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Scaffold hopping enables direct access to more potent PROTACs with in vivo activity.
In?vivo gene editing works in humans: Results of a phase 1 clinical trial for TTR amyloidosis.
Gene Therapy for Rare Neurological Disorders.
Senior Gene Therapy Scientists Take a Stand Against Human Embryo Gene Editing.
Lived Experience with Gene Therapy.
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Lived Experience with Gene Therapy.
Flotte TR. Lived Experience with Gene Therapy. Hum Gene Ther. 2023 Dec; 34(23-24):1178-1179.
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Terence R Flotte MD