Money is needed to do the research we have done and will perform in the (near) future, for some in collaboration with other research groups. We can happily announce and thank different companies, funding organisations and parent organisations for their funding for the DMD Genetic Therapy group.


AFM-Téléthon

* Cross-sectional study to assess detailed natural disease history of limb girdle muscular dystrophy mouse models
* The use of spatial transcriptomics on cross sections of neuromuscular disorder patients
* Study to increase dystrophin levels after exon skipping by stabiliz-ing targeted transcripts
* Study to assess detailed natural history of humanized Duchenne mouse models with various deletions


Duchenne Parent Project

* Improving the 2’-O-methyl phosphorothioate induced exon skipping for Duchenne muscular dystrophy through chemical modification and/or a combination with added components: summary of the project (in Dutch)
* Clarifying the role and therapeutic effect of myostatin/ TGF-β receptors: summary of the project (in Dutch)
* Preparing for double exon skipping therapy
* Validating a mouse model to test human specific antisense oligonucleotides
* In-depth characterisation of brain pathology in mice lacking one or multiple brain dystrophin isoforms
* Extension of the validation lab
* Pilot study in mice with anti-oxidants and antisense oligonucleotides


Entrada Therapeutics

* Collaborative work on developing exon skipping compounds for Duchenne muscular dystrophy


NIH

* A consortium to advance biomarkers for Duchenne muscular dystrophy
* Biomarker research in neurological and neuromuscular diseases


Parent Project Muscular Dystrophy

* Study of serum biomarkers in Duchenne muscular dystrophy


Prinses Beatrix Spierfonds

* Improving exon skipping therapy for Duchenne muscular dystrophy
* mRNA as key to improve possible Duchenne muscular dystrophy therapies
* Double therapy for Duchenne muscular dystrophy


USA Department of Defense

* The evaluation of chemically modified antisense oligonucleotides for exon skipping in Duchenne muscular dystrophy


ZonMw

* Biomarkers in DMD: from the discovery to the development toward clinical application and translation in other NMDs (ENMC WS 204)
* Antisense therapy for several major rare diseases
* Study the function of different dystrophin isoforms in neuronally differentiated iPSCs and to what extent restoring these isoforms rescues deficits

Exon skipping | DMD Genetic Therapy group Leiden