Medical research

RNA editing technique treats severe form of muscular dystrophy

An RNA editing technique called "exon skipping" has shown preliminary success in treating a rare and severe form of muscular dystrophy that currently has no treatment, based on a new study from Northwestern Medicine and the ...

Medical research

Understudy gene offers hope for spinal muscular atrophy

I began writing about genetics decades ago, and the best thing about getting older is witnessing the development of targeted treatments for single-gene diseases that I never thought would happen. But it is happening, for ...

Medical research

iPS cells discover drug target for muscle disease

Duchenne muscular dystrophy (DMD) is a muscular disease that shows symptoms in early childhood and causes progressive atrophy and eventual death. There is little in terms of treatment, partly because of poor understanding ...

Medical research

Using low-dose irradiation, researchers can now edit human genes

For the first time, researchers have employed a gene-editing technique involving low-dose irradiation to repair patient cells, according to a study published in the journal Stem Cells Translational Medicine. This method, ...

Medical research

New stem cell research uncovers causes of spinal muscular atrophy

New research from the Advanced Gene and Cell Therapy Lab at Royal Holloway, University of London has used pioneering stem cell techniques to better understand why certain cells are more at risk of degenerating in spinal muscular ...

page 38 from 40