Leading RNA Interference Therapeutics Part 1: Silencing Hereditary Transthyretin Amyloidosis, with a Focus on Patisiran
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Title
Leading RNA Interference Therapeutics Part 1: Silencing Hereditary Transthyretin Amyloidosis, with a Focus on Patisiran
Authors
Keywords
-
Journal
Molecular Diagnosis & Therapy
Volume -, Issue -, Pages -
Publisher
Springer Science and Business Media LLC
Online
2019-11-07
DOI
10.1007/s40291-019-00434-w
References
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Note: Only part of the references are listed.- Association of Patisiran, an RNA Interference Therapeutic, With Regional Left Ventricular Myocardial Strain in Hereditary Transthyretin Amyloidosis
- (2019) Masatoshi Minamisawa et al. JAMA Cardiology
- Hereditary transthyretin amyloidosis: a model of medical progress for a fatal disease
- (2019) David Adams et al. Nature Reviews Neurology
- Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis
- (2018) David Adams et al. NEW ENGLAND JOURNAL OF MEDICINE
- Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis
- (2018) Merrill D. Benson et al. NEW ENGLAND JOURNAL OF MEDICINE
- Lipid Nanoparticles Enabling Gene Therapies: From Concepts to Clinical Utility
- (2018) Jayesh A. Kulkarni et al. Nucleic Acid Therapeutics
- Delivery of miRNA-Targeted Oligonucleotides in the Rat Striatum by Magnetofection with Neuromag®
- (2018) Simoneide Titze de Almeida et al. MOLECULES
- Gene-silencing technology gets first drug approval after 20-year wait
- (2018) Heidi Ledford NATURE
- Tafamidis Treatment for Patients with Transthyretin Amyloid Cardiomyopathy
- (2018) Mathew S. Maurer et al. NEW ENGLAND JOURNAL OF MEDICINE
- Therapeutic Oligonucleotides: State of the Art
- (2018) C.I. Edvard Smith et al. Annual Review of Pharmacology and Toxicology
- An indirect treatment comparison of the efficacy of patisiran and tafamidis for the treatment of hereditary transthyretin-mediated amyloidosis with polyneuropathy
- (2018) Violaine Planté-Bordeneuve et al. EXPERT OPINION ON PHARMACOTHERAPY
- Natural history and survival in stage 1 Val30Met transthyretin familial amyloid polyneuropathy
- (2018) Teresa Coelho et al. NEUROLOGY
- Transthyretin familial amyloid polyneuropathy: an update
- (2017) Violaine Plante-Bordeneuve JOURNAL OF NEUROLOGY
- Clinical Proof of Concept for a Novel Hepatocyte-Targeting GalNAc-siRNA Conjugate
- (2017) Tracy S. Zimmermann et al. MOLECULAR THERAPY
- Lipid Nanoparticle Systems for Enabling Gene Therapies
- (2017) Pieter R. Cullis et al. MOLECULAR THERAPY
- The chemical evolution of oligonucleotide therapies of clinical utility
- (2017) Anastasia Khvorova et al. NATURE BIOTECHNOLOGY
- The Race of 10 Synthetic RNAi-Based Drugs to the Pharmaceutical Market
- (2017) Ricardo Titze-de-Almeida et al. PHARMACEUTICAL RESEARCH
- siRNA Delivery Strategies: A Comprehensive Review of Recent Developments
- (2017) Katyayani Tatiparti et al. Nanomaterials
- Chemical and structural modifications of RNAi therapeutics
- (2016) Sook Hee Ku et al. ADVANCED DRUG DELIVERY REVIEWS
- First European consensus for diagnosis, management, and treatment of transthyretin familial amyloid polyneuropathy
- (2016) David Adams et al. CURRENT OPINION IN NEUROLOGY
- Oligonucleotide conjugates – Candidates for gene silencing therapeutics
- (2016) Matt Gooding et al. EUROPEAN JOURNAL OF PHARMACEUTICS AND BIOPHARMACEUTICS
- Long-term treatment of transthyretin familial amyloid polyneuropathy with tafamidis: a clinical and neurophysiological study
- (2016) Violaine Planté-Bordeneuve et al. JOURNAL OF NEUROLOGY
- Ophthalmological manifestations in hereditary transthyretin (ATTR V30M) carriers: a review of 513 cases
- (2015) João Melo Beirão et al. AMYLOID-JOURNAL OF PROTEIN FOLDING DISORDERS
- Evolving landscape in the management of transthyretin amyloidosis
- (2015) Philip N. Hawkins et al. ANNALS OF MEDICINE
- Diagnosis, Prognosis, and Therapy of Transthyretin Amyloidosis
- (2015) Morie A. Gertz et al. JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY
- Efficacy and safety of patisiran for familial amyloidotic polyneuropathy: a phase II multi-dose study
- (2015) Ole B Suhr et al. Orphanet Journal of Rare Diseases
- Neuropathologic analysis of Tyr69His TTR variant meningovascular amyloidosis with dementia
- (2015) Jennifer L. Ziskin et al. Acta Neuropathologica Communications
- Repurposing Diflunisal for Familial Amyloid Polyneuropathy
- (2015) John L. Berk et al. JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION
- Recent Advances in Nanoparticle-Mediated siRNA Delivery
- (2014) John-Michael Williford et al. Annual Review of Biomedical Engineering
- Renaissance of mammalian endogenous RNAi
- (2014) Petr Svoboda FEBS LETTERS
- Recent progress in the understanding and treatment of transthyretin amyloidosis
- (2014) Y. Sekijima JOURNAL OF CLINICAL PHARMACY AND THERAPEUTICS
- CNS involvement in V30M transthyretin amyloidosis: clinical, neuropathological and biochemical findings
- (2014) L. F. Maia et al. JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY
- 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
- Molecular Mechanisms of RNA Interference
- (2013) Ross C. Wilson et al. Annual Review of Biophysics
- Delivery materials for siRNA therapeutics
- (2013) Rosemary Kanasty et al. NATURE MATERIALS
- Safety and Efficacy of RNAi Therapy for Transthyretin Amyloidosis
- (2013) Teresa Coelho et al. NEW ENGLAND JOURNAL OF MEDICINE
- Mutant KRAS is a druggable target for pancreatic cancer
- (2013) E. Zorde Khvalevsky et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Tafamidis for transthyretin familial amyloid polyneuropathy: A randomized, controlled trial
- (2012) T. Coelho et al. NEUROLOGY
- The Many Faces of RNAi
- (2011) René F. Ketting DEVELOPMENTAL CELL
- Origins and Mechanisms of miRNAs and siRNAs
- (2009) Richard W. Carthew et al. CELL
- The RNA-induced Silencing Complex: A Versatile Gene-silencing Machine
- (2009) Ashley J. Pratt et al. JOURNAL OF BIOLOGICAL CHEMISTRY
- Lipid-based nanotherapeutics for siRNA delivery
- (2009) A. Schroeder et al. JOURNAL OF INTERNAL MEDICINE
- A three-dimensional view of the molecular machinery of RNA interference
- (2009) Martin Jinek et al. NATURE
- Knocking down barriers: advances in siRNA delivery
- (2009) Kathryn A. Whitehead et al. NATURE REVIEWS DRUG DISCOVERY
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