Leading RNA Interference Therapeutics Part 2: Silencing Delta-Aminolevulinic Acid Synthase 1, with a Focus on Givosiran

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

(2019) Eliane Sardh et al.   NEW ENGLAND JOURNAL OF MEDICINE

Leading RNA Interference Therapeutics Part 1: Silencing Hereditary Transthyretin Amyloidosis, with a Focus on Patisiran

(2019) Simoneide S. Titze-de-Almeida et al.   Molecular Diagnosis & Therapy

From a dominant to an oligogenic model of inheritance with environmental modifiers in acute intermittent porphyria

(2018) Hugo Lenglet et al.   HUMAN MOLECULAR GENETICS

Advanced siRNA Designs Further Improve In Vivo Performance of GalNAc-siRNA Conjugates

(2018) Donald J. Foster et al.   MOLECULAR THERAPY

Journey of siRNA: Clinical Developments and Targeted Delivery

(2018) Rahul R. Nikam et al.   Nucleic Acid Therapeutics

Porphyria

(2017) D. Montgomery Bissell et al.   NEW ENGLAND JOURNAL OF MEDICINE

Impact of enhanced metabolic stability on pharmacokinetics and pharmacodynamics of GalNAc–siRNA conjugates

(2017) Jayaprakash K. Nair et al.   NUCLEIC ACIDS RESEARCH

Chemistry, mechanism and clinical status of antisense oligonucleotides and duplex RNAs

(2017) Xiulong Shen et al.   NUCLEIC ACIDS RESEARCH

The Race of 10 Synthetic RNAi-Based Drugs to the Pharmaceutical Market

(2017) Ricardo Titze-de-Almeida et al.   PHARMACEUTICAL RESEARCH

Preclinical and Clinical Advances of GalNAc-Decorated Nucleic Acid Therapeutics

(2017) Yuanyu Huang   Molecular Therapy-Nucleic Acids

Synergistic effect of phosphorothioate, 5′-vinylphosphonate and GalNAc modifications for enhancing activity of synthetic siRNA

(2016) Thazha P. Prakash et al.   BIOORGANIC & MEDICINAL CHEMISTRY LETTERS

Update review of the acute porphyrias

(2016) Penelope E. Stein et al.   BRITISH JOURNAL OF HAEMATOLOGY

Porphyria and Vampirism—A Myth, Sensationalized

(2016) Eric Laurent Maranda et al.   JAMA Dermatology

siRNA Conjugates Carrying Sequentially Assembled Trivalent N-Acetylgalactosamine Linked Through Nucleosides Elicit Robust Gene Silencing In Vivo in Hepatocytes

(2015) Shigeo Matsuda et al.   ACS Chemical Biology

Discovery of the magnetic behavior of hemoglobin: A beginning of bioinorganic chemistry

(2015) Kara L. Bren et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

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

(2015) Amy Chan et al.   Molecular Therapy-Nucleic Acids

Liver transplantation in the management of porphyria

(2014) Ashwani K. Singal et al.   HEPATOLOGY

Multivalent N-Acetylgalactosamine-Conjugated siRNA Localizes in Hepatocytes and Elicits Robust RNAi-Mediated Gene Silencing

(2014) Jayaprakash K. Nair et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

RNAi-mediated silencing of hepatic Alas1 effectively prevents and treats the induced acute attacks in acute intermittent porphyria mice

(2014) M. Yasuda et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

The incidence of inherited porphyrias in Europe

(2012) George Elder et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Porphyrins: One Ring in the Colors of Life

(2011) Franck Dayan et al.   AMERICAN SCIENTIST

Synthesis and characterization of 2′-modified-4′-thioRNA: a comprehensive comparison of nuclease stability

(2009) Mayumi Takahashi et al.   NUCLEIC ACIDS RESEARCH

Trivalent, Gal/GalNAc-containing ligands designed for the asialoglycoprotein receptor

(2008) Oleg Khorev et al.   BIOORGANIC & MEDICINAL CHEMISTRY