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Stanford University

Lysosomes in Macular Degeneration

Principal Investigator: Daria Mochly-Rosen

Research Team: Gouri Yogalingam

The work was performed by Dr. Yogalingam at the SENS Research Foundation Research Center (SRF-RC) in Mountain View, California and in the laboratory of Professor Daria Mochly-Rosen at Stanford University. Dr. Yogalingam’s work was divided between two projects as follows:

Targeting non-lysosomal enzymes to the lysosomal compartment of retinal epithelial cells

In this project the majority of work was initiated by SENS Research Foundation and continued to be performed at the SRF-RC through 2014. This work included:

  • Cloning, expressing and purifying recombinantly-expressed enzymes and characterizing their kinetics in vitro.
  • Assessing the uptake and lysosomal targeting of recombinantly-expressed, purified candidate enzymes into cultured mammalian cells, including human macrophage and retinal epithelial cell lines. Most of this involved cell biology and biochemical techniques, and was performed at the SRF-RC. Some imaging (immunofluorescence and electron microscopy) to verify correct targeting of candidate enzymes to lysosomes was done at Stanford.

Developing effective therapies for the treatment of AMD

In this work adeno-associated virus-based vectors were engineered to contain cDNA encoding candidate enzymes. Retinal epithelial cell lines were transduced with recombinant AAV containing candidate enzyme cDNAs. The recombinant proteins were assessed for their correct lysosomal targeting, folding and enzymatic activity towards A2E, an AMD storage product.