Incurable muscle disease: First successes in the development of a gene therapy Researchers at the Biozentrum have developed a gene therapy that could potentially treat a rare and currently fatal muscle disease in children. The study shows in animal models that a single treatment...

🧪 Prof. Markus Rüegg’s team has achieved promising early results with a gene therapy for LAMA2-related muscular dystrophy – a rare and fatal muscle disease affecting children. The challenge now is to bring this therapy into the clinic. @unibas.ch @snsf.ch www.biozentrum.unibas.ch/news/detail/...

LAMA2-related muscular dystrophy is a rare genetic disease for which there is currently no treatment available.

We are happy to share our recent publication in Molecular Therapy, where we describe a unique linker-mediated gene therapy approach. doi.org/10.1016/j.ym...

Check out our latest publication in @NatureComms, where we show that activated muscle stem cells remodel their niche with laminin-α2, and that the loss of this process intrinsically impairs their regenerative capacity in LAMA2-related muscular dystrophy:

doi.org/10.1038/s414...

Q&A with Dr. Daniel Ham

We’re excited to launch our Research Highlight Series! First up: an in-depth Q&A with Dr. Daniel Ham from @ruegglab.bsky.social‬ on his latest @natcomms.nature.com‬ paper, and insights into muscle biology, research, and more. #MyoBlue

Read the interview
aurorascientific.com/research-hig...

In our latest preprint, we show that activated muscle stem cells secrete laminin-α2 into their niche — and that the loss of this process intrinsically impairs their regenerative capacity in LAMA2-related muscular dystrophy:

doi.org/10.1101/2025...

New insights challenge previous views about a muscle growth factor The protein Myc has long been thought to support skeletal muscle growth. Prof. Markus Rüegg's team at the Biozentrum provides compelling evidence that Myc in muscle fibers is dispensable for muscle gr...

New study by @ruegglab.bsky.social challenges previous view about the muscle growth factor #Myc! The protein is dispensable for #musclegrowth. High Myc levels disrupt muscle fiber structure & function. @unibas.ch @snsf-ch.bsky.social @naturecomms.bsky.social www.biozentrum.unibas.ch/news/detail/...

AAV capsids target muscle-resident cells with different efficiencies—A comparative study between AAV8, AAVMYO, and AAVMYO2 Although AAVs are the leading platform for gene delivery to skeletal muscle, the extent to which they transduce muscle-residing mononuclear cells remains unclear. Here, McGowan and colleagues address ...

If you're using AAVs to develop a gene therapy or to study gene function in skeletal muscle, then this may be of interest to you. In our latest work, we show that AAVs commonly used to deliver transgenes to muscle fibers also target mononuclear cells:

www.cell.com/molecular-th...

Single-nuclei sequencing of skeletal muscle reveals subsynaptic-specific transcripts involved in neuromuscular junction maintenance - Nature Communications Here they use single nuclei RNA-seq to identify transcripts in skeletal muscle that maintain the neuromuscular junction, both under normal and denervated conditions, which allows them to characterize ...

Check out our latest publication where we use single-nuclei RNA-seq to study gene expression at the neuromuscular junction: www.nature.com/articles/s41...

The paper also characterizes three novel NMJ proteins including the transcription factor ETV4 and the MuSK-binding protein PDZRN4.