Novel gene therapy offers potential treatment for FCMD
Japanese researchers proposed using RNA interference for correcting a mistake in the genetic code of patients with Fukuyama Muscular Dystrophy (FCMD).
Using RNA interference, Japanese researchers from the Department of Clinical Genetics at Fujita Health University Hospital, restored the normal biological function of “fukutin” (FKTN) gene in patient-derived cells, offering hope for therapy.
Fukuyama congenital muscular dystrophy (FCMD) is the second most common form of childhood muscular dystrophy in Japan. The disease manifests itself in the form of a severe neuromuscular disorder. One form of FCMD is caused by a genetic abnormality in the FKTN gene. The genetic anomaly blocks the chemical glycosylation of a biologically important protein.
The findings, published in Human Molecular Genetics, used the experimental technique called exon skipping by antisense oligonucleotides. The team corrected a mistake in the FKTN gene that blocks the chemical glycosylation of a biologically important protein. Thus, the team designed specific antisense oligonucleotides—small pieces of DNA or RNA that can bind to specific RNA molecules.
The researchers then conducted experiments on patient-derived cells using these antisense oligonucleotides.
https://www.drugtargetreview.com/news/107272/novel-gene-therapy-offers-potential-treatment-for-fcmd/
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