Custom Antisense Oligonucleotide Aso Therapy For Retinal Disease In One Patient

Unveiling Antisense Oligonucleotide (ASO) Therapy: Prec...

Unveiling Antisense Oligonucleotide (ASO) Therapy: Prec... This research project entails delivery of a personalized antisense oligonucleotide (aso) drug designed for a single participant with retinal dystrophy due to prph2 mutation. We describe the pathogenic deep intronic mutation in the flvcr1 gene in a patient with posterior column ataxia with retinitis pigmentosa (pcarp), the development of an intravitreal custom antisense treatment, and the improved splicing ratio in patient derived cells treated with this aso.

Update On Neuroscience In The Clinic: Antisense Oligonucleotide (ASO ...

Update On Neuroscience In The Clinic: Antisense Oligonucleotide (ASO ... Since deep intronic mutations are potentially modifiable with aso therapy, a pathway to identify patients and a pipeline to therapy is important to scale these treatments. here we present the first creation of and treatment with a patient customized aso intravitreal therapy. Our findings demonstrate the potential utility of an aso based approach to treat retinal dysfunction in cln3 batten disease and generally supports the use of asos for treating eye diseases. This groundbreaking clinical trial represents a significant advancement in personalized medicine for retinal diseases. the study focuses on treating a single patient with retinal dystrophy caused by a specific genetic mutation in the prph2 gene using a custom designed antisense oligonucleotide (aso) therapy called nl prph2 001. Antisense oligonucleotide (aso) therapy holds promise in gene therapy but faces challenges due to poor delivery efficiency and limited evaluation models. this investigation employs magnetic nanoparticles (mnps) to augment the delivery efficiency of asos.

Antisense Oligonucleotides (ASO)

Antisense Oligonucleotides (ASO) This groundbreaking clinical trial represents a significant advancement in personalized medicine for retinal diseases. the study focuses on treating a single patient with retinal dystrophy caused by a specific genetic mutation in the prph2 gene using a custom designed antisense oligonucleotide (aso) therapy called nl prph2 001. Antisense oligonucleotide (aso) therapy holds promise in gene therapy but faces challenges due to poor delivery efficiency and limited evaluation models. this investigation employs magnetic nanoparticles (mnps) to augment the delivery efficiency of asos. Fortunately, improvements are on the horizon. sustained release formulations and nanoparticle systems offer promising approaches to improve efficacy and safety by optimising drug delivery and minimising the frequency of administration.4 ultimately, the ideal delivery method is determined by the unique properties of the aso, the targeted disease. In this study, we explored aon induced exon skipping as a potential treatment modality for patients with rp caused by mutations in exon 13 of the ush2a gene. The aso treatment resulted in higher amplitudes on electroretinograms, suggesting mitigation of retinal dysfunction at early timepoints of disease. one aso that efficiently induces exon skipping in vivo was well tolerated and targets a region conserved in humans, making it a promising candidate for clinical translation. Areas covered: we review the current status of clinical trials of aso therapies for inherited retinal diseases, which have demonstrated safety, viable durability, and early efficacy.

Custom Antisense Oligonucleotide (ASO) Therapy for Retinal Disease in One Patient