Preclinical evaluation of RNAi as a treatment for transthyretin-mediated amyloidosis

Evolving landscape in the management of transthyretin amyloidosis

(2015) Philip N. Hawkins et al.   ANNALS OF MEDICINE

A novel mechano-enzymatic cleavage mechanism underlies transthyretin amyloidogenesis

(2015) J. Marcoux et al.   EMBO Molecular Medicine

An RNAi therapeutic targeting antithrombin to rebalance the coagulation system and promote hemostasis in hemophilia

(2015) Alfica Sehgal et al.   NATURE MEDICINE

Liver Transplantation for Hereditary Transthyretin Amyloidosis

(2015) Bo-Göran Ericzon et al.   TRANSPLANTATION

Repurposing Diflunisal for Familial Amyloid Polyneuropathy

(2015) John L. Berk et al.   JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION

Nomenclature 2014: Amyloid fibril proteins and clinical classification of the amyloidosis

(2014) Jean D. Sipe et al.   AMYLOID-JOURNAL OF PROTEIN FOLDING DISORDERS

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

Liver as a target for oligonucleotide therapeutics

(2013) Alfica Sehgal et al.   JOURNAL OF HEPATOLOGY

Effect of an RNA interference drug on the synthesis of proprotein convertase subtilisin/kexin type 9 (PCSK9) and the concentration of serum LDL cholesterol in healthy volunteers: a randomised, single-blind, placebo-controlled, phase 1 trial

(2013) Kevin Fitzgerald et al.   LANCET

Safety and Efficacy of RNAi Therapy for Transthyretin Amyloidosis

(2013) Teresa Coelho et al.   NEW ENGLAND JOURNAL OF MEDICINE

Influence of Polyethylene Glycol Lipid Desorption Rates on Pharmacokinetics and Pharmacodynamics of siRNA Lipid Nanoparticles

(2013) Barbara L Mui et al.   Molecular Therapy-Nucleic Acids

Maximizing the Potency of siRNA Lipid Nanoparticles for Hepatic Gene Silencing In Vivo

(2012) Muthusamy Jayaraman et al.   ANGEWANDTE CHEMIE-INTERNATIONAL EDITION

Transthyretin (TTR) Cardiac Amyloidosis

(2012) Frederick L. Ruberg et al.   CIRCULATION

Diagnosis and Therapeutic Approaches to Transthyretin Amyloidosis

(2012) Y. Ando et al.   CURRENT MEDICINAL CHEMISTRY

Amyloid neuropathies

(2012) David Adams et al.   CURRENT OPINION IN NEUROLOGY

The Transthyretin Amyloidoses: From Delineating the Molecular Mechanism of Aggregation Linked to Pathology to a Regulatory-Agency-Approved Drug

(2012) Steven M. Johnson et al.   JOURNAL OF MOLECULAR BIOLOGY

Potential Use of Lactosylated Dendrimer (G3)/α-Cyclodextrin Conjugates as Hepatocyte-Specific siRNA Carriers for the Treatment of Familial Amyloidotic Polyneuropathy

(2012) Yuya Hayashi et al.   MOLECULAR PHARMACEUTICS

Liver transplantation and transthyretin amyloidosis

(2012) Merrill D. Benson   MUSCLE & NERVE

Tafamidis for transthyretin familial amyloid polyneuropathy: A randomized, controlled trial

(2012) T. Coelho et al.   NEUROLOGY

Tafamidis, a potent and selective transthyretin kinetic stabilizer that inhibits the amyloid cascade

(2012) C. E. Bulawa et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Biochemical effect of liver transplantation in two Swedish patients with familial amyloidotic polyneuropathy (FAP-met30)

(2010) Gösta Holmgren et al.   CLINICAL GENETICS

Targeted Delivery of RNAi Therapeutics With Endogenous and Exogenous Ligand-Based Mechanisms

(2010) Akin Akinc et al.   MOLECULAR THERAPY

Rational design of cationic lipids for siRNA delivery

(2010) Sean C Semple et al.   NATURE BIOTECHNOLOGY

Development of Lipidoid–siRNA Formulations for Systemic Delivery to the Liver

(2009) Akin Akinc et al.   MOLECULAR THERAPY

The heat shock response modulates transthyretin deposition in the peripheral and autonomic nervous systems

(2008) Sofia Duque Santos et al.   NEUROBIOLOGY OF AGING