Preclinical Development of a Subcutaneous ALAS1 RNAi Therapeutic for Treatment of Hepatic Porphyrias Using Circulating RNA Quantification

Author:

Chan Amy,Liebow Abigail,Yasuda Makiko,Gan Lin,Racie Tim,Maier Martin,Kuchimanchi Satya,Foster Don,Milstein Stuart,Charisse Klaus,Sehgal Alfica,Manoharan Muthiah,Meyers Rachel,Fitzgerald Kevin,Simon Amy,Desnick Robert J,Querbes William

Publisher

Elsevier BV

Subject

Drug Discovery,Molecular Medicine

Reference36 articles.

1. Disorders of heme biosynthesis: X linked sideroblastic anemia and the porphyrias;Anderson,2001

2. Induction of the synthesis of delta-aminolevulinic acid synthetase in liver parenchyma cells in culture by chemical that induce acute porphyria;Granick;J Biol Chem,1963

3. Disturbances of liver porphyrin metabolism caused by drugs;de Matteis;Pharmacol Rev,1967

4. Steroid induction of delta-aminolevulinic acid synthase and porphyrins in liver. Structure-activity studies and the permissive effects of hormones on the induction process;Sassa;J Biol Chem,1979

5. The effect of progesterone on activities of delta-aminolevulinic acid synthetase and delta-aminolevulinic acid dehydratase in estrogen-primed avian oviduct;Miller;Gen Comp Endocrinol,1974

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