New research published in the Journal of Chromatography A examines the effect of flow rate on plate height and resolution for antisense oligonucleotides (ONs) under hydrophilic interaction liquid ...

Sarepta Therapeutics has followed through on its promise to file for accelerated approval of its gene therapy SRP-9001 for Duchenne muscular dystrophy (DMD), as it aims for a launch in the ...

Get Instant Summarized Text (Gist) A new approach using personalized antisense oligonucleotides (ASOs) and patient-derived organoids offers promising treatment options for rare genetic diseases ...

NEW YORK – Belief BioMed announced Wednesday that the US Food and Drug Administration has cleared its investigational new drug application enabling it to begin testing its gene therapy candidate for ...

Duchenne muscular dystrophy, limb girdle muscular dystrophy R2, epilepsy and other inflammatory diseases. It is administered through subcutaneous, inhalation route of administration. It is formulated ...

Pfizer meanwhile has had other scares about the safety of its gene therapy, including most recently reports of muscle weakness and myocarditis in some patients who received the gene therapy, which ...

In this study, the team, which includes Dwight Stoll, the "LC Troubleshooting" columnist for LCGC International, worked to determine the quality antisense ONs (ASOs) attributes, such as purity, ...

Learn More Genetic Causes of Muscular Dystrophy Is muscular dystrophy fatal? Some types of muscular dystrophy can be fatal. Certain types, including Duchenne muscular dystrophy, can lead to problems ...

Antisense Oligonucleotide: A short strand of nucleic acid that can bind to RNA and inhibit the expression of specific genes, used in gene therapy to target genetic mutations. This Nature Research ...

They investigated the effects of antisense oligonucleotides targeting the regulatory RIα subunit of cAMP-dependent protein kinase (PKA). They treated human PC3 prostate cancer cells with antisense ...

Similar results were observed with an ASO targeting a different region of dystrophin that is currently being used in a phase II clinical trial for Duchenne muscular dystrophy. Furthermore ...