Alpha-1 antitrypsin deficiency: current therapy and emerging targets.

Oisín F McElvaney,Daniel D Fraughen,Oliver J McElvaney,Tomás P Carroll,Noel G McElvaney

Expert review of respiratory medicine（2023）

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摘要

With a relatively small population able to participate in clinical studies, increased awareness and diagnosis of AATD is urgently needed. Better, more sensitive clinical parameters will assist in the generation of acceptable and robust evidence of therapeutic effect for current and emerging treatments.

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关键词

Alpha1 antitrypsin deficiency,COPD,cirrhosis,emphysema,gene editing,gene therapy,panniculitis,protein folding,siRNA

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