Precision Biosciences Inc. recently presented a new gene-editing approach, PBGENE-DMD, which could allow life-long benefits to patients with Duchenne muscular dystrophy (DMD).
Researchers from Edgewise Therapeutics Inc. have previously developed and characterized a novel bmx mouse model of Becker muscular dystrophy (BMD), which harbors a deletion of murine Dystrophin exons 45-47 on a C57/BL6J background.
Prostaglandins induce the regeneration of muscle in rodents and humans through the prostaglandin E2 receptor EP4 subtype receptor, but this therapeutic pathway's potential is limited due to systemic tolerability. Researchers from Mesentech Inc. recently presented new results on their prostaglandin E2 receptor EP4 subtype receptor agonist irodanoprost trying to address this limitation issue.
Branchio-Oto-Renal syndrome 1 (BOR1) is caused by pathogenic variants in the EYA1 gene, and the gene behind the pathogenesis of BOR2 is SIX5. Growing evidence exists regarding GATA and PAX-SIX-EYA-DACH transcriptional networks playing a key role in normal development. A case report of a patient harboring a new variant in the DACH1 gene was recently presented.
Researchers from Suzhou Genassist Therapeutics Co. Ltd. have developed a novel canine model of Duchenne muscular dystrophy (DMD), expected to be more predictive of disease pathogenesis and treatment efficacy.
Researchers from Anew Therapeutics Pte Ltd. recently detailed the discovery of novel oral IL-17A small-molecule inhibitors as candidates for the treatment of psoriasis.
Charcot-Marie-Tooth disease type 2E (CMT2E) is a slow and progressive neuropathy characterized by axonal dysfunction. Its clinical phenotype includes muscle weakness and atrophy, sensory loss and reduced nerve conduction velocity, among others.
Mosaic variegated aneuploidy syndrome (MVAS) is an autosomal recessive disorder characterized by mosaic aneuploidy. Its clinical manifestations include growth and developmental delay, congenital malformations and increased cancer risk. Genetic variants involved in MVAS affect the chromosomal segregation during mitosis, where individuals often show mosaicism and chromosomal instability.
Atopic dermatitis (AD) is mainly triggered by immune dysregulation, barrier dysfunction and inflammation propagation, and chronic itching increases the susceptibility to infections.
Investigators from Insmed Inc. have presented new preclinical data on the efficacy of their adenoviral vector (AAV9)-based gene therapy INS-1201 for the treatment of Duchenne muscular dystrophy (DMD).
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