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Novel tissue‐targeted localized gene therapy for corneal scarring and neovascularization
Author(s) -
Mohan R.,
Gupta S.,
Sharma A.,
Anumanthan G.,
Sinha P.,
Fink M.,
Tripathi R.,
Raikwar S.,
Giuliano E.,
Rieger F.,
Hesemann N.,
Sinha N.,
Chaurasia S.
Publication year - 2016
Publication title -
acta ophthalmologica
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.534
H-Index - 87
eISSN - 1755-3768
pISSN - 1755-375X
DOI - 10.1111/j.1755-3768.2016.0452
Subject(s) - corneal neovascularization , genetic enhancement , cornea , neovascularization , medicine , in vivo , blindness , gene delivery , corneal diseases , angiogenesis , corneal endothelium , bioinformatics , pathology , gene , cancer research , ophthalmology , biology , microbiology and biotechnology , optometry , biochemistry
Summary Corneal scarring and neovascularization (NV) due to trauma, injury and/or infection are leading cause of global blindness. This presentation will provide an overview of corneal gene therapy and stipulate novel bench‐to‐bedside translational strategies for human application. The talk will include information about identified potent adeno‐associated virus (AAV) and nanoparticle vectors, simple and minimally invasive vector delivery techniques for delivering genes into desired corneal cells, and defined gene therapy approaches for introducing therapeutic genes selectively into corneal keratocytes or endothelium in vivo through single application of vector employing customized delivery techniques. Further, talk will discuss cornea‐specific mechanisms driving pathologic process, novel molecular targets for interrupting TGF β 1 signaling pathways, and therapeutic genes identified from basic‐science corneal wound healing investigations performed in my lab laboratory using established small and large animal and donor human corneas. Finally, it will show potential of single‐ and two‐gene combination therapy given locally in the cornea via optimized methods in inhibiting and eliminating corneal scarring and neovascularization in vivo in preclinical rabbit studies. The optimized gene therapy approaches could be easily applied in a clinical setting, if safety and toxicity are proven.