Tag: <span>mutant ALS gene</span>

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UMass Chan clinical trial shows antisense oligonucleotide safely suppresses mutant ALS gene in pilot human study

UNIVERSITY OF MASSACHUSETTS MEDICAL SCHOOL WORCESTER, Mass. – 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 the ability to suppress mutant forms of an ALS gene known as C9ORF72 in...