Research Database
Endoglin as a portal for transfection-free targeted delivery and gene silencing in diabetic retinopathy
MD Imam, PhD
Institution:
Vanderbilt University Medical Center
Grant Number:
11-22-IBSPM-12
Type of Grant:
Basic
Diabetes Type:
Both Type 1 and Type 2 Diabetes
Therapeutic Goal:
N/A
Project Date:
-
Project Status:
active

Research Description

Diabetic retinopathy is a leading cause of blindness in the working age population. Blindness in this disease is often caused by abnormal blood vessels growth in the eye, through a process called neovascularization. These faulty blood vessels are leaky and cause hemorrhage in the eye. To control this faulty blood vessel growth, physicians often use laser treatments or intraocular injections of anti-VEGF therapy. Laser treatment is not curative and can damage to the healthy tissues along with pathologic blood vessels. Anti-VEGF therapy is effective; however, a large number of diabetic patients do not respond to this therapy. To improve the current treatment options, efficient therapeutics are needed. Nanomedicines have the potential to overcome the limitations of current therapeutics by targeting disease specific tissues, avoiding repeated intraocular injections, reduced side effects, and effective for larger patient populations. This research proposal is outlined to develop a novel molecular targeted therapy to improve clinical management of diabetic retinopathy. This research will facilitate the clinical translation of this new therapy called nanomedicine for the treatment of diabetic retinopathy. The applicant has developed this nanotechnology, which are highly specific for diseased mRNA to treat pathologic blood vessels, and not to affect the healthy blood vessels. The efficacy of this strategy will be tested in a preclinical model that exhibits abnormal blood vessel growth in the eye. If successful, this nanomedicine will provide valuable information for clinical translation of this nanotechnology for the treatment of diabetic retinopathy.

Research Profile

What area of diabetes research does your project cover? What role will this particular project play in preventing, treating and/or curing diabetes?

The PI (Dr. Uddin) is a primary faculty member in the Department of Ophthalmology and Visual Sciences, Vanderbilt University School of Medicine, and has a secondary faculty appointment in the Department of Biomedical Engineering at Vanderbilt University. His long-term goal is to develop novel gene therapy methods to treat retinal vascular diseases, primarily diabetic retinopathy (DR). Dr. Uddin is a chemist and biomedical engineer by training, with expertise in the development of molecularly targeted imaging and therapeutic agents for retinal diseases. This ADA funded project will focus on development of a new gene therapy method for the treatment of proliferative diabetic retinopathy (PDR).

If a person with diabetes were to ask you how your project will help them in the future, how would you respond?

Pathogenesis of diabetic retinopathy (DR) involves two phases: an early non-proliferative DR (NPDR) and late-stage proliferative DR (PDR). PDR consists of abnormal blood vessels growth in the retina, called neovascularization (NV). Despite development of strategies for the treatment of ocular neovascularization, these treatment strategies are often associated with several limitations. This ADA funded project will help to develop a gene therapy method to inhibit target mRNA that contributes to DR pathogenesis. If successful, our gene therapy method will reduce or even eliminate repeated intraocular injections of therapy in patients for the treatment of diabetic retinopathy in the future.

Why important for you, personally, to become involved in diabetes research? What role will this award play?

Diabetic retinopathy (DR) is the leading cause of vision loss affecting a large number of working-age populations in US and worldwide. This ADA funded project will facilitate development of a novel nanotechnology-based strategy as gene therapy method to overcome the limitations of current treatment options.

In what direction do you see the future of diabetes research going?

Nanotechnology has the promise of enhancing diagnostic capabilities for early disease detection thereby improving outcomes in diabetic retinopathy, and eventually other diseases such as neovascular age related macular degeneration.