This repository contains the R scripts for bulk RNAseq of lung biopsies from mice infected with Pseudomonas aeruginosa with/without anti-ANGPTL4 ASO treatment. The study aims to establishes ...

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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 ...

Discover the importance of vision, genetic eye disorders, and RNA-based therapies for precision treatment in India and ...

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 ...

Current work is focussing on regulating splicing of the SMN2 gene in spinal muscular atrophy and antisense-oligonucleotide-mediated exon skipping in Duchenne Muscular Dystrophy. We are also ...

(IN BRIEF) Roche has reported positive results from the second year of the EMBARK Phase III trial of Elevidys (delandistrogene moxeparvovec), the first approved gene therapy ... spinal muscular ...

The companies have teamed up to use Alloy’s AntiClastic Antisense platform. Credit: Bevan Goldswain via Getty Images. Sanofi has entered into a collaboration and license agreement with Alloy ...

Pfizer is “evaluating appropriate next steps” for its Duchenne muscular dystrophy (DMD) gene therapy after it failed a phase 3 trial. Fordadistrogene movaparvovec (PF-06939926) was unable to ...