Past Award Recipients

 2008 Recipients

Jacque Duncan, M.D.

Beckman Vision Center, University of California - San Francisco

Project:  High resolution retinal imaging in patients with retinal degenerations
Summary: Dr. Duncan and her team will correlate the images of retinal structure produced by the Adaptive Optics Scanning Laser Ophthalmoscope (AOSLO) with standard measures of retinal structure created with an instrument known as Optical Coherence Tomography (OCT).  OCT images provide information about retinal structure in cross-section, while AOSLO gives en face images of individual cones.  By combining these two techniques Dr. Duncan hopes to learn more about how changes in cone structure relate to vision loss in patients with retinal degenerations.  In addition, Dr. Duncan will correlate the images of cone structure obtained using AOSLO with standard clinical measures of retinal function used in Ophthalmology clinics, including visual acuity, automated perimetry, fundus photography including fundus autofluorescence and multifocal electroretinography (mfERG), to learn how well the cones work or are able to see.

Gustavo Aguirre, D.V.M., Ph.D.

Dept of Genetics, School of Veterinary Medicine, University of Pennsylvania

Project: Developing Retinal Therapies in Models of Retinal Degeneration
Proof of principle studies in retinal disease models have demonstrated that experimental therapies can be evaluated and transitioned into clinical trials for retinal diseases in human patients (e.g., gene therapy for LCA; CNTF via encapsulated cell based therapy technology). Dr. Aguirre will continue to use disease relevant models to develop or evaluate novel therapies that have the potential for restoring sight or preventing/slowing down the loss of vision in human patients.

Muayyad Al-Ubaidi, Ph.D.

Dept of Cell Biology, University of Oklahoma

Project:  To evaluate pharmacological and neurotrophic agents to rescue photoreceptor cells in RP animal models.
Summary: Since a greater proportion of retinal degenerative diseases are still of unknown causes, there exist the need to further study other aspects of normal retinal function. Furthermore, in most cases where the mutation is known, it is not known how that mutation has altered the function of the protein leading to the degenerative disease phenotype. Dr. Al-Ubaidi and his team chose to understand how modulations in a post-translational modification can alter retinal function. The gained knowledge will help them understand how a protein functions and therefore, how a mutation would alter that function leading to a blinding disease.  They will identify all sulfated retinal proteins and will choose those that have been involved in retinal disease for further study. They will then study how lack of sulfation altered that function. This will allow them to develop strategies, gene based or pharmacological, to counter the effects of the mutation.

Tomas Aleman, M.D.

Scheie Eye Institute, University of Pennsylvania

Project: Retinitis Pigmentosa: Paving the Path to Treatments Through Detailed Understanding of Disease Expression
Summary: Success of ongoing gene therapy trials for Leber Congenital Amaurosis has raised expectations that other forms of inherited retinal degeneration may also be treatable. Detailed characterizations of patients, part of the groundwork that was necessary to initiate these trials, will be performed in other inherited retinal degenerative disorders to increase understanding of human disease expression and mechanism. The knowledge gained will be used to ask specific questions relevant for the planning of future treatment trials for these conditions.

Jonathan Ash, Ph.D.

Dept of Ophthalmology, University of Oklahoma

Project:  To perform research on neuroprotection that would be relevant to genetic photoreceptor degenerations
Summary: In most cases of retinal degeneration, photoreceptor death is the result of long-term inflammation, exposure to environmental insults, and genetics. While disease causing genes are present before birth, patients with retinitis pigmentosa or age related macular degeneration typically do not develop disease for 50 to 80 years. The protracted time to develop symptoms suggests that retinal neurons have an endogenous mechanism for protection from chronic injury. The main focus of Dr. Ash’s work is to identify the mechanism of stress-induced endogenous protection of photoreceptors, and once identified, he and his team will develop these protective mechanisms into new therapeutics, with the goal of delaying or preventing blindness resulting from inherited retinal degenerations.

Joseph Carroll, Ph.D.

Eye Institute, Medical College of Wisconsin

Project: Advanced Retinal Imaging and Improving the Success of Gene Therapy
Summary: Dr. Carroll and his team will use adaptive optics, allowing them to see individual photoreceptor cells in the retina, and optical coherence tomography, allowing them to see the layers of the retina.  This information will be combined with genetic data and clinical data on each patient to develop a high-resolution genotype-phenotype correlation that carries with it significant predictive power with regard to treating this and other retinal degenerations.

Arlene Drack, M.D.

University of Iowa Hospitals and Clinics

Project:  To perform clinically-relevant molecular genetics research in syndromic and non-syndromic RP
Summary: Dr. Drack’s research is focused on treatment strategies for mouse models of human retinitis pigmentosa and Bardet Biedl syndrome (BBS).  Dr. Drack’s group was the first to demonstrate genetic heterogeneity of BBS by identifying linkage to three different chromosomal locations in three large consanguineous tribes of Bedouin Arabs from Israel.  Furthermore, her laboratory has independently identified seven of the twelve known BBS genes and recently developed knockout mouse models of many of those genes.  In the next few years, Dr. Drack and her team intend to develop and test viral-mediated gene replacement therapy in mouse models of these diseases; and clinically and molecularly characterize a large number of patients affected with BBS to establish the natural history of the vision loss in this disease as well as to identify a large number of individuals who could be invited to participate in a future human clinical trials of gene therapy for these diseases.

Erica Farber, Ph.D.

Jules Stein Eye Institute, University of California – Los Angeles

Project Title: Characterization of microRNAs in stem cell microvesicles
Summary: Fish and amphibians retain stem cells with the ability to regenerate the retina after injury.  However, mammals have lost this ability. Recently, dormant stem cells were discovered in the mammalian eye.  Further elucidation of the communication role of microvesicles within stem cell niches may reveal ways for microvesicles to awaken these dormant stem cells and lead to retina repair. Additionally, microvesicles released from cells engineered to express mRNA, proteins, or siRNA may be useful to deliver these small molecules to the eye.  Dr. Farber and her collegues will characterize the micro RNAs in stem cell microvesicles and establish a way to use microvesicles as vehicles for the transfer of specific small molecules to retinas affected by degenerative disease.

Jeffrey Goldberg, M.D., Ph.D.

Bascom Palmer Eye Institute, University of Miami

Project: Research leading to the development of therapeutic tools for photoreceptor diseases
Summary: Retinal degenerations such as retinitis pigmentosa often end with the death of retinal neurons such as rod and cone photoreceptors. Although it may be possible to salvage these cells before they die, for the many patients who have lost these cells, we must figure out a way to replace them. Little is known about how to harvest retinal stem cells, how to direct them to the proper location in the retina, how to push them to differentiate into photoreceptors, and how to integrate them into the retina. Dr. Goldberg and his team are now attempting to address retinal degenerative disease by combining cell culture and nanotechnology to provide novel approaches to stem cell and cell replacement therapy. Dr. Goldberg’s hope with these experiments is to enhance the efficacy of stem cell and photoreceptor transplantation and integration into the retina, and thereby bring back vision in retinal degenerative diseases.

Michael Grassi, MD

University of Chicago

Project: Collaborative effort to facilitate the discovery and testing of effective compounds to treat retinal degenerative disease.
Summary: Dr. Grassi has developed a multidisciplinary, inter-institutional collaboration between basic scientists and clinicians in Chicago to investigate the mechanisms that may result in apoptotic photoreceptor death in RP.  Using a cellular model of RP, the research team will screen hundreds of thousands of small molecules to identify those compounds and genes that retard or prevent apoptosis.   In addition, the cell culture system will enable a genome scale screen using RNA interference to assess the role of over 25,000 individual genetic perturbations in photoreceptor cell survival.  Results from the screenings will be extended to animal models, which will enable the development and introduction of new therapies to better treat, and perhaps even prevent RP.

Neena Haider, PhD

University of Nebraska

Project: Research into genetic modifiers of retinal degenerations
Summary: Dr. Haider’s research uses molecular genetics to identify and characterize genes important in vision loss. The goal of her lab is to identify novel genes associated with retinal disease, determine the gene networks that regulate retinal stem cells, and identify modifier genes that can prevent retinal degeneration and restore vision in degenerating retinas. The goal of this project is to evaluate the efficacy of molecular and genetic modifiers in a degenerating retina. Her studies will greatly enhance understanding of genetic factors that influence severity of retinal disease, and provide potentially powerful targets for improved therapies to treat or prevent multiple forms of retinal disease.

Yuk Fai Leung, Ph.D.

Dept of Biological Sciences, Purdue University

Project:  Research in gene regulatory networks in retina and RPE
There have been many research studies that have identified the underlying genetic causes of retinal degenerative diseases. However, without a fundamental understanding of the development of retina and retinal pigment epithelium (RPE), it would be difficult to elucidate the developmental mechanisms that are altered by these genes, not to mention designing effective treatments. Dr. Leung and his team are studying a novel irx7 (gene) regulatory network and its function in retinal development of zebra fish. Dr. Leung’s work will (1) elucidate the extent to which irx7 is regulating cellular differentiation in retina, and (2) clarify a framework of the irx7 regulatory network. This will greatly facilitate the investigation of molecular controls of normal zebra fish retinal development, which will in turn establish a strong scientific foundation for studying disease genes that cause retinal degeneration, and ultimately assist the design of better treatments for retinal degeneration.

Kristina Narfstom, D.V.M.

Dept of Veterinary Medicine, University of Missouri

Project: Research in large animal models of retinal degeneration with an emphasis on proof-of-concept
Summary:  Dr. Narfstrom is investigating the implantation of light-sensing microchips in the retinas of animal models. The chips contain thousands of miniature solar cells that turn light into electrical current and early studies show that they may have even further benefits for people with RP in that they seem to actually slow progression of the disease.  In addition, Dr. Narfstrom hopes to use gene replacement therapy to restore sight in the same animal model. Success with animal models would pave the way for using this approach in people with RP.  Narfstrom already has had success using gene therapy to restore sight to French sheepdogs that suffer from another inherited retinal disorder. The dogs, called Briards, are born with night blindness and poor daylight vision that get progressively worse with age.

David Pepperberg, PhD

University of Illinois at Chicago

Project: Exploring the use of nanotechnology to restore vision to damaged retinal cells.
Photoreceptor degenerative diseases such as age-related macular degeneration (AMD) destroy the ability of rod and cone photoreceptors to respond to light and to transmit visual signals to “post-photoreceptor” nerve cells in the inner layers of the retina.  However, the post-photoreceptor nerve cells themselves often appear to remain healthy in the diseased retina.  As a possible therapy for AMD and related retinal diseases, Dr. Pepperberg and his colleagues are working to develop implantable, nanoscale molecular structures that can directly stimulate the post-photoreceptor nerve cells in response to light, and thus bypass the non-functioning photoreceptors.  The immediate focus of their research is to develop prototypes of the desired molecular device, and to test the activities of these prototypes in two kinds of biological systems: (1) single, isolated cells that have been engineered to express a given type of postsynaptic receptor protein; and (2) retinal tissue obtained from animal models.  We anticipate that this research will identify structures ultimately suitable for testing in human subjects.

Vladlen Slepak, Ph.D.

Bascom Palmer Eye Institute, University of Miami

Project: The role of light-dependent movement of transducin in retinal rods.
Summary: Bright light damages photoreceptor cells because the eye focuses reflected sunlight on the retina.  It is also known that light can exacerbate retinal degeneration. The exact molecular mechanisms that protect photoreceptor cells and the reasons these protective mechanisms malfunction in disease are not completely understood.  Several years ago scientists discovered that a crucial protein responsible for light reception, transducin, which in darkness localizes to the rod outer segments, and then re-localizes across the cell in bright light.  Dr. Slepak’s research will uncover new information about photoreceptor cell biology that will allow us to understand how rod cells protect themselves from damaging levels of light. Specifically, this project will test the current hypothesis that the translocation of transducin to the inner compartments of rod cells has a cytoprotective function.  Potentially, this research will identify novel proteins that influence cell survival, which can be targeted pharmacologically or through gene therapy.

Alexander Sumaroka, Ph.D.

Scheie Eye Institute, Univ of Pennsylvania

Project:  Retinal Mapping For Targeting Treatment in Retinitis Pigmentosa
Summary: Inherited retinal degenerations in the family of diseases known as retinitis pigmentosa will be studied with optical coherence tomography, mainly to inquire about the integrity of the photoreceptor layer. Structural abnormalities of the inner retina, suggesting the process of retinal remodeling, will also be defined. The present work will use state-of the-art high-resolution instrumentation. Mapping of the photoreceptor layer has already been used in the University of Pennsylvania gene therapy clinical trial to target appropriate retinal regions for treatment and this approach has proven valuable.

Veena Theendakara, Ph.D.

Jules Stein Eye Institute, UCLA

Project:  To perform research into novel cone photoreceptor gene and mechanisms of
ZBED4, a novel protein with mutations causing cone-rod dystrophy
Summary: Dr. Theendakara and her colleagues have recently identified ZBED4, a novel protein present in the nucleus and cytoplasm of cone photoreceptors. In addition, they found a mutation in the ZBED4 gene associated with disease in patients with cone-rod dystrophy.  The objectives of their work are to investigate the movement of the ZBED4 protein between the nucleus and cytoplasm of cone photoreceptor cells; and to screen patients for mutations in the ZBED4 gene and establish genotype-phenotype correlations for early detection of disease and future intervention.

2007 Recipients

Novrouz Akhmedov, Ph.D.
Jules Stein Eye Institute, UCLA School of Medicine
Dr. Novrouz Akhmedov is in the research series at UCLA (Assistant Research Ophthalmologist, Step III). His work involves the characterization of a gene that he has recently isolated, 7R, and its protein product. A mutation in this gene cosegregates with disease in a family affected with arRP. He is trying to determine the mechanism by which mutations in 7R cause retinal degeneration. He is also planning to screen the DNA of the collection of arRP cohorts to find other mutations in 7R and thereby establish it as a novel arRP locus. His studies will increase the understanding of the disease caused by abnormal 7R and allow the design of strategies for the prevention or cure of this retinal degeneration.

Tomas S. Aleman, M.D.
University of Pennsylvania
Dr. Aleman, Research Assistant Professor of Ophthalmology at the University of Pennsylvania School of Medicine, is a retinal degeneration expert at the Scheie Eye Institute. His work includes pre-clinical and clinical experiments to further knowledge of disease mechanisms in inherited retinal degenerations en route to treatment. Most recently, he has made contributions to understanding the natural history of common molecular-defined forms of retinitis pigmentosa and Leber congenital amaurosis.

Dan Chung, Ph.D.
University of Pennsylvania
Dr. Daniel Chung aims to develop his career as a pediatric retinal degeneration specialist and, in this capacity, will bridge the gap between laboratory research and development of treatments for blinding retinal diseases. Dr. Chung is working to develop both non-viral and virus-based gene transfer to treat early onset retinal degeneration. He is evaluating novel non-viral gene transfer approaches that could potentially minimize immunologic complications of gene transfer. In addition, he is involved in testing virus-based gene transfer strategies in a number of different genetic models of early onset retinal degeneration, including retinitis pigmentosa, Leber congenital Amaurosis, Stargardt disease and Usher Syndrome.

Artur V. Cideciyan, Ph.D.
University of Pennsylvania
Dr. Cideciyan is a unique and key contributor to the field of retinal degenerations. He is internationally recognized for his expertise in the use of state-of-the-art non-invasive methods to understand causes for the loss of vision in patients with retinal degenerations. His expertise is wide ranging and includes electrophysiological, psychophysical, imaging and mathematical methods. Dr. Cideciyan’s strong quantitative background together with his deep understanding of retinal physiology has allowed his studies to bridge the enormous gap between molecular abnormalities and loss of visual function. What sets him particularly apart is his innovative multidisciplinary approach to testing hypotheses of scientific and clinical importance to patients. In addition to the clinical research in molecularly characterized patients, Dr. Cideciyan also studies genetically-engineered or naturally occurring animal models of inherited retinal degenerations with methodology that can be specifically compared to that in human work. This tactic has immensely helped with the interpretation of patient results.

Jacque Duncan, M.D.
Beckman Vision Center, University of California - San Francisco
Dr. Duncan is a talented clinician scientist who is setting up her independent laboratory at UCSF to study retinal degenerative disease. She has trained with Dr. Matthew LaVail in the area of neuroprotective mechanism for photoreceptor and RPE rescue in retinal disease.

Jeffrey Goldberg, M.D., Ph.D.
Bascom Palmer Eye Institute, University of Miami
Dr. Goldberg’s lab works on keeping neurons alive, and replacing retinal neurons using new nanotechnology approaches with stem cells. Recently, the lab began purifying stem cells from human donor eyes, keeping them alive in culture, and loading them with nanoparticles to direct them to specific locations in the eye, for example, to replace photoreceptors. The goal is to use this work to complement work on stem cells and neuroprotection, and to make rapid advances in the lab that can be translated for human use.

Byron Lam, M.D.
Bascom Palmer Eye Institute, University of Miami
Dr. Lam’s main research objectives are: 1) to develop and test treatments for retinal degeneration and 2) to develop new methods of assessing retinal health and function in retinal degeneration and to correlate these findings with specific disease-causing genetic alterations. His lab is currently involved in testing neurotrophic factor treatment (CNTF trial) and stem cell treatment (CNTO trial) and will collaborate with investigators in Philadelphia for gene therapy (RPE65 AAV trials). The lab also aims to develop novel techniques such as the wide-field three-dimensional optical coherence tomography (OCT) to examine anatomical changes in retinal degenerations in infants and adults. They also plan to release a prototype database program for phenotypic-genotypic correlation to facilitate clinical research and patient selection for clinical trials.

Alan Mears, Ph.D.
Department of Ophthalmology, University of Ottawa
Dr. Alan Mears is working on elucidating transcriptional regulatory networks required for photoreceptor development and function, and his studies may yield great insight into the development of future targetted therapies. His research may have a direct impact on stem cell research, as he is working to find the factors/molecules that guide photoreceptor differentiation, which could then be applied into triggering stem cells to differentiate into new photoreceptor cells. His lab utilizes many techniques of genetics, cell/molecular biology, biochemistry, genomics, and bioinformatics.

Daniel V. Palanker, Ph.D.
Stanford School of Medicine
Dr. Palanker is trained in physics and lasers, and is a rising star in the field of electrical field interactions with biological tissue. This has applications in retinal surgery (he invented the PEAK surgical device), in gene therapy (electroporation), and perhaps most interestingly, in the area of retinal prosthesis. His lab has been doing tremendous work over the past couple of years and is both highly innovative and cutting edge, as well as extremely prolific.

David R. Pepperberg, Ph.D.
University of Illinois at Chicago
Dr. Pepperberg is the Director of the Photoreceptor Research Laboratory in the Department of Ophthalmology & Visual Science at the University of Illinois at Chicago. He is an established, internationally recognized visual biochemist who has made seminal contributions to our understanding of mechanisms underlying phototransduction and photoreceptor adaptation. Recently, Dr. Pepperberg significantly expanded the scope of his research by taking the lead role in developing a program project grant to explore the use of nanotechnology to restore vision to damaged retinal cells. To do this, Dr. Pepperberg assembled a multidisciplinary group of scientists that includes experts in the fields of bioengineering, chemistry, molecular biology, ophthalmology, pharmacology, and physics. Working together this group will develop a nanotech structure that can be introduced into the eye and repair vision loss.

Baerbel Rohrer, Ph.D.
Medical University of South Carolina
Dr. Rohrer is pursuing important work on mechanisms of degeneration and rescue in RPE65 knockout mice.

Steven Tsang, M.D., Ph.D.
Department of Ophthalmology, Columbia University
Dr. Stephen Tsang’s studies involve manipulation of genes that cause photoreceptor degeneration in mice and humans. His goal is to control the expression of the phosphodiesterase-6 gamma gene in mice by using inducible gene targeting, which will disrupt the activity of a gene in a specific tissue at the desired time during the life of the mouse. By following the effects of the genetic abnormality after the photoreceptors have fully developed, he hopes to gain an understanding of the early events controlling photoreceptor signaling and degeneration in mice, which could lead to new drug targets for the prevention or delay of human retinal degenerations. Gaining temporal and spatial control of gene expression is essential for the elucidation of gene function in the whole organism. The reagents that Dr. Tsang will develop could be built into gene therapy vectors to provide temporal and spatial control of gene expression. An inducible gene targeting system can be used to address several previously unapproachable problems in sensory biology as well as gene therapy.

Rong Wen, M.D., Ph.D.
Bascom Palmer Eye Institute, University of Miami
The long-term objectives of Dr. Wen’s research are to understand the mechanism of retinal degeneration and to develop new therapeutic strategies. One of his current projects is to study cone photoreceptor degeneration. In the late stages of retinitis pigmentosa, it is the cone degeneration that causes severe vision loss. How to save cones is a major challenge to the retinal degeneration research community. One of the obstacles is the lack of cone degeneration experimental models. Dr. Wen’s lab is characterizing cone degeneration in a transgenic rat with retinal degeneration, which can serve as a good model of cone degeneration. Using this model, he has found that CNTF (ciliary neurotrophic factor) is able to stimulate cones to regenerate outer segments. That means that, at least in this rat model, CNTF is able to reverse cone degeneration and restore the functionality of cones. With such results, Dr. Wen endeavors to help the research community to think about restoration and maintaining useful vision instead of merely slowing the progression of disease.

Michael Grassi, M.D.
University of Chicago
Dr. Grassi is developing a collaborative effort to facilitate the discovery and testing of effective compounds to treat retinal degenerative disease. The project will involve multiple investigators based at three different institutions.

Wen-Hsiang Lee, Rong Wen and Elizabeth Fini
Bascom Palmer Eye Institute, University of Miami
Stem cells might accomplish retinal repair if they can differentiate into photoreceptors and make the proper synapses with other retinal cells. SanBio’s proprietary bone-marrow-derived human neural progenitor cells (SB623) are currently being developed for central nervous system indications and might be also useful for therapy of retinal degenerations. In order to determine the neuroprotective and regenerative properties of SB623 cells in the degenerating neural retina, this team will delineate the survival, integration, and differentiation of SB623 cells in three well-characterized rodent retinal degeneration models: The rat S334ter-3 and S334ter-4 models (rhodopsin mutation) and a retinal degeneration (rd1) mouse model (rod photoreceptor-specific cGMP phosphodiesterase mutation). Future plans are to explore environmental interactions that might enhance survival, integration, and differentiation of these and other candidate cell lines using knock out mouse and chemical biology approaches.

Ronald Carr, M.D. and William Seiple, Ph. D.
New York University
This grant will support retinal research at New York University’s Department of Ophthalmology.

2006 Recipients
Tomas Aleman, MD
Dr. Aleman is a Research Assistant Professor of Ophthalmology at the University of Pennsylvania School of Medicine. He is a retinal specialist and a retinal degeneration clinical expert at the Scheie Eye Institute and is actively involved in pre-clinical experimental efforts that seek inherited retinal degeneration disease mechanisms.

Sanjoy K. Bhattacharya, PhD
Dr. Bhattacharya is a young neuroscientist who recently joined the Bascom Palmer Eye Institute from the Cleveland Clinic and who furthers his research interests in degenerative diseases of the eye by applying a multidisciplinary approach to his work with particular focus on the use of proteomics to identify biomarkers and therapeutic targets.

Janet C. Blanks, PhD
Dr. Blanks is a senior neuroscientist who recently moved to Florida Atlantic University in Boca Raton; whose career has been dedicated to the study of retinal degenerative disease; and who has recently begun to focus on developing novel therapeutics that make use of gene therapy tools.

Sander R. Dubovy, MD
Dr. Dubovy is a young retina specialist and an ophthalmic pathologist who also serves as medical director of the Lion’s Eye Bank at the Bascom Palmer Eye Institute; whose research interest is to establish clinico-pathologic correlation in eye diseases; and whose long-term interest is in inherited eye diseases, including retinitis pigmentosa.

Dana Garcia, PhD
Professor, Department of Biology, Texas State University. Dr. Garcia’s research is directed toward understanding mechanisms by which the retina communicates with the retinal pigment epithelium (RPE). Findings from Dr. Garcia’s research will offer new directions for developing therapeutics that could enhance survival of rod photoreceptors in retinal degenerative disease.

Neena B. Haider, PhD
Dr. Haider is an assistant professor of genetics and biochemistry at the University of Nebraska. She works on the regulatory gene Nr2e3 that controls cone development and which when mutated causes a cone rod dystrophy.

Byron Lam, MD, PhD
Dr. Lam is a neuro-ophthalmologist who serves as Professor of Ophthalmology. His research interests cover the areas of visual electrophysiology, visual function tests, clinical neuro-ophthalmology, hereditary retinal disease, and population-based visual impairment. He is known in the eye and vision research community for his contributions to the field of clinical neuro-ophthalmology and electrophysiology. Dr. Lam currently serves as head of the Florida Inherited Eye Disease Project, the cross-state component of the nation-wide genetic testing network led by Howard Hughes investigator Dr. Edwin Stone.

Yun-Zheng Le, PhD
Dr. Le is an assistant professor at the University of Oklahoma Health Science Center. He works in the area of targeted cell specific knockouts and has recently made a number of lines of mice that can be used for targeted knockouts of retinal pigment epithelial cell specific genes.

Wen-Hsiang Lee, MD, PhD
Dr. Lee is a young retina specialist and molecular biologist who recently joined Bascom Palmer from Johns Hopkins University to develop an integrated clinical/laboratory research program in inherited retinal degeneration.

Zhuo-Hua Pan, PhD
Associate Professor, Department of Anatomy & Cell Biology, Wayne State University School of Medicine. Dr. Pan is looking for new strategies to treat blinding retinal degenerations. In his lab, he has converted inner retinal neurons into light sensors that could restore light sensitivity to retinas lacking photoreceptors. Importantly, the neurons generated light signals that were transmitted to visual cortex. Dr. Pan’s approach to treating retinal degenerative disease has the advantage of not introducing foreign tissue or devices thus avoiding immune reactions or biocompatibility issues.

Neal S. Peachey, PhD
Dr. Peachey is a research scientist at the Cole Eye Institute of the Cleveland Clinic Foundation and Research Service of the Cleveland VA Medical Center. Dr. Peachey has identified two different mouse models of the human condition, congenital stationary night blindness (CSNB1 and CSNB2). These mice carry mutations in the same genes that are involved in the respective human conditions. Dr. Peachey’s continuing studies will help define the roles of these affected proteins in retinal function, thus suggesting new avenues for treatment.

Vladen Z. Slepak, PhD
Dr. Slepak is a neuroscientist based in the Department of Molecular and Cellular Pharmacology at University of Miami who is investigating basic molecular mechanisms of signal transduction in order to understand how molecules involved in photo-transduction relocate within rod photoreceptors in response to light with the goal of providing new direction for developing therapeutics to bolster adaptation and rod survival in retinal degenerative disease.

Tonia Rex, PhD
Dr. Rex is a post-doctoral fellow in Dr. Jean Bennett’s lab at the University of Pennsylvania. Her work is on retinal degeneration, retinal toxicity (protein-induced and light-induced), and gene therapy treatments for retinal disease. Her publication record is very strong and she is poised to make major contributions to this field.

Nancy K. Wills, PhD
Professor, Department of Neuroscience and Cell Biology, University of Texas Medical Branch. Dr. Wills is an internationally recognized physiologist who has made seminal contributions in the field of epithelial ion channels. Dr. Wills uses a multidisciplinary approach that includes electrophysiology, molecular cloning and optical imaging. Her present research focuses on two proteins (1) ClC-3-type chloride channels in RPE, and (2) IMPDH, an enzyme that catalyzes the first unique step in GMP.
Mutations in IMPDH and loss of ClC-3 channels both lead to photoreceptor degeneration. Better understanding of the regulators of ClC-3 channels and IMPDH could reveal new drug therapies for retinal degenerative disease.