Debora B. Farber, Ph.D.

Laboratory Address:
Los Angeles, CA 90095

Office Address:
Los Angeles, CA 90095

Distinguished Professor, Ophthalmology
Karl Kirchgessner Chair, Ophthalmology
Member, Stein Eye Institute, Brain Research Institute, Molecular Biology Institute
Research Interests

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.


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.


A selected list of publications:

Gao Y-Q, Danciger D, Ozgul RK, Jacobson SF and Farber DB   Association of Asn306Ser variants in the genes for the SP4 transcription factor and the beta-subunitof transducin with digenic disease, Molecular Vision, 2007; (13): 287-92.
Kitamura E, Danciger M, Yamashita C, Rao NP, Nusinowitz S, Chang B, Farber DB   Disruption of the gene encoding the beta1-subunit of transducin in the Rd4/+ mouse, Ophthalmol Vis Sci. , 2006; 47(4): 1293-1301.
Lerner LE. Piri N. Farber DB   Transcriptional and post-transcriptional regulation of the rod cGMP- phosphodiesterase beta-subunit gene, Adv Exp Med Biol, 2006; (572): 328-29.
Piri N, Mendoza E , Shih J, Yamashita, CK, Akhmedov NB, and Farber DB   Translational regulation of the Rod Photoreceptor cGMP-Phosphodiesterase: the role of the 5���- and 3���-Untranslated Regions, Exp Eye Res, 2006; 83: 841-848.
Piriev NI, Gao Y, Danciger M, Mendoza E, Fishman GA, and Farber DB   A substitution of G to C in the cone cGMP-phosphodiesterase gamma subunit, Ophthalmology, 2005; 112(1): 159-166.
Reid, SN Farber, DB   Glial transcytosis of a photoreceptor-secreted signaling protein, retinoschisin Glia. , 2005; 49(3): 397-406.
Lerner, LE Peng, GH Gribanova, YE Chen, S Farber, DB   Sp4 is expressed in retinal neurons, activates transcription of photoreceptor-specific genes, and synergizes with Crx The Journal of biological chemistry. , 2005; 280(21): 20642-50.
Akhmedov, NB Yamashita, CK Tran, D Piri, NI Aguirre, GD Farber, DB   Two forms of the large tumor suppressor gene (Lats1) protein expressed in the vertebrate retina Biochimica et biophysica acta. , 2005; 1728(1-2): 11-7.
Viczian AS, Verardo M, Zuber ME, Knox, BE and Farber DB   Conserved cone photoreceptor transcriptional regulation in vertebrates, FEBS Letters, 2004; 577(1-2): 259-264.
Ozgul RK, Durukan H, Turan A, Oner C, Ogus A, Farber DB   Molecular Analysis of the ABCA4 gene in Turkish patients with Stargardt disease and retinitis pigmentosa, Human Mutat, 2004; 23(5): 523.
Piri, N Yamashita, CK Shih, J Akhmedov, NB Farber, DB   Differential expression of rod photoreceptor cGMP-phosphodiesterase alpha and beta subunits: mRNA and protein levels The Journal of biological chemistry. , 2003; 278(39): 36999-7005.
Nusinowitz S, Nguyen L, Radu R, Kashani Z, Farber D and Danciger M   Electroretinographic evidence for altered phototransduction gain and slowed recovery from photobleaches in albino mice with a MET450 variant in RPE65, Exp Eye Res, 2003; 77(5): 627-38.
Saghizadeh M, Brown, DJ, Tajbakhsh J, Chen Z, Kenney MC, Farber DB and Nelson SF   Evaluation of techniques using amplified nucleic acid probes for gene expression profiling, Biomol Eng, 2003; 20(3): 97-106.
Piriev, NI Yamashita, CK Shih, J Farber, DB   Expression of cone photoreceptor cGMP-phosphodiesterase alpha' subunit in Chinese hamster ovary, 293 human embryonic kidney, and Y79 retinoblastoma cells Molecular vision [electronic resource]. , 2003; 9: 80-6.
Reid, SN Yamashita, C Farber, DB   Retinoschisin, a photoreceptor-secreted protein, and its interaction with bipolar and muller cells The Journal of neuroscience : the official journal of the Society for Neuroscience. , 2003; 23(14): 6030-40.
Farber DB and Tsang SH   Stationary night-blindness or progressive retinal degeneration in mice carrying different alleles of PDEgamma, Front Biosci, 2003; 8: S666-675.
Akhmedov NB, Baldwin V, Minoofar KD, Zanger B, Kijas JW, Hunter L, Mellerssh C, Ostrander E, Acland G, Farber DB, Aguirre GD   Cloning and characterization of the canine photoreceptor-specific cone-rod homeobox gene (CRX) and evaluation as a candidate for early onset photoreceptor disease in the dog, Mol Vis, 2002; 8: 79-84.
Lerner LE, Gribanova YE, Whitaker L, Knox BE, Farber DB   The rod cGMP-phosphodiesterase beta-subunit promoter is a specific target for Sp4 and is not activated by other Sp proteins or CRX, J Biol Chem, 2002; 277: 25877-25883.
Danciger, M Hendrickson, J Lyon, J Toomes, C McHale, JC Fishman, GA Inglehearn, CF Jacobson, SG Farber, DB   CORD9 a new locus for arCRD: mapping to 8p11, estimation of frequency, evaluation of a candidate gene Investigative ophthalmology & visual science. , 2001; 42(11): 2458-65.
Lerner, LE Gribanova, YE Ji, M Knox, BE Farber, DB   Nrl and Sp nuclear proteins mediate transcription of rod-specific cGMP-phosphodiesterase beta-subunit gene: involvement of multiple response elements The Journal of biological chemistry. , 2001; 276(37): 34999-5007.
Farber Debora B, Tsang Stephen H   Stationary night blindness or progressive retinal degeneration in mice carrying different alleles of PDE gamma Frontiers in bioscience : a journal and virtual library, 2003; 8(1): s666-75.
Saghizadeh Mehrnoosh, Brown Donald J, Tajbakhsh Jian, Chen Zugen, Kenney M Cristina, Farber Debora B, Nelson Stanley F   Evaluation of techniques using amplified nucleic acid probes for gene expression profiling Biomolecular engineering, 2003; 20(3): 97-106.
Farber Debora B, Katsman Diana   Embryonic Stem Cell-Derived Microvesicles: Could They be Used for Retinal Regeneration? Advances in experimental medicine and biology, 2016; 854: 563-9.
Ahmedli Novruz B, Gribanova Yekaterina, Njoku Collins C, Naidu Akash, Young Alejandra, Mendoza Emmanuel, Yamashita Clyde K, Ozgül Riza Köksal, Johnson Jerry E, Fox Donald A, Farber Debora B   Dynamics of the rhomboid-like protein RHBDD2 expression in mouse retina and involvement of its human ortholog in retinitis pigmentosa The Journal of biological chemistry, 2013; 288(14): 9742-54.
Young Alejandra, Wang Ying, Ahmedli Novruz B, Jiang Meisheng, Farber Debora B   A constitutively active Gαi3 protein corrects the abnormal retinal pigment epithelium phenotype of Oa1-/- mice PloS one, 2013; 8(9): e76240.
Katsman Diana, Stackpole Emma J, Domin Daniel R, Farber Debora B   Embryonic stem cell-derived microvesicles induce gene expression changes in Müller cells of the retina PloS one, 2012; 7(11): e50417.
Mokhonov Vladislav V, Theendakara Veena P, Gribanova Yekaterina E, Ahmedli Novruz B, Farber Debora B   Sequence-specific binding of recombinant Zbed4 to DNA: insights into Zbed4 participation in gene transcription and its association with other proteins PloS one, 2012; 7(5): e35317.
Saghizadeh Mehrnoosh, Gribanova Yekaterina, Akhmedov Novrouz B, Farber Debora B   ZBED4, a cone and Müller cell protein in human retina, has a different cellular expression in mouse Molecular vision, 2011; 17(5): 2011-8.
Young Alejandra, Jiang Meisheng, Wang Ying, Ahmedli Novruz B, Ramirez John, Reese Benjamin E, Birnbaumer Lutz, Farber Debora B   Specific interaction of Gαi3 with the Oa1 G-protein coupled receptor controls the size and density of melanosomes in retinal pigment epithelium PloS one, 2011; 6(9): e24376.
Kitamura Eiko, Gribanova Yekaterina E, Farber Debora B   Regulation of retinoschisin secretion in Weri-Rb1 cells by the F-actin and microtubule cytoskeleton PloS one, 2011; 6(6): e20707.
Parry David A, Toomes Carmel, Bida Lina, Danciger Michael, Towns Katherine V, McKibbin Martin, Jacobson Samuel G, Logan Clare V, Ali Manir, Bond Jacquelyn, Chance Rebecca, Swendeman Steven, Daniele Lauren L, Springell Kelly, Adams Matthew, Johnson Colin A, Booth Adam P, Jafri Hussain, Rashid Yasmin, Banin Eyal, Strom Tim M, Farber Debora B, Sharon Dror, Blobel Carl P, Pugh Edward N, Pierce Eric A, Inglehearn Chris F   Loss of the metalloprotease ADAM9 leads to cone-rod dystrophy in humans and retinal degeneration in mice American journal of human genetics, 2009; 84(5): 683-91.
Jacobson Samuel G, Sumaroka Alexander, Aleman Tomas S, Cideciyan Artur V, Danciger Michael, Farber Debora B   Evidence for retinal remodelling in retinitis pigmentosa caused by PDE6B mutation The British journal of ophthalmology, 2007; 91(5): 699-701.
Yellore Vivek S, Papp Jeanette C, Sobel Eric, Khan M Ali, Rayner Sylvia A, Farber Debora B, Aldave Anthony J   Replication and refinement of linkage of posterior polymorphous corneal dystrophy to the posterior polymorphous corneal dystrophy 1 locus on chromosome 20 Genetics in medicine : official journal of the American College of Medical Genetics, 2007; 9(4): 228-34.
Tsang Stephen H, Woodruff Michael L, Jun Lin, Mahajan Vinit, Yamashita Clyde K, Pedersen Robert, Lin Chyuan-Sheng, Goff Stephen P, Rosenberg Thomas, Larsen Michael, Farber Debora B, Nusinowitz Steven   Transgenic mice carrying the H258N mutation in the gene encoding the beta-subunit of phosphodiesterase-6 (PDE6B) provide a model for human congenital stationary night blindness Human mutation, 2007; 28(3): 243-54.
Piri Natik, Mendoza Emmanuel, Shih Jennifer, Yamashita Clyde K, Akhmedov Novrouz B, Farber Debora B   Translational regulation of the rod photoreceptor cGMP-phosphodiesterase: the role of the 5'- and 3'-untranslated regions Experimental eye research, 2006; 83(4): 841-8.
Linberg Kenneth A, Fariss Robert N, Heckenlively John R, Farber Debora B, Fisher Steven K   Morphological characterization of the retinal degeneration in three strains of mice carrying the rd-3 mutation Visual neuroscience, 2006; 22(6): 721-34.
Acosta Monica L, Fletcher Erica L, Azizoglu Serap, Foster Lisa E, Farber Debora B, Kalloniatis Michael   Early markers of retinal degeneration in rd/rd mice Molecular vision, 2005; 11(6): 717-28.
Piri Natik, Gao Yong Qing, Danciger Michael, Mendoza Emmanuel, Fishman Gerald A, Farber Debora B   A substitution of G to C in the cone cGMP-phosphodiesterase gamma subunit gene found in a distinctive form of cone dystrophy Ophthalmology, 2005; 112(1): 159-66.