CAROT

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Clinical Core

Welcoming Dr. Erin O'Neil to the Clinical Core!

A critical mission of our center is to bring novel therapies to the affected patients. We structured our center to facilitate translational research into clinical trials with speed and caution. We have collaborated on and assisted with developing multiple ocular gene therapy INDs, documents required by the FDA before initiating a clinical trial. In particular, our collaborative work on Leber’s Congenital Amaurosis type 2 (LCA caused by RPE65 mutations) with the Children’s Hospital of Philadelphia (CHOP) has resulted in the only Phase 3 gene therapy study currently underway in the world. 

 

 

 

Albert M. Maguire, M.D.

Albert M. Maguire, M.D.
Co-Director, CAROT Professor, Ophthalmology, University of Pennsylvania Perelman School of Medicine Attending, Retina & Vitreo-retinal Surgery, Scheie Eye Institute and Perelman Center for Advanced Medicine Attending, Pediatric Retina & VItreo-retinal Surgery, Department of Pediatric Ophthalmology, The Children’s Hospital of Philadelphia
 amaguire@pennmedicine.upenn.edu

Dr. Maguire has had a long-standing interest in retinal gene transfer/gene therapy and developed surgical approaches with which to deliver genes in proof-of-concept studies involving gene therapy. Dr. Maguire has participated in numerous clinical trials for retinal diseases. His research resulted in approval of the first gene therapy product for a genetic disease approved by the US FDA and the first gene therapy for retinal disease worldwide.

 


Tomas S. Aleman, M.D.

Tomas S. Aleman, M.D.
Associate Clinical Professor; Ophthalmologist & Fellowship-trained Medical Retina & Retinal Degeneration Specialist; Director of the Retinal Degeneration Service at the Perelman Center of Advanced Medicine and CAROT
 aleman@pennmedicine.upenn.edu

Dr. Aleman is an Associate Clinical Professor, an ophthalmologist and fellowship-trained medical retina and retinal degeneration specialist and is Director of the Retinal Degeneration Service at the Perelman Center of Advanced Medicine and CAROT (UPenn). He is also an attending physician at The Children’s Hospital of Philadelphia. Dr. Aleman obtained his initial medical training in Cuba and then carried out additional clinical and research training in the US. He has had more than 3 decades of experience performing careful genotype-phenotype and clinical-histopathologic correlation studies, conducting outcome measure research, and developing new in-vivo imaging technologies and functional assessments. Dr. Aleman is the PI of several ongoing gene therapy clinical trials and now evaluates and manages patients who receive gene therapy for RPE65 mutations (i.e. “Luxturna”) by prescription. Dr. Aleman has published more than 100 peer-reviewed papers on the cellular origin of retinal signals, the structural and functional impact of primary photoreceptor degenerations on the visual pathway, electrophysiology and imaging signals of retinal remodeling, retinal function and structural consequences of systemic diseases, exploration of treatment potential of gene therapy, small molecules and retinal transplantation in animal models, development of outcome measures for a diverse set of retinal diseases, preclinical studies leading to human clinical trials, and results of human clinical trials.


Bart Peter Leroy, M.D., Ph.D.

Bart Peter Leroy, M.D., Ph.D.
Attending Physician at the Division of Ophthalmology and the Center for Cellular & Molecular Therapeutics at Children's Hospital of Philadelphia
 leroyb@email.chop.edu, bart.leroy@ugent.be

Dr. Leroy is an ophthalmologist and ophthalmic geneticist specializing in inherited eye disorders and systemic conditions affecting the eye. He is Chairman and Head of the Department of Ophthalmology, and Staff Member at the Center for Medical Genetics at the Ghent University Hospital and Ghent University (Belgium), where he has been working since 2001. He was appointed as Associate Professor of Ophthalmic Genetics and Visual Electrophysiology at the Ghent University in October 2009. Since 2013, Bart also has a part-time position (20%) as an attending physician at the Division of Ophthalmology and the Center for Cellular & Molecular Therapeutics at Children's Hospital of Philadelphia, Philadelphia, PA, USA.


Denise Pearson, C.O.M.T.

Denise Pearson, C.O.M.T.
Certified Ophthalmic Medical Technologist
 denise.pearson@uphs.upenn.edu

Denise Pearson is a Certified Ophthalmic Medical Technologist and has worked at Scheie Eye Institute, University of Pennsylvania since 1995. There, she has served as Clinical Research Coordinator and Ophthalmic Technologist and while in these positions, also carried out a large set of procedures including serving as a Refractive Surgery Technician, Contact Lens Examiner, and Retinal Photographer. She joined the UPenn Reading Center to gain experience in working on clinical trials and was then recruited to CAROT in 2014 to serve as Clinical Coordinator for gene therapy clinical trials. She is the Clinical Coordinator for a Phase I/II gene therapy clinical trial for choroideremia and will soon be serving this position on several additional gene therapy clinical trials. Simultaneous with her clinical work, Ms. Pearson is completing requirements to receive a Master of Regulatory Affairs degree from UPenn.


Katherine Uyhazi, M.D., Ph.D.

Katherine Uyhazi, M.D., Ph.D.
Attending, Medical Retina and Retinal Degeneration, University of Pennsylvania Perelman School of Medicine
 Katherine.Uyhazi@UPHS.upenn.edu

Dr. Uyhazi received her B.S. from the College of New Jersey, summa cum laude, and her MD/PhD from Yale School of Medicine with her PhD in Cell Biology. She completed her residency in ophthalmology at Scheie Eye Institute, University of Pennsylvania where she will be completing a fellowship in Medical Retina and Retinal Degeneration in July 2019.  In addition to her clinical work, Dr. Uyhazi is carrying out independent basic and translational research at CAROT.


Dr. Erin O'Neil

Dr. Erin O'Neil
 Erin.O'Neil@pennmedicine.upenn.edu

Dr. Erin O'Neil graduated from Pomona College with a B.A. in International Relations & Economics. She worked in the finance and technology industries for several years prior to finding her way to medicine. She graduated from the Perelman School of Medicine at the University of Pennsylvania in 2016 and completed her Ophthalmology Residency at the Scheie Eye Institute, University of Pennsylvania where she will complete a fellowship in Medical Retina, Retinal Degeneration & Ophthalmic Genetics in 2022. She is also currently a candidate for the Master's of Science in Translational Research from the University of Pennsylvania.


Keli O'Connor COT, ABOC

Keli O'Connor COT, ABOC
Board-Certified Optician
 Keli.O'connor@pennmedicine.upenn.edu

Keli B. O’Connor, COT, ABOC has been working in ophthalmology since 2008. A board-certified optician, she regularly teaches lensometry and optics to ophthalmology residents and fellows, trains new technicians for the Scheie Eye Institute, and has instructed at national conferences for eye care personnel since 2017. Keli lives in Delaware with her two sons and enjoys writing, hiking, and traveling.



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