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SIGNIFICANT ITEMS IN HOUSE, SENATE, AND CONFERENCE APPROPRIATIONS COMMITTEE REPORTS

FY 2006 House Appropriations Committee Report Language (H. Rpt. 109-143)

Item
Diabetic retinopathy - The Committee commends NEI for its continued support of the diabetic retinopathy clinical research network. The Committee encourages NEI to expand this network through collaboration with other institutes, such as NIBIB, and private and public partners and through the introduction of new treatments to prevent, treat, or cure diabetic retinopathy. (p.39)

Action taken or to be taken
NEI continues to fund the Diabetic Retinopathy Clinical Research Network (DRCRnet). The NEI supports the DRCRnet efforts to actively pursue additional research and funding collaborations with other NIH institutes and private and public partners. In addition to the NEI support, the DRCRnet has received funding from the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) and Allergan, Inc. The Juvenile Diabetes Research Foundation International has also recently committed additional funds to help support the network. As of September 2005, the network has developed seven clinical protocols and is actively pursuing identification and design of new clinical studies.

FY 2006 Senate Appropriations Committee Report Language (H. Rpt 109-103)

Item
Basic Behavioral and Social Sciences Research - The Committee encourages NEI to participate in trans-institute initiatives organized by OBSSR or another institute to strengthen basic behavioral research and enhance opportunities for behavioral science research training. (p.127)

Action taken or to be taken
The NEI is an active participant in the ongoing program announcement (TPA-04-014), "Research Partnerships for Improving Functional Outcomes". The objective of the PA is to support interdisciplinary medical and psychosocial research related to rehabilitation of individuals with chronic conditions which includes individuals who are blind or have low vision. The NEI organized and sponsored a workshop, Blindness and Spatial Functioning. The aim of this workshop was to consider changes in scientific thinking about blindness from the brain and cognitive sciences and consider educational, rehabilitative, and behavioral implications for people who are blind or have low vision. This has generated research applications in this area. The NEI is a principal participant in the NIH Neurosciences Blueprint (BP) which has a number of initiatives in behavioral-related research including "The NIH Unified Measures of Neural Health." This initiative addresses a need cited by the NIH Cognitive and Emotional Health Project (CHEP) and aims to establish uniformity for measures of cognitive, sensory and motor function and deficits across NIH BP Institutes. The NEI also participates in the BP initiative, "The Pediatric MRI study of Normal Brain Development." The data collected from this project will enable the study of normal brain development including the development of cognitive function.

NEI senior staff has met with the new Director of the NIH Office of Basic Behavioral and Social Sciences Research in order to explore areas for future collaboration. The NEI plans to participate in joint Program Announcements and consider possible avenues for co-funding.

Item
Ocular Albinism - The Committee recognizes recent advances in the treatment of ocular albinism by using gene therapy. The Committee encourages the National Eye Institute to favorably consider research grant proposals that seek to expand upon these important findings. (p.128)

Action taken or to be taken
The X-linked form of albinism (ocular albinism type 1) in humans is associated with the OA1 gene. OA1 is involved with the production of melanin pigments in cellular organelles called melanosomes. Affected patients have normal skin pigmentation, but suffer from debilitating eye problems such as nystagmus or rapid eye movements and misrouting of nerves from the eye to the brain. In the past year, NEI-supported research has led to a significant new insight into the biological role of the OA1 gene in the pathogenesis of ocular albinism. Scientists have studied a mutated form of the gene in a mouse model. They examined the retinal pigment epithelium (RPE) during embryonic development and after birth. They found that OA1 is involved in pigment regulation at two distinct steps. In early embryogenesis, the gene controls the abundance of melanosomes in the RPE. But after birth, OA1 plays a maintenance function which controls melanosome size.

These scientific findings allow a deeper understanding of the molecular mechanisms which underlie the OA phenotype caused by the lack of this protein. The goal is that the mutated form of the gene can be corrected through application of gene transfer technology. To accomplish this, NEI will fund a new R03 grant titled "AAV-Mediated Gene Correction in Retina" to test a novel method of gene targeting to improve gene therapy. This kind of study, because it aims to correct a specific mutation, will have application to a wide spectrum of retinal diseases including ocular albinism.



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