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History (145)
Gender differences in risk factors for cigarette smoking initiation in childhood.
The 2024 Nobel Prize: Impact of the Discovery of miRNA on the Field of Gene Therapy.
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.
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Gene Therapy for Rare Neurological Disorders.
Flotte TR, Gessler DJ. Gene Therapy for Rare Neurological Disorders. Clin Pharmacol Ther. 2022 04; 111(4):743-757.
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PubMed
subject areas
Gene Editing
Genetic Therapy
Genetic Vectors
Humans
Muscular Atrophy, Spinal
Mutation
authors with profiles
Terence R Flotte MD
Dominic J Gessler MD, PhD