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Vivet wins $5.3m grant to develop gene therapy for rare metabolic disorder
The AAV vector gene therapy targets the ATP7B gene, responsible for causing the condition. It is currently being investigated in an open-label Phase I/II GATEWAY trial (NCT04537377), that has a ...
The Telegraph1mon
Telomir Pharmaceuticals Confirms Copper Binding Capabilities of Telomir-1 and Expands Pipeline Into Wilson’s Disease
The condition is caused by mutations in the ATP7B gene, which impair the body's ability to properly regulate copper metabolism. As a result, copper accumulates to toxic levels, particularly in the ...
Nature9d
Wilson's Disease Research
This condition is caused by mutations in the ATP7B gene, which is responsible for copper transport. Recent research has focused on improving the understanding of Wilson's disease through ...