A novel nonviral gene delivery system for treating Leber's congenital amaurosis

Ther Deliv. 2017 Oct;8(10):823-826. doi: 10.4155/tde-2017-0072.

Authors

Da Sun¹, Rebecca M Schur¹, Zheng-Rong Lu^{1

2}

Affiliations

¹ Case Center for Biomolecular Engineering, Department of Biomedical Engineering, School of Engineering, Case Western Reserve University, Cleveland, OH 44106, USA.
² M. Frank Rudy & Margaret Domiter Rudy Professor of Biomedical Engineering, Department of Biomedical Engineering, Case Western Reserve University, Wickenden 427, Mail Stop 7207, 10900 Euclid Avenue, Cleveland, OH 44106, USA.

PMID: 28944740
DOI: 10.4155/tde-2017-0072

No abstract available

Keywords: ECO; LCA; RPE65; gene therapy; nanoparticle; nonviral; retina.

MeSH terms

Administration, Ophthalmic
Animals
Cysteine / analogs & derivatives*
Cysteine / chemistry
DNA / administration & dosage
Ethylenediamines / chemistry*
Fatty Alcohols / chemistry*
Genetic Therapy / methods*
Humans
Leber Congenital Amaurosis / genetics*
Leber Congenital Amaurosis / therapy*
Nanoparticles / administration & dosage
Plasmids / administration & dosage

Substances

((1-aminoethyl)iminobis(N-(oleicylcysteinyl-1-amino-ethyl)propionamide))
Ethylenediamines
Fatty Alcohols
DNA
Cysteine