

Oligonucleotides-Based Therapeutics
od: Bernard Lebleu
Izdano: (2021-09-01)

Therapeutic Oligonucleotides for Patients with Inflammatory Bowel Diseases
od: Marafini I, i dr.
Izdano: (2020-06-01)

Challenges for antisense oligonucleotide-based therapeutics, in particular for exon 51-skipping in Duchenne muscular dystrophy
od: Aoki, Y, i dr.
Izdano: (2011)

Therapeutic Oligonucleotides Targeting Liver Disease: TTR Amyloidosis
od: Christoph Niemietz, i dr.
Izdano: (2015-09-01)

Delivery of therapeutic oligonucleotides in nanoscale
od: Lei Wu, i dr.
Izdano: (2022-01-01)

Fine tuning of phosphorothioate inclusion in 2'-O-methyl oligonucleotides contributes to specific cell targeting for splice-switching modulation
od: Aoki, Y, i dr.
Izdano: (2021)

Immortalized canine dystrophic myoblast cell lines for development of peptide-conjugated splice-switching oligonucleotides
od: Tone, Y, i dr.
Izdano: (2021)

The endosomal escape vehicle platform enhances delivery of oligonucleotides in preclinical models of neuromuscular disorders
od: Xiang Li, i dr.
Izdano: (2023-09-01)

Mesyl phosphoramidate backbone modified antisense oligonucleotides targeting miR-21 with enhanced in vivo therapeutic potency
od: Patutina, OA, i dr.
Izdano: (2020)

Splicing therapy for neuromuscular disease.
od: Douglas, A, i dr.
Izdano: (2013)

The role of chemistry in the success of oligonucleotides as therapeutics
od: Pawan Kumar, i dr.
Izdano: (2022-02-01)

TRIM32 and Malin in Neurological and Neuromuscular Rare Diseases
od: Lorena Kumarasinghe, i dr.
Izdano: (2021-04-01)

Selective Neuromuscular Denervation in Taiwanese Severe SMA Mouse Can Be Reversed by Morpholino Antisense Oligonucleotides.
od: Te-Lin Lin, i dr.
Izdano: (2016-01-01)

Antisense oligonucleotide therapeutics in clinical trials for the treatment of inherited retinal diseases
od: Xue, K, i dr.
Izdano: (2020)

Short G-rich oligonucleotides as a potential therapeutic for Huntington's Disease
od: Parekh-Olmedo Hetal, i dr.
Izdano: (2006-10-01)

Antisense Oligonucleotide: Basic Concepts and Therapeutic Application in Inflammatory Bowel Disease
od: Davide Di Fusco, i dr.
Izdano: (2019-03-01)

Covalently attached intercalators restore duplex stability and splice-switching activity to triazole-modified oligonucleotides
od: Dysko, A, i dr.
Izdano: (2022)

Targeting RNA to treat neuromuscular disease.
od: Muntoni, F, i dr.
Izdano: (2011)

Spinal muscular atrophy: antisense oligonucleotide therapy opens the door to an integrated therapeutic landscape
od: Wood, M, i dr.
Izdano: (2017)

Neuromuscular disorders: therapeutic advances.
od: Talbot, K
Izdano: (2007)

Delivery of oligonucleotide‐based therapeutics: challenges and opportunities
od: Hammond, SM, i dr.
Izdano: (2021)

Amphiphilic Oligonucleotide Derivatives—Promising Tools for Therapeutics
od: Irina A. Bauer, i dr.
Izdano: (2024-11-01)

Delivery of oligonucleotide‐based therapeutics: challenges and opportunities
od: Suzan M Hammond, i dr.
Izdano: (2021-04-01)

Peptide–Oligonucleotide Conjugation: Chemistry and Therapeutic Applications
od: Anna L. Malinowska, i dr.
Izdano: (2024-09-01)

Nonviral delivery systems for antisense oligonucleotide therapeutics
od: Si Huang, i dr.
Izdano: (2022-09-01)

Chemistry of Therapeutic Oligonucleotides That Drives Interactions with Biomolecules
od: Chisato Terada, i dr.
Izdano: (2022-11-01)

Oligonucleotide Therapeutics: From Discovery and Development to Patentability
od: Lara Moumné, i dr.
Izdano: (2022-01-01)

Mesyl phosphoramidate oligonucleotides as potential splice-switching agents: impact of backbone structure on activity and intracellular localization
od: Hammond, SM, i dr.
Izdano: (2021)

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

Therapeutic approaches for Duchenne muscular dystrophy
od: Roberts, TC, i dr.
Izdano: (2023)

Regeneration mechanisms and therapeutic strategies for neuromuscular junctions in aging and diseases
od: Masashi Fujitani, i dr.
Izdano: (2025-01-01)

Editorial: Diagnosis, animal models and therapeutic interventions for neuromuscular diseases
od: Muthukumar Karuppasamy, i dr.
Izdano: (2024-09-01)