the monaciano symposium - Fondo Studenti Italiani
Transcription
the monaciano symposium - Fondo Studenti Italiani
THE MONACIANO SYMPOSIUM PONTE A BOZZONE SIENA, ITALY OCTOBER 1–4, 2013 THE MONACIANO SYMPOSIUM A Strategic Planning Retreat to Advance the Treatment of Hereditary Retinal Degeneration Dear Participants, On behalf of Celeste and Bruno Piperno, the symposium organizing committee would like to welcome you to Tenuta di Monaciano. The beautiful villa, houses, and grounds of this estate are the site for our meeting of experts focused on the development of treatments for retinal dystrophies and degenerations. The past ten years have seen tremendous progress toward our shared goals. Genereplacement therapy, and to some extent stem-cell therapy, have been transformed from a dream into an evolving reality. As a result, the field has reached a critical juncture, with many challenges and opportunities ahead. The Monaciano Symposium is aimed at defining these challenges, and at identifying the strategies and solutions needed to move the field forward in the next ten years. The agenda for our meeting was developed to reflect the priorities indicated by your responses to the survey conducted earlier this year. By now meeting together, we hope to further delineate these priorities and to build the consensus needed to solve the critical problems that confront us. A planned outcome of our meeting is a “white paper” that will report our recommendations to the international vision community. In addition, we hope that new opportunities for collaboration will be identified, and that existing collaborations will be deepened as a result of our deliberations. It is thus with sincere pleasure that we welcome this distinguished group to what promises to be a rich and productive meeting where we will create a blueprint for our work in the years ahead. Sincerely, The Symposium Organizing Committee Robin R. Ali, Ph.D. William W. Hauswirth, Ph.D. John R. Heckenlively, M.D. Paul P. Lee, M.D., J.D. Paul R. Lichter, M.D. Alessandro Iannaccone, M.D., M.S. Debra A. Thompson, Ph.D. David N. Zacks, M.D., Ph.D. THE HOSTS OF THE MONACIANO SYMPOSIUM Celeste and Bruno Piperno It has long been a dream of Celeste and Bruno Piperno to host a meeting at their beloved Tenuta di Monaciano that would advance the treatment of retinal degenerative diseases. As steadfast advocates for patients in Rome affected by these diseases, they are known for their personal commitment and dedication to making a difference in the lives of many individuals in their community. Through their close ties and friendships throughout Europe and the United States, Celeste and Bruno have come to understand the importance of international collaborative efforts in advancing translational studies. They identified the goal of bringing together leaders in retinal dystrophy research in an ideal location in which the existing challenges and shared goals needed to move the field forward could be defined. Tenuta di Monaciano is that location, a jewel in the Tuscan countryside complete with working vineyards and an elegant villa that has been in the Piperno family since early in the 20th century. Celeste and Bruno have opened the doors of their villa for our meetings, and are providing meals and accommodations for the participants and guests in the houses of the estate. We owe our deep thanks to Celeste and Bruno for imagining and making the Monaciano Symposium a reality. The most meaningful thanks we can offer will be our progress toward making another of their long-held dreams come true — the successful treatment of retinal dystrophy patients. AGENDA TUESDAY, OCTOBER 1 Reception 3:00 p.m. – 4:00 p.m. Session A 4:00 p.m. – 6:00 p.m. Welcome Participants and guests Alessandro Iannaccone Presentation of the meeting charge Introductory lectures: Progress and challenges John Heckenlively, Debra Thompson, Paul Lichter Dinner at Monaciano 7:00 – 9:00 p.m. Robin Ali, William Hauswirth WEDNESDAY, OCTOBER 2 Session B 8:30 a.m. – 12:30 p.m. Topic 1: Identifying the most compelling therapeutic targets Discussion leaders: Robert Molday, Thomas Reh 8:30 a.m. – 9:00 a.m. Introduction by discussion leaders 9:00 a.m. – 9:50 a.m. Small group discussions Coffee Break 9:50 a.m. – 10:10 a.m. Topic 2: Refining and expanding Discussion leaders: therapeutic strategies John Flannery, David Gamm 10:10 a.m. – 10:40 a.m. Introduction by discussion leaders 10:40 a.m. – 11:30 a.m. Small group discussions 11:30 a.m. – 12:30 p.m. Solution reporting by small group leaders; large group discussion Small group leaders Topics 1 & 2: Lunch 12:30 p.m. – 1:30 pm Isabelle Audo, Alberto Auricchio, Eyal Banin, Deniz Dalkara, Henry Klassen, Enrica Strettoi, Debra Thompson, Richard Weleber Session C 1:30 p.m. – 4:00 p.m. Topic 3: Streamlining regulatory processes and solving infrastructure needs 1:30 p.m. – 2:20 p.m. 2:20 p.m. – 3:00 p.m. Coffee Break 3:00 p.m. – 3:20 p.m. 3:20 p.m. – 4:00 p.m. Panel discussion Leader: David Zacks Members: Robin Ali, William Hauswirth, Naheed Khan, Bart Leroy, Stuart Naylor Panel discussion and Q & A Small group discussions Solution reporting and large group discussion Small group leaders Topic 3: Stephen Daiger, Elise Heon, Thiran Jayasundera, Henry Klassen, András Komáromy, Michel Michaelides, Simon Petersen-Jones, Paul Sieving Dinner/Tour at Castello di Brolio (travel by coach) 5:00 p.m. – 9:30 p.m. THURSDAY, OCTOBER 3 Session D 8:30 a.m. – 12:00 noon Topic 4: Identifying the patients who could benefit from treatment Discussion leaders: Kari Branham, Thiran Jayasundera, Richard Weleber 8:30 a.m. – 9:00 a.m. Introduction by discussion leaders 9:00 a.m. – 9:50 a.m. Small group discussions Coffee Break 9:50 a.m. – 10:10 a.m. THURSDAY, OCTOBER 3 (cont) Topic 5: Formalizing outcome measures Discussion leaders: John Heckenlively, Mark Pennesi, Paul Sieving 10:10 a.m. – 10:40 a.m. Introduction by discussion leaders 10:40 a.m. – 11:30 a.m. Small group discussions 11:30 a.m. – 12:30 p.m. Solution reporting by small group leaders; large group discussion Small group leaders Topics 4 & 5: Eyal Banin, Deniz Dalkara, Alessandro Iannaccone, Naheed Khan, András Komáromy, Bart Leroy, Michel Michaelides, Benedetto Falsini Lunch 12:30 p.m. – 1:30 p.m. Session E 1:30 p.m. – 4:00 p.m. Topic 6: Optimizing collective efforts: Enhancing funding, increasing collaboration, reducing redundancy Panel discussion Leader: Eyal Banin Members: Alberto Auricchio, Stephen Daiger, John Flannery, Elise Heon 1:30 p.m. – 2:20 p.m. 2:20 p.m. – 2:40 p.m. Coffee Break 2:40 p.m. – 3:00 p.m. 3:00 p.m. – 4:00 p.m. Panel discussion Q & A Small group leaders Topic 6: Robin Ali, William Hauswirth, David Gamm, Robert Molday, Simon Petersen-Jones, Rajesh Rao, Thomas Reh, David Zacks Dinner at Monaciano 7:00 – 9:00 p.m. Small group discussions Solution reporting and large group discussion FRIDAY, OCTOBER 4 Session F 8:30 a.m. – 10:30 a.m. Topic 7: Recommendations report planning 8:30 a.m. – 9:00 a.m Topic 1 & 2 small group leaders Discussion leaders: John Heckenlively, Alessandro Iannaccone, Paul Lichter Therapeutic targets and strategies 9:00 a.m. – 9:30 a.m. Topic 3 small group leaders Advancing regulatory and treatment issues 9:30 a.m. – 10:00 a.m. Topic 4 & 5 small group leaders Patient identification/ characterization and outcomes analysis 10:00 a.m. – 10:30 a.m. Topic 6 small group leaders Increasing collaboration and shared funding Departure Airport shuttle available by request 10:30 a.m. – 11:00 a.m. WEDNESDAY AM —Session B (Topics 1 & 2) Red Group Blue Group Green Group Yellow Group Audo (Leader) Auricchio (Leader) Banin (Leader) Dalkara (Leader) Klassen (Leader) Strettoi (Leader) Thompson (Leader) Weleber (Leader) Branham Daiger Jayasundera Ali Flannery Gamm Michaelides Falsini Hauswirth Khan Sieving Heckenlively Heon Leroy Rao Iannaccone Komáromy Petersen-Jones Reh Molday Pennesi Zacks Naylor WEDNESDAY PM —Session C (Topic 3) Red Group Blue Group Green Group Yellow Group Daiger (Leader) Klassen (Leader) Petersen-Jones (Leader) Komáromy (Leader) Jayasundera (Leader) Sieving (Leader) Heon (Leader) Michaelides (Leader) Audo Banin Auricchio Ali Branham Flannery Leroy Dalkara Falsini Gamm Naylor Heckenlively Hauswirth Khan Pennesi Molday Iannaccone Rao Reh Zacks Weleber Strettoi Thompson THURSDAY AM —Session D (Topics 4 & 5) Red Group Blue Group Green Group Yellow Group Khan (Leader) Banin (Leader) Iannaccone (Leader) Dalkara (Leader) Michaelides (Leader) Leroy (Leader) Komáromy (Leader) Falsini (Leader) Ali Auricchio Audo Gamm Branham Hauswirth Daiger Heckenlively Flannery Jayasundera Klassen Heon Pennesi Petersen-Jones Thompson Molday Reh Rao Weleber Naylor Sieving Zacks Strettoi THURSDAY PM —Session E (Topic 6) Red Group Blue Group Green Group Yellow Group Ali (Leader) Rao (Leader) Hauswirth (Leader) Gamm (Leader) Molday (Leader) Petersen-Jones (Leader) Reh (Leader) Zacks (Leader) Dalkara Branham Banin Audo Falsini Flannery Khan Auricchio Heon Iannaccone Komáromy Daiger Heckenlively Jayasundera Klassen Leroy Weleber Michaelides Pennesi Naylor Sieving Thompson Strettoi PRE-MEETING SURVEY RESPONSES Therapeutic Intervention in Inherited Retinal Degeneration: Strategies, Opportunities, Challenges, Needs 1. the most compelling therapeutic strategies 1. IIdentifying den2fying the most compelling therapeu2c strategies 0.0% 10.0% 20.0% 30.0% 40.0% 50.0% 60.0% Which biological mechanisms and pathways are the most accessible targets? 63.0% Which aspects of pathology can be delayed or reversed? 63.0% Is cell therapy the treatment of choice for rare forms of re2nal dystrophy? What are the most promising strategies for improving delivery of therapeu2c agents? 55.6% 55.6% What are the treatment op2ons for early-‐ vs. advanced-‐stage disease? 48.1% What can be done to prevent or delay cone photoreceptor cell death in rod-‐cone degenera2ons? 40.7% What informa2on can be obtained that could help match the 2ming of treatment to the therapeu2c window of opportunity? 25.9% Which small molecule therapeu2cs are the best candidates for future development? Which currently available tradi2onal forms of therapy should be more widely employed to improve quality of life and/or prolong the therapeu2c window of opportunity? 14.8% 14.8% 70.0% 2. Maximizing the identification of patients likely to benefit from treatment 0.00% 10.00% 20.00% 30.00% 40.00% 50.00% 60.00% 70.00% 80.00% What criteria should be used to select the subset of pa>ents who would benefit most from a given therapy (e.g., muta>on, gene, effects)? What can be done to facilitate the iden>fica>on of eligible pa>ents who meet study criteria for inclusion in specific clinical trials? How can we establish a shared database of pa>ent muta>ons that could be used to enhance recruitment of eligible pa>ents for inclusion in specific clinical trials? What strategies are needed to provide newly approved therapies to large numbers of pa>ents, including those in underdeveloped countries? 73.10% 69.20% 46.20% 46.20% How can more eligible pa>ents gain access to currently available tradi>onal forms of therapy that could reduce or delay their symptoms? Are rigorous studies of disease natural history needed to improve our ability to evaluate the effec>veness of targeted interven>ons? What resources are available for studies of disease natural history? 23.10% 19.20% 11.50% 3. Formalizing outcome measures and facilitating regulatory approval 0.00% 10.00% 20.00% 30.00% 40.00% 50.00% 60.00% 70.00% 80.00% What is the reproducibility and reliability of currently available outcome measures for detecAng therapeuAc benefit? 73.10% Which funcAonal tests are essenAal for opAmizing the evaluaAon of outcomes in clinical trials, while at the same Ame reducing the burden on paAents? 61.50% What addiAonal detecAon technologies or paradigms need to be developed to more quickly and meaningfully measure therapeuAc outcomes? 46.20% How should controls be designed for studies evaluaAng the effects of systemically delivered therapeuAcs, including supplements? 38.50% What technologies and tesAng protocols should be set for child paAents who cannot reliably perform tests that adults can? 34.60% What are the possibiliAes for streamlining regulatory requirements as the field develops? 30.80% 4. Enhancing collaboration and reducing redundancy of effort 0.00% 10.00% 20.00% 30.00% 40.00% 50.00% 60.00% 70.00% 80.00% What kinds of collabora=ve interac=ons would be needed to accelerate progress and reduce costs? 70.40% What are the typical road blocks to collabora=on for which solu=ons can be iden=fied and widely applied? 44.40% What mechanisms can be put in place to enhance resource sharing and coordinate project planning? What criteria and mechanisms should be used to priori=ze the selec=on of target genes? 40.70% 37.00% Should common protocols and FDA paperwork be prepared so that each center does not have to duplicate the same work? 33.30% What are the advantages and disadvantages of centralized produc=on of therapeu=c agents? 29.60% What infrastructure requirements will be needed to support large-‐scale treatment of pa=ents? 18.50% 5. Enhancing funding & fundraising processes to optimize collective efforts 0.00% 10.00% 20.00% 30.00% 40.00% 50.00% 60.00% How can the field a9ract disease-‐neutral donors to fund research development and clinical trials? What can be done to reduce the cost of treaFng paFents? 65.40% 61.50% Is there a financial model that could distribute funding across mulFple centers that have the capacity to contribute to progress on specified projects? 57.70% What financial model could be developed to provide therapeuFc coverage for the largest number of genes and paFents? 42.30% How can we use the Internet to raise funding for joint efforts targeted to specified projects in underdeveloped countries? 19.20% How can we tap into exisFng medical infrastructure and resources for joint efforts targeted to specified projects in underdeveloped countries? 7.70% 70.00% 6. Are there additional questions you would like to include, and/or an issue that you feel is essential for the meeting to address? Reappraising the role of secondary autoimmune complications in hereditary retinal degenerations -- there is ample evidence that this is a true and often very important issue and therapeutic target. Does early intervention slow or prevent disease progression in a genetic photoreceptor disease? What are the disease and/or genetic features that make one therapeutic strategy (e.g. comparison of different gene therapy vectors, cell therapies and therapeutic proteins or small molecules) preferable to others? What is the role and importance of large animal models? Considering the cost, shall resources be invested in the development of new large animal models? Are combination therapies worth pursuing at this time? How can we lobby national governments to help finance gene therapy, stem cell therapy and subretinal implant technology to aid our patients? How do we get inexpensive yet accurate genetic testing on patients? How much will new treatments cost? What cost/benefit can be justified. Discussion about QALYs We should explore the potential of multi-center grants from the NIH, such as the R24 mechanism for developing new collaborative trials. How to optimize the identification, development and use of animal models for preclinical testing. What are the outcomes that we can reasonably expect, and how do we manage expectations/avoid hype? Are there ways to streamline the IND process? Security protected website to help with collaborations, sharing information, and agreed upon data storage. How can we define the optimal window of opportunity for therapeutic intervention? What are the most important environmental factors influencing the disease progression, and how can we modulate them? (For instance: nutritional aspects, exposure to oxidative damage, etc.) 7. What are some key outcomes you would like the meeting to achieve? Ways to accelerate and facilitate clinical application of promising novel therapeutic interventions. Identify priorities for the years to come and define strategies to achieve them. Find ways to enhance collaboration between investigators to maximize access to patients with rare conditions. Publish the outcome of our meeting to impact the course of research in the field for the next decade or so. A realistic perspective of the relative strengths and difficulties in implementing various approaches to the treatment of retinal disease. 1. Identify ways to enhance collaboration and reduce redundancy. 2. Identify ways to develop a patient database. 3. Identify novel outcome measures that reflect clinically relevant improvements. Recommendations about specific role that comparative ophthalmology group can plan in advancing the overall mission of the field. More interactions and collaborations; more streamlined procedures to get potential therapeutic treatments to clinical trials. To help establish how we shall be able to fund further development of gene therapy, stem cell therapy and treatment with subretinal implants to help more patients see. Develop collaborations, enhance mechanisms for resource and information sharing. Develop a more collaborative approach to investigating these diseases. Agreement over stringent therapeutic outcome measures. People’s views on current trials. People’s views on best emerging therapeutic approaches (for given diseases). Scope and feasibility of collaborative ideas. Guidelines for best testing methods in different patient populations (based on age or disease). More effective collaboration between groups. Identify tangible mechanisms of collaboration to move the field forward in a defined manner. Consensus statements on as many of the issues raised in 1-8 as possible. Some new collaborative efforts, and agreement on sharing some resources. Promotion of a collaborative spirit and identification of complementary expertise. 8. Are there any key papers that you think everyone should be aware of? Adamus G, Wang S, Kyger M, Worley A, Lu B, Burrows GG. Systemic immunotherapy delays photoreceptor cell loss and prevents vascular pathology in Royal College of Surgeons rats. Mol Vis. 2012;18:2323-37. http://www.molvis.org/molvis/v18/ a246. Berger W, Kloeckener-Gruissem B, Neidhardt J. The molecular basis of human retinal and vitreoretinal diseases. Prog. Ret. Eye Res. 2010;29:335-375. http://dx.doi.org/10.1016/j.preteyeres.2010.03.004. Birch DG, Locke KG, Wen Y, Locke KI, Hoffman DR, Hood DC. Spectral-Domain Optical Coherence Tomography Measures of Outer Segment Layer Progression in Patients With X-Linked Retinitis Pigmentosa. JAMA Ophthalmol. 2013; http://dx.doi.org/10.1001/jamaophthalmol.2013.4160. Cideciyan AV, Jacobson SG, Beltran WA, Sumaroka A, Swider M, Iwabe S, Roman AJ, Olivares MB, Schwartz SB, Komáromy AM, Hauswirth WW, Aguirre GD. Human retinal gene therapy for Leber congenital amaurosis shows advancing retinal degeneration despite enduring visual improvement. Proc Natl Acad Sci U S A 2013; 110:517-525. http://dx.doi.org/10.1073/pnas.1218933110. Csaky KG, Richman EA and Ferris FL, 3rd. Report from the NEI/FDA Ophthalmic Clinical Trial Design and Endpoints Symposium. Invest Ophthalmol Vis Sci 2008;49:479-89. http://dx.doi.org/10.1167/iovs.07-1132. Eiraku M, Takata N, Ishibashi H, Kawada M, Sakakura E, Okuda S, Sekiguchi K, Adachi T, Sasai Y. Self-organizing optic-cup morphogenesis in three-dimensional culture. Nature. 2011;472:51-6. http://dx.doi.org/10.1038/nature09941. Kyger M, Worley A, Adamus G. Autoimmune responses against photoreceptor antigens during retinal degeneration and their role in macrophage recruitment into retinas of RCS rats. J Neuroimmunol. 2013;254:91-100. http://dx.doi.org/10.1016/j.jneuroim.2012.10.007. Pearson RA, Barber AC, Rizzi M, Hippert C, Xue T, West EL, Duran Y, Smith AJ, Chuang JZ, Azam SA, Luhmann UF, Benucci A, Sung CH, Bainbridge JW, Carandini M, Yau KW, Sowden JC, Ali RR. Restoration of vision after transplantation of photoreceptors. Nature. 2012;485:99-103. http://dx.doi.org/10.1038/nature10997. Ratnam K, Carroll J, Porco TC, Duncan JL, Roorda A. Relationship between Foveal Cone Structure and Clinical Measures of Visual Function in Patients with Inherited Retinal Degenerations. Invest Ophthalmol Vis Sci. 2013;54:5836-47. http://dx.doi.org/10.1167/iovs.13-12557. Schachar IH, Zahid S, Comer GM, Stem M, Schachar AG, Saxe SJ, Gardner TW, Elner VM, Jayasundera T. Quantification of Fundus Autofluorescence to Detect Disease Severity in Nonexudative Age-Related Macular Degeneration. JAMA Ophthalmol. 2013;131:1009-15. http://dx.doi.org/10.1001/jamaophthalmol.2013.4014. Tremblay JP, Xiao X, Aartsma-Rus A, Barbas C, Blau HM, Bogdanove AJ, Boycott K, Braun S, Breakefield XO, Bueren JA, Buschmann M, Byrne BJ, Calos M, Cathomen T, Chamberlain J, Chuah M, Cornetta K, Davies KE, Dickson JG, Duchateau P, Flotte TR, Gaudet D, Gersbach CA, Gilbert R, Glorioso J, Herzog RW, High KA, Huang W, Huard J, Joung JK, Liu D, Liu D, Lochmüller H, Lustig L, Martens J, Massie B, Mavilio F, Mendell JR, Nathwani A, Ponder K, Porteus M, Puymirat J, Samulski J, Takeda S, Thrasher A, VandenDriessche T, Wei Y, Wilson JM, Wilton SD, Wolfe JH, Gao G. Translating the genomics revolution: the need for an international gene therapy consortium for monogenic diseases. Mol Ther. 2013;21:266-8. http://dx.doi.org/10.1038/mt.2013.4. Zahid S, Khan N, Branham K, Othman M, Karoukis AJ, Sharma N, Moncrief A, Mahmood MN, Sieving PA, Swaroop A, Heckenlively JR, Jayasundera T. Phenotypic Conservation in Patients With X-Linked Retinitis Pigmentosa Caused by RPGR Mutations. JAMA Ophthalmol. 2013;131:1016-25. http://dx.doi.org/10.1001/jamaophthalmol.2013.120. PARTICIPANTS Robin R. Ali, Ph.D. Div of Molecular Therapy UCL Institute of Ophthalmology 11-43 Bath Street London, England EC1V 9EL United Kingdom 442076086817 [email protected] Deniz Dalkara, Ph.D. Therapeutics Institut de la Vision/INSERM 17 rue Moreau Paris, 75012 France 5106420209 [email protected] Isabelle S. Audo, M.D., Ph.D. Department of Genetics Institut de la Vision /INSERM /UPMC/CNRS/CHNO 17 Rue Moreau Paris, 75012 France 33153462540 [email protected] Benedetto Falsini, M.D. Ophthalmology Catholic University Lgo Vito 1 Rome, 00168 Italy 390630154929 [email protected] Alberto Auricchio, M.D. Tigem Fondazione Telethon Via Carlo Spinola 16 Rome, 00154 Italy 39-081-6132228 [email protected] John Gerard Flannery, Ph.D. Helen Wills Neuroscience Institute University of California-Berkeley 132 Barker Hall MC3190 Berkeley, CA 94720-3190 United States (510)642-0178 [email protected] Eyal Banin, M.D., Ph.D. Ophthalmology Hadassah-Hebrew University Med Ctr Ein-Karem PO Box 12000 Jerusalem, 91120 Israel (972)2-6776585 [email protected] David M. Gamm, M.D., Ph.D. Ophthalmology and Visual Sciences University of Wisconsin-Madison 1500 Highland Ave, Rm 621 Madison, WI 53705-2280 United States (608)261-1516 [email protected] Kari E. Branham, M.S. Ophthalmology and Visual Sciences University of Michigan Medical School Kellogg Eye Center 1000 Wall St Ann Arbor, MI 48105 United States (734)936-8638 [email protected] William W. Hauswirth, Ph.D. Department of Ophthalmology University of Florida College of Medicine Box 100284 JHMHC Gainesville, FL 32610-0284 United States (352)392-0679 [email protected] Stephen P. Daiger, Ph.D. Human Genetics Center, School Pub Hlth University of Texas Hlth Sci Ctr Houston 1200 Herman Pressler Drive Houston, TX 77030 United States (713)500-9829 [email protected] John R. Heckenlively, M.D. Ophthalmology and Visual Sciences University of Michigan Medical School Kellogg Eye Center 1000 Wall St, Rm 326 Ann Arbor, MI 48105 United States (734)763-2280 [email protected] Elise Heon, M.D. Ophthalmology and Vision Sciences Hospital for Sick Children 555 University Ave Toronto, ON M5G 1X8 Canada (416)8138606 [email protected] Alessandro Iannaccone, M.D., M.S. Ophthal/Hamilton Eye Institute University of Tennessee Health Sci Ctr 930 Madison Ave Ste 731 Memphis, TN 38163 United States (901)448-7831 [email protected] Thiran Jayasundera, M.D., FRCSC FRANZCO Ophthalmology and Visual Sciences University of Michigan Medical School Kellogg Eye Center 1000 Wall Street Ann Arbor, MI 48105 United States (734)7302453 [email protected] András Komáromy, DrMetVet, Ph.D. Small Animal Clinical Studies Michigan State University College of Veterinary Medicine D-208 Veterinary Med Center East Lansing, MI 48824-1314 United States (517)353-5420 [email protected] Bart Peter Leroy, M.D., Ph.D. Dept Ophthalmology & Ctr Med Genetics Ghent University Hospital & University De Pintelaan 185 Ghent, 9000 Belgium 3293322311 [email protected] Paul R. Lichter, M.D. Ophthalmology and Visual Sciences University of Michigan Medical School Kellogg Eye Center 1000 Wall St Ann Arbor, MI 48105 United States (734)764-6468 [email protected] Naheed W. Khan, Ph.D. Ophthalmology and Visual Sciences University of Michigan Medical School Kellogg Eye Center 1000 Wall Street, Rm 346 Ann Arbor, MI 48105 United States (734)763-0691 [email protected] Michel Michaelides, M.D., FRCOphth Medical Retina Moorfields Eye Hospital 162 City Road London, England EC1V 2PD United Kingdom 44 207 608 6885 (6866) [email protected] Henry J. Klassen, M.D., Ph.D. Ophthalmology University of California-Irvine 843 Hlth Sci Rd Hewitt Hall, Rm 2014 Irvine, CA 92697-4390 United States (949)824-7750 [email protected] Robert S. Molday, Ph.D. Biochemistry/Molecular Biology University of British Columbia 2350 Health Sciences Mall Vancouver, BC V6T 1Z3 Canada (604)822-6173 [email protected] Stuart Naylor, Ph.D. London, England United Kingdom 44(0)7501 011680 [email protected] Mark E. Pennesi, M.D., Ph.D. Ophthalmology Casey Eye Institute Oregon Health Science Center 3375 SW Terwilliger Blvd Portland, OR 97239 United States (415)676-1721 [email protected] Debra A. Thompson, Ph.D. Ophthalmology and Visual Sciences University of Michigan Medical School Kellogg Eye Center 1000 Wall St Ann Arbor, MI 48105-0714 United States (734)936-9504 [email protected] Simon M. Petersen-Jones, DVetMed, Ph.D. Small Animal Clinical Sciences Michigan State University College of Veterinary Medicine 736 Wilson Rd, D-208 East Lansing, MI 48824-1314 United States (517)353-3278 [email protected] Richard G. Weleber, M.D. Ophth & Molecular Med Genetics Casey Eye Institute Oregon Health Science Center 3375 SW Terwilliger Blvd Portland, OR 97221 United States (503)494-3016 [email protected] Rajesh Rao, M.D. Ophthalmology and Visual Sciences University of Michigan Medical School Kellogg Eye Center 1000 Wall St Ann Arbor, MI 48105-0714 United States (734)763-8122 [email protected] David N. Zacks, M.D., Ph.D. Ophthalmology and Visual Sciences University of Michigan Medical School Kellogg Eye Center 1000 Wall St Ann Arbor, MI 48105 United States (734)763-7711 [email protected] Thomas A. Reh, Ph.D. Department of Biological Structure University of Washington I-516 Hlth Sci Bldg; 1959 NE Pacific St Seattle, WA 98195 United States (206)543-5069 [email protected] Paul A. Sieving, M.D., Ph.D. National Eye Institute NIH 31 Center Dr, MS 2510 Bethesda, MD 20892-0001 United States (301)496-2234 [email protected] Enrica Strettoi, Ph.D. CNR Neuroscience Institute Area della Ricerca CNR, Via G Moruzzi 1 Pisa, 56100 Italy 390503153213 MEETING STAFF MargaretAnn Cross Scribe/Writer University of Michigan Health System Ann Arbor, MI 48105 United States (734)546-3364 [email protected] Sofia Piperno Rome, Italy Cameron Strong Ann Arbor, MI USA ACKNOWLEDGMENTS Support for the Monaciano Symposium was provided by: Celeste and Bruno Piperno University of Michigan Department of Ophthalmology and Visual Sciences University of Tennessee Health Science Center, Hamilton Eye Institute University of Michigan Office of the Vice President for Research Lions International of Tuscany Paul P. Lee, M.D., J.D. A special thanks to Dierdre Jeske for providing outstanding administrative assistance, to Lisa Burkhart for developing the meeting website and survey, and to Nancy Mariani and Pina Vuocolo Giuseppina for handling logistics at the estate.