Research Interest:

Studies in Dr. Farber’s laboratory are aimed to increase basic knowledge about inherited diseases of the retina and to test and/or develop appropriate methodologies for the treatment and eventual cure of these disorders. Her research has focused on the isolation and characterization of genes responsible for these diseases using biochemistry, molecular biology, molecular genetics, animal models of retinal degeneration and the DNA from affected patients. In addition, she is trying to elucidate the molecular pathways by which mutations in the OA1 gene cause ocular albinism, a developmental disease involving deficient melanogenesis in the retinal pigment epithelium and misrouting of optic axons at the brain's optic chiasm. Most important, Dr. Farber is interested in regenerating diseased retinas with the use of stem cell-derived micro-vesicles.

Past and Current Research

Dr. Farber's laboratory has cloned and characterized several genes encoding enzymes and proteins that play a key role in vision. They have demonstrated that genetic abnormalities in one of the subunits of these enzymes, beta- phosphodiesterase, leads to blindness in some animal models (mice and dogs) and in some recessive forms of human retinitis pigmentosa. With the application of gene therapy, they have rescued degenerating mouse photoreceptors using modified adenoviral vectors to deliver the normal beta-phosphodiesterase gene to these cells. Moreover, they successfully used a non-viral,non-invasive delivery system, iontophoresis, for the same purpose.

Dr. Farber's laboratory has also isolated and characterized the mouse homolog of the gene causing X-linked juvenile retinoschisis; the gene causing disease in the rd7 mouse, which is responsible for Enhanced S-cone syndrome in humans; RP1, a gene that when mutated is responsible for a type of human dominant retinitis pigmentosa; the beta-transducin gene, causing disease in the Rd4 mouse, which together with the Sp4 gene (that Dr. Farber's group found regulates the beta-phosphodiesterase gene) cause digenic disease in humans; the mouse Rhbdd2 gene,that when mutated causes recessive retinitis pigmentosa in humans.

Other areas of investigation in Dr. Farber's lab have included isolation of cone-specific genes such as ZBED4, which causes autosomal dominant rod-cone dystrophy. In addition, they have worked on the mechanisms that regulate transcription of the above genes and studied the encoded proteins’ functions and the pathways that they control.

Lately, Dr. Farber pioneered the identification and characterization of micro-vesicles released by cultured mouse embryonic stem cells (ESMVs), determined how they transfer their cargo to other stem cells or cells of other origin, and studied the effects that they have on cultured retinal progenitor Müller cells. These studies lead her to investigate a totally unexplored possibility: the use of ESMVs in the rescue of damaged retinas. Her laboratory is currently carrying out preliminary studies on this, in vivo, and the exciting results that they are obtaining will allow them to develop a completely new line of research, first comparing mouse and human ESMVs and then investigating the involvement of human ESMVs in maintaining the normal structure and function of the retina, as well as their possible use to cure or delay the demise of damaged retinas.

Detailed Biography:

Debora B. Farber is a biochemist and molecular biologist who has been a faculty member of the UCLA School of Medicine since she joined the Department of Ophthalmology in 1977. She became a full professor in 1984, a Karl Kirchgessner Endowed Chair in 2001 and a Distinguished Professor in 2007. She has been Associate Director for Research of the Brain Research Institute (1992 -1994) and Associate Director of the Jules Stein Eye Institute as well as Co-Chief of its Vision Science Division (1994 – 2004). Dr. Farber earned her M.S. in Chemistry and her Ph.D. in Organic Chemistry at the University of Buenos Aires, Argentina. She was a postdoctoral fellow in the Chemistry Department, University of California at Santa Barbara, where she studied the cyclic nucleotide regulation of different processes. She started working on the retina and diseases affecting it when she joined UCLA, and soon thereafter she received a National Eye Institute Research Career Development Award. In addition to her continued support from NIH, Dr. Farber also was granted an NIH MERIT Award. She received the highest honorary degree, Doctor Honoris Causa, from the University of Göteborg, Sweden, the Proctor Medal from the Association for Research in Vision and Ophthalmology, and the "Marraine 2005" honorary title from L'Association Degenerescence Maculaire Liee a l'Age, France. She also was the recipient of the Louisiana State University School of Medicine Distinguished Lecturer Award for Outstanding Contributions to the Neurosciences, the Alcon Research Institute Award for Outstanding Contributions to Vision Research, The Foundation Fighting Blindness Trustee Award, the Paul Kayser International Award of Merit in Retina Research, two Research to Prevent Blindness Senior Scientific Investigators Awards and the Visionary Award from The Vision of Children Foundation. UCLA granted Dr. Farber the Woman of Science Award, the Franklin D. Murphy, M.D. Prize from the UCLA School of Medicine and the David Geffen School of Medicine MAA Medical Science Award. She is an Editorial Board member of several journals and has given Plenary lectures at national and international meetings. She also was elected Trustee and Vice-President of the Association for Research in Vision and Ophthalmology.