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Two patients with a rare inherited disease are showing early signs of improvement after being injected with a groundbreaking genome-editing fix from Sangamo Therapeutics.

That’s the encouraging news emphasized by the biotech on Wednesday from its latest clinical trial, the first in which gene-editing is being used in patients to try to repair disease-causing DNA. But Sangamo is simultaneously downplaying other results from the study that are more confounding and may give outsiders reason to be concerned.

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  • My daughter has a rare disease called Dermatomyositis. She is 8 years old. Do you have anything that could help her?

  • To which person (and company to whom this person is affiliated) has the Court of Appeals “awarded” the Foundational Crown Patent for the “Gene Editing Process, cas 9” ? To Ms. Doudna of Intellia, Inc., Ms. Charpentier of Crisper Therapeutics AG, or Mr. Zhang of Editas Medicine, Inc. and on what date ?

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