Targeted mRNA Therapy for Ornithine Transcarbamylase Deficiency

Author:

Prieve Mary G.,Harvie Pierrot,Monahan Sean D.,Roy Debashish,Li Allen G.,Blevins Teri L.,Paschal Amber E.,Waldheim Matt,Bell Eric C.,Galperin Anna,Ella-Menye Jean-Rene,Houston Michael E.

Funder

PhaseRx, Inc.

Publisher

Elsevier BV

Subject

Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine

Reference57 articles.

1. Ornithine transcarbamylase deficiency;Lichter-Konecki,1993

2. Systemic delivery of factor IX messenger RNA for protein replacement therapy;Ramaswamy;Proc. Natl. Acad. Sci. USA,2017

3. Therapeutic efficacy in a hemophilia B model using a biosynthetic mRNA liver depot system;DeRosa;Gene Ther.,2016

4. Advances in non-viral DNA vectors for gene therapy;Hardee;Genes (Basel),2017

5. A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency;Hacein-Bey-Abina;N. Engl. J. Med.,2003

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