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Small molecules for regenerative medicine
In this episode, we discuss how new insights into stem cell biology are leading to novel therapeutics in regenerative medicine beyond cell therapy.
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Duchenne muscular dystrophy (DMD)
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Developing an mRNA therapy for Duchenne muscular dystrophy
In this Q&A, Aki Ko, CEO and co-founder of Elixirgen Therapeutics, elucidates how their new mRNA technology could potentially restore muscle function in those suffering from Duchenne muscular dystrophy.
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How engineered RNA binding proteins delivered in vivo by gene therapy could treat myotonic dystrophy
In this article, Dr Jim Burns discusses promising pre-clinical results of how a new platform could treat the root cause of many devastating genetic diseases including myotonic dystrophy type 1.
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Novel CRISPR strategy developed for Duchenne muscular dystrophy
A CRISPR gene editing technique has been developed to restore dystrophin, which is missing in many Duchenne muscular dystrophy (DMD) patients.
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Chemical cocktail creates new avenues for generating muscle stem cells
A combination of chemicals has shown promise in pre-clinical studies to produce muscle stem cells, giving hope for regenerative medicine.
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CRISPR-based treatment successfully targets glioblastoma and ovarian cancers in vivo
Researchers say this is the first time that CRISPR-Cas9 gene editing has been used to treat cancer effectively in a living animal and that the technique could be revolutionary.
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Researchers target previously “undruggable” enzyme to treat Duchenne muscular dystrophy in cells
By targeting a previously undiscovered allosteric site on a DMD-associated enzyme, researchers found muscle cell conditions improved.
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Complete DNA sequence of the X chromosome revealed
Researchers capitalised on novel sequencing technologies to produce the first end-to-end DNA sequence of the X chromosome.
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Experimental mouse model of Duchenne muscular dystrophy developed
Researchers have created an accurate mouse model of Duchenne muscular dystrophy (DMD), showing that TGFβ is a potential target for the condition.
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Utrophin as a novel therapeutic approach for Duchenne muscular dystrophy
Upregulating utrophin using small molecules could be a new therapeutic approach for Duchenne muscular dystrophy (DMD).
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Gene therapy for DMD safely preserves muscle function
Gene therapy for the treatment of Duchenne muscular dystrophy has safely stopped the muscle deterioration associated with the disease.
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Researchers find DNA therapy could treat patients with DMD
A study has revealed that using DNA-like molecules to repair gene mutations in models could act as a successful therapy for patients.
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Inhibiting NF-κB improves heart function in Duchenne muscular dystrophy
Breakthrough research has uncovered an unexpected mechanism that underlies cardiomyopathy in DMD...
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BDNF protein could be key to preserving heart function in DMD
A protein known to drive nerve cell survival in the brain and spinal cord may also protect failing hearts in children and young adults with DMD...
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CRISPR method efficiently corrects DMD defect in heart tissue
Scientists have developed a CRISPR gene-editing technique that can potentially correct a majority of mutations that cause DMD...