Recent advances in antisense oligonucleotide therapy in genetic neuromuscular diseases

Verma, Ashok

doi:10.4103/aian.aian_298_17

Cited by 32 publications

(3 citation statements)

References 43 publications

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“…Fomiversen, an antiviral, was the first ASO to receive FDA approval for the treatment of cytomegalovirus retinitis in 1998 [20]. Following a 15-year lull, mipomersen also received FDA approval in 2013 for the treatment of familial hypercholesterolemia [21,22]. Since then, the rate and number of ASOs under development have increased dramatically, and numerous ASOs have received FDA approval in the last decade: eteplirsen (DMD-2016), nusinersen (spinal muscular atrophy-2016), inotersen (hereditary transthyretin-mediated amyloidosis-2018), golodirsen (DMD-2019), viltolarsen (DMD-2020), casimersen (DMD-2021), and tofersen (amyotrophic lateral sclerosis-2023) [23][24][25][26][27][28][29].…”

Section: Antisense Therapymentioning

confidence: 99%

Preparing for Patient-Customized N-of-1 Antisense Oligonucleotide Therapy to Treat Rare Diseases

Wilton-Clark,

Yan,

Yokota

2024

Genes

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The process of developing therapies to treat rare diseases is fraught with financial, regulatory, and logistical challenges that have limited our ability to build effective treatments. Recently, a novel type of therapy called antisense therapy has shown immense potential for the treatment of rare diseases, particularly through single-patient N-of-1 trials. Several N-of-1 antisense therapies have been developed recently for rare diseases, including the landmark study of milasen. In response to the success of N-of-1 antisense therapy, the Food and Drug Administration (FDA) has developed unique guidelines specifically for the development of antisense therapy to treat N-of-1 rare diseases. This policy change establishes a strong foundation for future therapy development and addresses some of the major limitations that previously hindered the development of therapies for rare diseases.

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Section: Antisense Therapymentioning

confidence: 99%

Preparing for Patient-Customized N-of-1 Antisense Oligonucleotide Therapy to Treat Rare Diseases

Wilton-Clark,

Yan,

Yokota

2024

Genes

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“…Different chemical modifications have been tested to give the AONs drug-like characteristics in trying to enhance their bioavailability, improving their resistance to exonucleases and endonucleases, and increasing their affinity to the target RNA transcripts ( Saleh et al, 2012 ). 2ʹ-O-methyl phosphorothioate (2OMePS) AONs and phosphorodiamidate morpholino oligomers (PMOs) are used among other chemistries for DMD exon skipping clinical development approaches ( Verma, 2018 ; Schneider and Aartsma-Rus, 2021 ).…”

Section: Therapeutic Strategies For Dmdmentioning

confidence: 99%

Duchenne muscular dystrophy: disease mechanism and therapeutic strategies

et al. 2023

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Duchenne muscular dystrophy (DMD) is a severe, progressive, and ultimately fatal disease of skeletal muscle wasting, respiratory insufficiency, and cardiomyopathy. The identification of the dystrophin gene as central to DMD pathogenesis has led to the understanding of the muscle membrane and the proteins involved in membrane stability as the focal point of the disease. The lessons learned from decades of research in human genetics, biochemistry, and physiology have culminated in establishing the myriad functionalities of dystrophin in striated muscle biology. Here, we review the pathophysiological basis of DMD and discuss recent progress toward the development of therapeutic strategies for DMD that are currently close to or are in human clinical trials. The first section of the review focuses on DMD and the mechanisms contributing to membrane instability, inflammation, and fibrosis. The second section discusses therapeutic strategies currently used to treat DMD. This includes a focus on outlining the strengths and limitations of approaches directed at correcting the genetic defect through dystrophin gene replacement, modification, repair, and/or a range of dystrophin-independent approaches. The final section highlights the different therapeutic strategies for DMD currently in clinical trials.

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“…In this approach, the oligonucleotide payload is covalently attached to a carrier or ligand, such as lipid particles, liposomes, nanoparticles, and more recently, the sugar N‐acetyl galactosamine. This enhances the safe and efficient delivery of ASOs to the intended target sites (Verma, 2018). They feature nucleotide modifications, particularly at the furanose ring.…”

Section: Introduction To Antisense Oligonucleotides (Asos): Mechanism...mentioning

confidence: 99%

Unveiling the potential of antisense oligonucleotides: Mechanisms, therapies, and safety insights

Ersöz,

Demir‐Dora

2024

Drug Development Research

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Antisense oligonucleotides (ASOs) are short, synthetic, single‐stranded deoxynucleotide sequences composed of phosphate backbone‐connected sugar rings. Designing of those strands is based on Watson‐Crick hydrogen bonding mechanism. Thanks to rapidly advancing medicine and technology, evolving of the gene therapy area and ASO approaches gain attention. Considering the genetic basis of diseases, it is promising that gene therapy approaches offer more specific and effective options compared to conventional treatments. The objective of this review is to explain the mechanism of ASOs and discuss the characteristics and safety profiles of therapeutic agents in this field. Pharmacovigilance for gene therapy products is complex, requiring accurate assessment of benefit‐risk balance and evaluation of adverse effects.

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Recent advances in antisense oligonucleotide therapy in genetic neuromuscular diseases

Cited by 32 publications

References 43 publications

Preparing for Patient-Customized N-of-1 Antisense Oligonucleotide Therapy to Treat Rare Diseases

Preparing for Patient-Customized N-of-1 Antisense Oligonucleotide Therapy to Treat Rare Diseases

Duchenne muscular dystrophy: disease mechanism and therapeutic strategies

Unveiling the potential of antisense oligonucleotides: Mechanisms, therapies, and safety insights

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