ID of Optimal Gene Delivery Vectors in Primate Retina for Treatment of Human Disorders

Technology #16134

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Researchers
Shannon Elizabeth Boye
Sanford L. Boye
Paul D. Gamlin
Managed By
April Kilburn
Assistant Director 352-392-8929

Labels Non-Human Primate Eyes with Fluorescent Proteins and/or Fluorescent Dyes, Creating Sortable Cell Populations, Allowing for Screening of Capsid and Promoter Libraries

This strategy uses both an AAV vector to drive fluorescent protein expression and an injectable fluorescent dye to label different retinal cell types in life, thus allowing for the selective recovery of these cells. When coupled with AAV capsid libraries or promoter libraries, the methodology provides a means of identifying those key vectors that will ultimately be of use in targeted therapies to treat eye diseases, such as age-related macular degeneration, retinitis pigmentosa, or Leber congenital amaurosis (LCA). In the United States, more than 10 million people suffer from these diseases, which have treatments that only slow the progression of the disease and no cure. University of Florida researchers have developed a strategy that can differentially label and sort retinal cell types in non-human primates. This can be used to identity novel gene therapy vectors from highly complex libraries as well as to evaluate the cell-type specificity and effectiveness of specific gene therapy vectors.

Application

Creates sortable retinal cell populations prior to capsid or promoter library injection, allowing for the optimal gene delivery vectors to be selected for specificity to different retinal cell types

Advantages

  • Creates sortable cell populations prior to capsid/promoter library injection, leading to improved specificity and potency of gene-based therapies for retinal diseases
  • Employs a clinically relevant species, increasing translatability of the results for treating human eye disorders

Technology

Labeling of different retinal cell types is achieved with either injection of an AAV vector containing a fluorescent reporter or injection with a fluorescent dye. After these reagents are administered in vivo, researchers can then administer additional gene delivery vectors and reagents to the eye (i.e. capsid or promoter libraries). They can then sort the fluorescently labeled cells using fluorescence-activated cell sorting (FACS) and perform transcript analysis using polymerase chain reaction (PCR) to determine the most effective gene delivery vector for specific retinal cell types. Since the anatomy of the non-human primate eyes is most similar to man, vectors identified with this approach have potential for treating human eye disorders as well.