Abstract Highlights New Preclinical Data on Exosome-Based Delivery of PMOs for the Treatment of DMD-SAN DIEGO, Nov. 12, 2024 ...

Capricor presented preclinical data at the 2024 American Association of Extracellular Vesicles (AAEV) Annual Meeting ...

Sarepta Therapeutics has ended development of a late-phase Duchenne muscular dystrophy (DMD) drug candidate over a safety problem. The safety signal deprived Sarepta of a near-term shot at accelerated ...

SRP-5051 is designed to bind to exon 51 of dystrophin pre-mRNA, resulting in exclusion of this exon during mRNA processing in patients with genetic mutations amenable to exon 51 skipping. With this ...

23 Antisense oligonucleotide (ASO) knockdown: Human FLS were grown to 90% confluence and treated with 2.5 µM morpholino antisense oligonucleotides (ASO) (Gene Tools). ASO was replenished in fresh ...

After that, 2 mL mixed buffer (pH = 4.5) containing 15 mM 1-ethyl-3-[3-dimethylaminopropyl] carbodiimide hydrochloride (EDC), 25 mM N-hydroxysulfosuccinimide (NHS), 100 mM 2-(N-morpholino) ethane ...

Good day, and welcome to the Sarepta Therapeutics third quarter 2024 for financial results conference call. After the speakers' presentation, there will be a question-and-answer session. [Operator ...

Erratum: Prospective External Validation of the Esbenshade Vanderbilt Models Accurately Predicts Bloodstream Infection Risk in Febrile Non-Neutropenic Children With Cancer Twenty-four of 58 adults (41 ...

Department of Genetics, Graduate School of Pharmaceutical Sciences, University of Tokyo, Tokyo, Japan In this review, we provide an overview of the current understanding of apoptotic signaling that ...