Using a short, synthetic chain of chemically modified nucleotides engineered in the RNA Therapeutics Institute at UMass Chan Medical School, Robert H. Brown Jr., DPhil, MD, Jonathan Watts, Ph.D., and colleagues have shown ...
Dec 23, 2021
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In a new study published in Brain Prof. Xi Chen from Nanjing University and Prof. Zhong Pei from Sun Yat-sen University report the therapeutic reversal of Huntington's disease by in vivo self-assembled siRNAs.
Dec 22, 2021
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Alexander disease is a progressive and rare neurological disorder with no cure or standard course of treatment. But a new study led by researchers at the University of Wisconsin–Madison involving a rat model of the disease ...
Nov 17, 2021
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Skoltech researchers and their colleagues from Russia and the UK investigated the safety and efficacy of new chemistry in antisense oligonucleotides used to treat spinal muscular atrophy (SMA), a debilitating genetic disease. ...
Jun 29, 2021
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Scientists from Nanjing University and University of Macau have discovered nano-scaled apoptotic bodies (ABs) as a new brain-targeting drug carrier, bringing new promise for the Parkinson's Disease as well as other brain ...
May 7, 2021
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Nagoya University researchers and colleagues in Japan have uncovered a molecular pathway that enhances chemotherapy resistance in some pancreatic cancer patients. Targeting an RNA to interrupt its activity could improve patient ...
Apr 21, 2021
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Many patients with multiple myeloma, a type of blood cancer, eventually develop resistance to one treatment after another. That's in part because cancer stem cells drive the disease—cells that continually self-renew. If ...
Jan 20, 2021
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Adding exercise to a genetic treatment for myotonic dystrophy type 1 (DM1) was more effective at reversing fatigue than administering the treatment alone in a study using a mouse model of the disease. In fact, exercise alone ...
Nov 30, 2020
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Seizure disorders in babies are frightening and heartbreaking. A new basic science breakthrough offers hope for a potential treatment for rare developmental and epileptic encephalopathies resulting from a single genetic mutation. ...
Feb 20, 2020
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Duchenne muscular dystrophy occurs in boys and is characterized by progressive muscle degeneration and weakness leading to a decline in respiratory function. Strategies to arrest this severe progressive deterioration are ...
Jul 9, 2019
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