Survey of Health Care Provider Understanding of Gene Therapy Research for Inherited Metabolic Disorders

Joyanna Hansen,Radha Ramachandran,Jerry Vockley

Clinical Therapeutics（2022）

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摘要

There is an urgent need for education on topics related to gene therapy modalities. Professional education on gene therapies is desired across all specialties and will be important for unified treatment practices in IMD care.

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关键词

gene therapy,gene transfer,inborn errors of metabolism,inherited metabolic disorders,phenylketonuria,survey

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