Researchers at Iowa State University have identified an RNA structure in humans that could lead to a new treatment for spinal muscular atrophy, the leading genetic cause of death in babies and young children.
Jul 19, 2013
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Adopting some of the strategies behind successfully treating the childhood disease spinal muscular atrophy may enable development of therapies to curb the muscle decline that accompanies aging, new research suggests.
Nov 15, 2022
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A study by scientists from the Motor Neuron Center at Columbia University Medical Center (CUMC) suggests that spinal muscular atrophy (SMA), a genetic neuromuscular disease in infants and children, results primarily from ...
Oct 11, 2012
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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, ...
Jul 22, 2015
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Spinal muscular atrophy, or SMA, is the leading genetic cause of infant death. Less than a decade ago, Cold Spring Harbor Laboratory (CSHL) Professor Adrian Krainer showed this brutal disease can be treated by tweaking a ...
Mar 6, 2024
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The advent of therapeutic interventions for spinal muscular atrophy (SMA) has increased the importance of presymptomatic diagnosis and treatment. When to start treatment in children with less severe disease remains controversial. ...
Jun 11, 2020
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Today the journal Science published results of a preclinical study demonstrating that treatment with orally available RNA splicing modifiers of the SMN2 gene starting early after birth is preventing deficits in a mouse model ...
Aug 7, 2014
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A study by scientists at the Motor Neuron Center at Columbia University Medical Center suggests that spinal muscular atrophy (SMA), a genetic neuromuscular disease in infants and children, results primarily from problems ...
Nov 20, 2012
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NYU biologists have created model neurons with greater precision and efficiency than have been achieved in the past. Their breakthrough, which appeared this fall in a pair of papers in the journal Nature Neuroscience, has ...
Dec 12, 2013
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Mutations can be good and bad. Sometimes they help an organism adapt and survive. Other times they are so harmful that an organism can't survive or reproduce. Cold Spring Harbor Laboratory (CSHL) Professor Adam Siepel's team ...
Sep 22, 2022
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