

Elisabetta Morini
Massachusetts General Hospital, Center for Genomic Medicine; Harvard Medical School, Department of Neurology
Elisabetta Morini is an Instructor in Neurology at Massachusetts General Hospital (MGH) and Harvard Medical School (HMS). Her research focuses on understanding the regulation of mRNA splicing and how its disruption leads to human disease, with the goal of developing new therapeutics. After receiving her PhD in Molecular and Regenerative Medicine from the University of Modena, she joined the laboratory of Dr. Susan Slaugenhaupt in the Center for Genomic Medicine (CGM) at MGH, where she worked on the generation of a new mouse model for Familial Dysautonomia. This mouse was truly a breakthrough in the field and allowed her to test novel splicing modulator therapies for FD. Since becoming a junior faculty at MGH and HMS, she has been working on developing treatments targeting splicing alterations across a variety of neurologic disorders.

Anil Chekuri
Massachusetts Eye and Ear Infirmary
Anil Chekuri is an Instructor in Ophthalmology at Massachusetts Eye and Ear Infirmary (MEEI) and Harvard Medical School (HMS). His research interests include understanding the molecular mechanisms of Inherited Retinal Degenerations (IRDs) and developing novel therapeutic approaches using AAV delivery. Anil Received his PhD in Biotechnology from the University of Hyderabad, India. In 2019, he joined the laboratories of Dr. Susan Slaugenhaupt and Dr. Luk Vandenberghe in the departments of Neurology and Ophthalmology at Harvard Medical School (HMS). Since then, his work has been focused on understanding the retinal pathology characteristic of familial dysautonomia (FD) and testing the in vivo efficacy of small molecules and U1small nuclear RNA to rescue retinal degeneration. He has also been working on developing novel treatment modalities for other IRDs such as Usher’s syndrome, Retinitis pigmentosa 28, and Bietti’s crystalline dystrophy.
Over the past decade, there has been significant progress in developing therapeutic options for several ocular diseases such as diabetic retinopathy, inherited retinal dystrophies, age-related macular degeneration, and retinal vascular disorders. The treatment options for retinal diseases have been markedly expanded following the FDA approval of Luxturna, an AAV-based drug for Leber's Congenital Amaurosis (LCA). A collection of methods, including preparation of the retina and Retinal Pigment Epithelium (RPE) and the delivery of drugs to the retina, will be very beneficial for the ongoing pre-clinical research aimed at treating eye disorders. This collection wants to include methods for isolation and staining of retinal and RPE flat mounts, retinal cross-sections, single-cell dissociation, and FACS sorting of specific retinal cell populations for biochemical and molecular analysis. We will also extensively describe existing and novel developments in ocular drug delivery to the retina, including but not limited to: subretinal, intravitreal, suprachoroidal, subconjunctival, and intrastromal injection.
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2025
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Maria Clara da Silva de Jesus*1, Douglas Penaforte Cruz*2, Greysa Saraí Barrios León1, Millena Costa Crisóstomo2, Camila Feitosa Magalhães2,3, Aline Estacio Ribeiro de Mattos2, Lucianne Fragel-Madeira1,2
1Neural Development and Regeneration Laboratory, Department of Neurobiology, Science and Biotechnology Graduate Program, Institute of Biology, Fluminense Federal University, 2Neural Development and Regeneration Laboratory, Department of Neurobiology, Neuroscience Graduate Program, Institute of Biology, Fluminense Federal University, 3Instituto Estadual de Hematologia Arthur de Siqueira Cavalcanti (HEMORIO)
Optimizing retinal research: a protocol for drug delivery and histological analysis
Greysa Leon1,
Aline Mattos1,
Maria Clara Jesus1,
Millena Crisostomo1,
Douglas Penaforte-Cruz1,
Rafael Azevedo-Repossi1,
Lucianne Fragel-Madeira*1
1Fluminense Federal University
Investigation of mouse retinal pigment epithelium structure and function by high-resolution microscopy and advanced biochemical techniques
Aparna Lakkaraju*1,
Colin Germer1,
Renuka Chaphalkar1,
Li Xuan Tan1,
Nilsa La Cunza1
1UCSF