UMass Chan clinical trial shows antisense oligonucleotide safely suppresses mutant ALS gene in pilot human study - UMass Medical School
Robert H. Brown Jr., DPhil, MD, Jonathan Watts, PhD, and colleagues have shown the ability to suppress mutant forms of an ALS gene in a single-patient pilot study.
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, PhD, and c… [+7150 chars]
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