At least 88 people, including children, are being treated in hospital. The government announced a day of mourning, with flags at half-mast and special prayers at all places of worship.

A third patient, a 51 year-old in a Phase I study of SRP-9004 for Limb-Girdle muscular dystrophy Type 2E, has died from a Sarepta gene therapy.Like two dea...

Buckley and colleagues report a strategy for in situ imaging of mRNA-lipid nanoparticle vaccines in live animals. Lipid nanoparticles were observed in muscle tissue and draining lymph nodes, with stoc...

Biomolecules morph between conformations with distinct lifetimes essential for function. This study reveals the cores of flaviviral RNAs stay stable up to ten million times longer than canonical base ...

Dysferlinopathy is a progressive muscular disorder caused by the deficiency of dysferlin, a protein crucial for muscle membrane repair. Until now, there has been no effective treatment, significantly ...

Anwar and colleagues have demonstrated that antisense oligonucleotides targeting DYSF exon 27 effectively restore functional dysferlin protein in patient-derived muscle cells with dysferlinopathy. The...

Lack of muscle stem cell targeting limits the treatment of muscular dystrophies. Here, the authors describe a platform based on functionalized gold nanoparticles that allows selective delivery of ther...

Dysferlin-deficient muscular dystrophy is a devastating and untreatable disease. Using Cas9, the authors restored dysferlin in muscle stem cells from patients ex vivo and show proof-of-concept for aut...

Antisense oligonucleotides effectively decrease the inclusion of exon  8A of CACNA1C in human cells both in vitro and in rodents transplanted with human brain organoids, and a single intrath...

When formulating mRNA into lipid nanoparticles (LNP), various copy numbers of mRNA are encapsulated, leading to a distribution of mRNA loading levels within the LNPs. It is unclear whether the mRNA loading level affects the functional delivery of the message. Here we show that depending on the mRNA loading level, LNPs exhibit distinct mass densities and can be fractionated via ultracentrifugation. Upon fractionation, we investigated if mRNA loading levels influence LNP sizing, lipid composition, and morphology. We further conducted in vitro and in vivo functional delivery of mRNA and found that the LNP fraction with the highest mRNA loading levels was the least transfection competent.

A mutant gene is coming to steal Linde Jacobs’s mind. Can she find a way to stop it?

YouTube video by UC Davis Health

FDA has approved Tryngolza (olezarsen), used with diet, to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS).

FDA approves Ryoncil, the first MSC therapy to treat steroid-refractory acute graft-versus-host disease (SR-a GVHD) in pediatric patients 2 months of age and ol

An articular-cavity enriched lipid nanoparticle (LNP) formulation is optimized, and a rhFGF18 mRNA sequence for osteoarthritis (OA) therapy is designed. Utilizing the LNP-rhFGF18 mRNA complex via int...

New DNA-like molecule treatment for children with Fibrodysplasia ossificans progressiva Fibrodysplasia ossificans progressiva (FOP) is a rare genetic disorder causing children’s muscles and connective...

Last week, I attended the Breakthroughs in Muscular Dystrophies Conference, a brand-new event jointly organized by the American Society of Gene and Cell Therapy (ASGCT) and the Muscular Dystrophy Asso...