Systemic delivery of factor IX messenger RNA for protein replacement therapy

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

(2016) James S. Butler et al.   AMYLOID-JOURNAL OF PROTEIN FOLDING DISORDERS

Therapeutic efficacy in a hemophilia B model using a biosynthetic mRNA liver depot system

(2016) F DeRosa et al.   GENE THERAPY

Lipid nanoparticles for targeted siRNA delivery – going from bench to bedside

(2016) Victor Kotelianski et al.   International Journal of Nanomedicine

The past and future of haemophilia: diagnosis, treatments, and its complications

(2016) Flora Peyvandi et al.   LANCET

Recent progress in nanomaterials for gene delivery applications

(2016) Erhan Keles et al.   Biomaterials Science

Employing a Gain-of-Function Factor IX Variant R338L to Advance the Efficacy and Safety of Hemophilia B Human Gene Therapy: Preclinical Evaluation Supporting an Ongoing Adeno-Associated Virus Clinical Trial

(2015) Paul E. Monahan et al.   HUMAN GENE THERAPY

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

mRNA-based therapeutics — developing a new class of drugs

(2014) Ugur Sahin et al.   NATURE REVIEWS DRUG DISCOVERY

Non-viral vectors for gene-based therapy

(2014) Hao Yin et al.   NATURE REVIEWS GENETICS

Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B

(2014) Amit C. Nathwani et al.   NEW ENGLAND JOURNAL OF MEDICINE

Modified mRNA directs the fate of heart progenitor cells and induces vascular regeneration after myocardial infarction

(2013) Lior Zangi et al.   NATURE BIOTECHNOLOGY

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

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

Guidelines for the management of hemophilia

(2012) A. Srivastava et al.   HAEMOPHILIA

Expression of therapeutic proteins after delivery of chemically modified mRNA in mice

(2011) Michael S D Kormann et al.   NATURE BIOTECHNOLOGY

Prolonged activity of factor IX as a monomeric Fc fusion protein

(2010) R. T. Peters et al.   BLOOD

Improved treatment feasibility in children with hemophilia using arteriovenous fistulae: the results after seven years of follow-up

(2009) M. E. Mancuso et al.   HAEMATOLOGICA

Bioengineered Factor IX Molecules with Increased Catalytic Activity Improve the Therapeutic Index of Gene Therapy Vectors for Hemophilia B

(2009) Nicola Brunetti-Pierri et al.   HUMAN GENE THERAPY