RNA-targeting and gene editing therapies for transthyretin amyloidosis
Published 2022 View Full Article
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Title
RNA-targeting and gene editing therapies for transthyretin amyloidosis
Authors
Keywords
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Journal
Nature Reviews Cardiology
Volume -, Issue -, Pages -
Publisher
Springer Science and Business Media LLC
Online
2022-03-24
DOI
10.1038/s41569-022-00683-z
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Note: Only part of the references are listed.- Association of Left Ventricular Global Longitudinal Strain With Cardiac Amyloid Load in Light Chain Amyloidosis
- (2021) Darae Kim et al. JACC-Cardiovascular Imaging
- Biological Functions of RBP4 and Its Relevance for Human Diseases
- (2021) Julia S. Steinhoff et al. Frontiers in Physiology
- Pathophysiology and Therapeutic Approaches to Cardiac Amyloidosis
- (2021) Jan M. Griffin et al. CIRCULATION RESEARCH
- Longitudinal strain in the management of cardiac AL amyloidosis: do we need it?
- (2021) Claudio Rapezzi et al. EUROPEAN HEART JOURNAL
- Longitudinal strain is an independent predictor of survival and response to therapy in patients with systemic AL amyloidosis
- (2021) Oliver C Cohen et al. EUROPEAN HEART JOURNAL
- CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis
- (2021) Julian D. Gillmore et al. NEW ENGLAND JOURNAL OF MEDICINE
- Gene Editing — A Cure for Transthyretin Amyloidosis?
- (2021) Mathew S. Maurer NEW ENGLAND JOURNAL OF MEDICINE
- HELIOS-A: 9-month results from the phase 3 study of vutrisiran in patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy
- (2021) Davide Adams et al. JOURNAL OF THE NEUROLOGICAL SCIENCES
- Cost-Effectiveness of Tafamidis Therapy for Transthyretin Amyloid Cardiomyopathy
- (2020) Dhruv S. Kazi et al. CIRCULATION
- RNA-based therapeutics in cardiovascular disease
- (2020) Tan Phát Pham et al. CURRENT OPINION IN CARDIOLOGY
- Prevalence and outcome of dual aortic stenosis and cardiac amyloid pathology in patients referred for transcatheter aortic valve implantation
- (2020) Paul R Scully et al. EUROPEAN HEART JOURNAL
- Single‐Dose Pharmacokinetics and Pharmacodynamics of Transthyretin Targeting N‐acetylgalactosamine–Small Interfering Ribonucleic Acid Conjugate, Vutrisiran, in Healthy Subjects
- (2020) Bahru A. Habtemariam et al. CLINICAL PHARMACOLOGY & THERAPEUTICS
- A phase II, open-label, extension study of long-term patisiran treatment in patients with hereditary transthyretin-mediated (hATTR) amyloidosis
- (2020) Teresa Coelho et al. Orphanet Journal of Rare Diseases
- Long-term safety and efficacy of patisiran for hereditary transthyretin-mediated amyloidosis with polyneuropathy: 12-month results of an open-label extension study
- (2020) David Adams et al. LANCET NEUROLOGY
- Reduction in CMR Derived Extracellular Volume With Patisiran Indicates Cardiac Amyloid Regression
- (2020) Marianna Fontana et al. JACC-Cardiovascular Imaging
- Ligand conjugated antisense oligonucleotide for the treatment of transthyretin amyloidosis: preclinical and phase 1 data
- (2020) Nicholas J. Viney et al. ESC Heart Failure
- Effects of Patisiran, an RNA Interference Therapeutic, on Cardiac Parameters in Patients With Hereditary Transthyretin-Mediated Amyloidosis
- (2019) Scott D. Solomon et al. CIRCULATION
- New Frontiers in Lp(a)-Targeted Therapies
- (2019) Matthew J. Borrelli et al. TRENDS IN PHARMACOLOGICAL SCIENCES
- The current state and future directions of RNAi-based therapeutics
- (2019) Ryan L. Setten et al. NATURE REVIEWS DRUG DISCOVERY
- Strategies, design, and chemistry in siRNA delivery systems
- (2019) Yizhou Dong et al. ADVANCED DRUG DELIVERY REVIEWS
- Cardiac troponins as biomarkers for cardiac disease
- (2019) Claudio Passino et al. Biomarkers in Medicine
- Treatment of cardiac transthyretin amyloidosis: an update
- (2019) Michele Emdin et al. EUROPEAN HEART JOURNAL
- Strong and Sustained Antihypertensive Effect of Small Interfering RNA Targeting Liver Angiotensinogen
- (2019) Estrellita Uijl et al. HYPERTENSION
- Transthyretin Amyloid Cardiomyopathy
- (2019) Frederick L. Ruberg et al. JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY
- Hereditary transthyretin amyloidosis: a model of medical progress for a fatal disease
- (2019) David Adams et al. Nature Reviews Neurology
- Expert Consensus Recommendations for the Suspicion and Diagnosis of Transthyretin Cardiac Amyloidosis
- (2019) Mathew S. Maurer et al. Circulation-Heart Failure
- Keys to early diagnosis of cardiac amyloidosis: red flags from clinical, laboratory and imaging findings
- (2019) Giuseppe Vergaro et al. European Journal of Preventive Cardiology
- siRNA drug development against hepatitis B virus infection
- (2018) Robert Flisiak et al. EXPERT OPINION ON BIOLOGICAL THERAPY
- Estimating the global prevalence of transthyretin familial amyloid polyneuropathy
- (2018) Hartmut H. Schmidt et al. MUSCLE & NERVE
- 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
- GalNAc-siRNA Conjugates: Leading the Way for Delivery of RNAi Therapeutics
- (2018) Aaron D. Springer et al. Nucleic Acid Therapeutics
- A Single Administration of CRISPR/Cas9 Lipid Nanoparticles Achieves Robust and Persistent In Vivo Genome Editing
- (2018) Jonathan D. Finn et al. Cell Reports
- Cobomarsen, an oligonucleotide inhibitor of miR-155, co-ordinately regulates multiple survival pathways to reduce cellular proliferation and survival in cutaneous T-cell lymphoma
- (2018) Anita G. Seto et al. BRITISH JOURNAL OF HAEMATOLOGY
- Epidemiology of Transthyretin Familial Amyloid Polyneuropathy in Portugal: A Nationwide Study
- (2018) Mónica Inês et al. NEUROEPIDEMIOLOGY
- Tafamidis Treatment for Patients with Transthyretin Amyloid Cardiomyopathy
- (2018) Mathew S. Maurer et al. NEW ENGLAND JOURNAL OF MEDICINE
- Recent Advances in Oligonucleotide-Based Therapy for Transthyretin Amyloidosis: Clinical Impact and Future Prospects
- (2018) Yuya Hayashi et al. BIOLOGICAL & PHARMACEUTICAL BULLETIN
- Clinical Proof of Concept for a Novel Hepatocyte-Targeting GalNAc-siRNA Conjugate
- (2017) Tracy S. Zimmermann et al. MOLECULAR THERAPY
- Exosomes facilitate therapeutic targeting of oncogenic KRAS in pancreatic cancer
- (2017) Sushrut Kamerkar et al. NATURE
- Targeting of Antithrombin in Hemophilia A or B with RNAi Therapy
- (2017) K. John Pasi et al. NEW ENGLAND JOURNAL OF MEDICINE
- Suppressing transthyretin production in mice, monkeys and humans using 2nd-Generation antisense oligonucleotides
- (2016) Elizabeth J. Ackermann et al. AMYLOID-JOURNAL OF PROTEIN FOLDING DISORDERS
- Nonbiopsy Diagnosis of Cardiac Transthyretin AmyloidosisCLINICAL PERSPECTIVE
- (2016) Julian D. Gillmore et al. CIRCULATION
- Long-term treatment of transthyretin familial amyloid polyneuropathy with tafamidis: a clinical and neurophysiological study
- (2016) Violaine Planté-Bordeneuve et al. JOURNAL OF NEUROLOGY
- Monitoring effectiveness and safety of Tafamidis in transthyretin amyloidosis in Italy: a longitudinal multicenter study in a non-endemic area
- (2016) A. Cortese et al. JOURNAL OF NEUROLOGY
- Genotype and Phenotype of Transthyretin Cardiac Amyloidosis
- (2016) Mathew S. Maurer et al. JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY
- An Investigational RNAi Therapeutic Targeting Glycolate Oxidase Reduces Oxalate Production in Models of Primary Hyperoxaluria
- (2016) Abigail Liebow et al. JOURNAL OF THE AMERICAN SOCIETY OF NEPHROLOGY
- Genotype-phenotype correlation and course of transthyretin familial amyloid polyneuropathies in France
- (2015) Louise-Laure Mariani et al. ANNALS OF NEUROLOGY
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- (2015) Esther González-López et al. EUROPEAN HEART JOURNAL
- Efficacy and safety of patisiran for familial amyloidotic polyneuropathy: a phase II multi-dose study
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- (2015) Bo-Göran Ericzon et al. TRANSPLANTATION
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- Myotonic dystrophy: time for evidence-based therapy
- (2014) Perry Elliott EUROPEAN HEART JOURNAL
- Proteolytic cleavage of Ser52Pro variant transthyretin triggers its amyloid fibrillogenesis
- (2014) P. P. Mangione et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Effect on disability and safety of Tafamidis in late onset of Met30 transthyretin familial amyloid polyneuropathy
- (2013) P. Lozeron et al. EUROPEAN JOURNAL OF NEUROLOGY
- Phase I Clinical Trial of SYL040012, a Small Interfering RNA Targeting β-Adrenergic Receptor 2, for Lowering Intraocular Pressure
- (2013) Javier Moreno-Montañés et al. MOLECULAR THERAPY
- In Vitro and In Vivo Efficacy of SYL040012, a Novel siRNA Compound for Treatment of Glaucoma
- (2013) Tamara Martínez et al. MOLECULAR THERAPY
- Safety and Efficacy of RNAi Therapy for Transthyretin Amyloidosis
- (2013) Teresa Coelho et al. NEW ENGLAND JOURNAL OF MEDICINE
- Transthyretin (TTR) Cardiac Amyloidosis
- (2012) Frederick L. Ruberg et al. CIRCULATION
- Natural history of transthyretin Val30Met familial amyloid polyneuropathy: analysis of late-onset cases from non-endemic areas
- (2012) Haruki Koike et al. JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY
- Tafamidis for transthyretin familial amyloid polyneuropathy: A randomized, controlled trial
- (2012) T. Coelho et al. NEUROLOGY
- Development of cardiomyopathy after liver transplantation in Swedish hereditary transthyretin amyloidosis (ATTR) patients
- (2011) Sadahisa Okamoto et al. AMYLOID-JOURNAL OF PROTEIN FOLDING DISORDERS
- RBP4: a controversial adipokine
- (2011) Primoz Kotnik et al. EUROPEAN JOURNAL OF ENDOCRINOLOGY
- Familial amyloid polyneuropathy
- (2011) Violaine Planté-Bordeneuve et al. LANCET NEUROLOGY
- Biochemical effect of liver transplantation in two Swedish patients with familial amyloidotic polyneuropathy (FAP-met30)
- (2010) Gösta Holmgren et al. CLINICAL GENETICS
- Progression of transthyretin amyloid neuropathy after liver transplantation
- (2010) J. J. Liepnieks et al. NEUROLOGY
- Knocking down barriers: advances in siRNA delivery
- (2009) Kathryn A. Whitehead et al. NATURE REVIEWS DRUG DISCOVERY
- Senile systemic amyloidosis affects 25% of the very aged and associates with genetic variation inalpha2‐macroglobulinandtau: A population‐based autopsy study
- (2008) Maarit Tanskanen et al. ANNALS OF MEDICINE
- Trivalent, Gal/GalNAc-containing ligands designed for the asialoglycoprotein receptor
- (2008) Oleg Khorev et al. BIOORGANIC & MEDICINAL CHEMISTRY
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