Professor Megumu K. Saito and his colleagues have revealed that SMN, a protein involved in spinal muscular atrophy (SMA), promotes the functional maturation of mitochondria by regulating miRNA expression along with MYOD1 ...
Feb 17, 2023
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Cedars-Sinai's Regenerative Medicine Institute has pioneered research on how motor-neuron cell-death occurs in patients with spinal muscular atrophy, offering an important clue in identifying potential medicines to treat ...
Jun 20, 2012
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Neuroscientists at Lund University in Sweden have developed a new technology that engineers the shell of a virus to deliver gene therapy to the exact cell type in the body that needs to be treated. The researchers believe ...
Dec 13, 2019
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Spinal muscular atrophy (SMA), the most common form of neurodegenerative disease in childhood, is caused by defects in the SMN1 gene. SMA patients potentially benefit from three recently approved disease-modifying therapies. ...
Nov 7, 2022
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As British lawmakers vote Tuesday on whether to legalize the creation of babies made from the DNA of three people, here are some questions and answers about the proposed techniques and the controversy surrounding them.
Feb 3, 2015
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Body image issues, social media, gender biases and coaching styles may be causing young athletes to quit sports, according to research presented by Nemours Children's Health at the 2023 American Academy of Pediatrics (AAP) ...
Oct 20, 2023
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Researchers at Boston University College of Health & Rehabilitation Sciences: Sargent College have identified a combinatorial therapeutic approach that has proven effective in treating muscular dystrophy in a mouse model. ...
Aug 2, 2013
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Scientists at Royal Holloway, University of London, along with colleagues at UCL, have developed a novel artificial human chromosome which could be used to reverse the genetic defect in muscle stem cells from patients with ...
Jan 25, 2018
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Early use of available heart failure drugs slows the progressive decline in heart function before symptoms are apparent in boys and young men with Duchenne muscular dystrophy (DMD), according to a new study published online ...
Dec 29, 2014
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Scientists in The Research Institute at Nationwide Children's Hospital have found a way to overcome one of the biggest obstacles to using viruses to deliver therapeutic genes: how to keep the immune system from neutralizing ...
Oct 30, 2013
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