Phase 1 Trial of an RNA Interference Therapy for Acute Intermittent Porphyria

Eliane Sardh,Pauline Harper,Manisha Balwani,Penelope E. Stein,David Rees,D. Montgomery Bissell,Robert J. Desnick,Charles J. Parker,John D. Phillips,Herbert L. Bonkovsky,Daphne Vassiliou,Craig Penz,Amy Chan-Daniels,Qiuling He,William Querbes,Kevin Fitzgerald,Jae B. Kim,Pushkal P. Garg,Akshay Vaishnaw,Amy Simon,Karl E. Anderson

Phase 1 Trial of an RNA Interference Therapy for Acute Intermittent Porphyria

2019

Abstract Background Induction of delta aminolevulinic acid synthase 1 (ALAS1) gene expression and accumulation of neurotoxic intermediates result in neurovisceral attacks and disease manifestations...

Keywords:

Surgery
Gastroenterology
RNA interference
Diabetes mellitus
Acute intermittent porphyria
Internal medicine
Medicine
Disease
ATP synthase
Gene expression
ALAS1
Delta Aminolevulinic Acid
Pharmacology

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