Silencing of PRLΔE1 expression: a gene-agnostic treatment for retinal degenerations
Viral vector-mediated gene silencing of a novel short retinal isoform of Prolactin promotes photoreceptor survival. Problem: Age-related macular degeneration (AMD) and inherited retinal degeneration (IRDs) have extreme mutational heterogeneity, and many IRDs are orphan diseases. Developing individual gene therapies for each causative gene is impractical...A Method for Promoting Structural and Functional Rescue of Photoreceptors in the Eye to Treat Advanced Retinal Degeneration.
Discovery and use of retina-specific promoters to rescue photoreceptor function specifically at clinically relevant, advanced stages of disease. Problem: Despite the fact that retinal gene therapy studies have shown promising results in animal models, treatment of advanced retinal degeneration with currently available gene therapies have not been successful....Improving Diagnosis and Treatment of Sarcoidosis
Using a blood or tissue biomarker to improve diagnosis and identification of a new targeted treatment. Problem: Sarcoidosis is an inflammatory disease that can affect any organ, predominantly the lung, skin, and the lymphatic system. Symptoms include difficulty breathing, extreme fatigue, skin inflammation, and joint pain. Sarcoidosis compromises patients’...Researcher(s):
Thomas Hin-Chai Leung, Misha Rosenbach, Satish SatiKeyword(s):
Dermatology, Immunology, Inflammation, Ophthalmology, PulmonaryAn Improved and Targeted Approach to Genetic Screening for Primary Open-Angle Glaucoma in African Ancestry Population
A method for more targeted pre-screening for Primary Open-Angle Glaucoma (POAG) in individuals of African ancestry. Problem: POAG, the leading cause of irreversible blindness worldwide, currently affects 44 million individuals globally and is expected to impact 80 million people by the year 2040. Of particular concern is its disproportionate burden...Researcher(s):
Yuki Bradford, Joan O'Brien, Marylyn Ritchie, Rebecca Salowe, Shefali VermaKeyword(s):
OphthalmologyA Precise, Analytical Model to Measure Visual Function and Monitor Retinal Disease
A model to estimate the change in photoreceptor outer segment length that occurs upon light stimulation – a biomarker of cone function in the retina. Problem: Vision relies on the isomerization of photopigments within photoreceptors (cones and rods) by light. Traditionally, researchers and clinicians have relied on functional assessments of photoreceptor...Researcher(s):
David Brainard, Jessica Morgan, Raymond WarnerKeyword(s):
Neurology, OphthalmologymRNA Therapeutics for Corneal Endothelium Regeneration
A novel mRNA-based therapy that induces corneal endothelium regeneration to restore vision loss Problem: Corneal endothelial cells are critical for maintaining a clear cornea, mainly by pumping fluid out of the tissue. Decrease in number of these cells results in the clouding of the cornea that leads to vision loss. The standard of care for Fuchs Endothelial...Researcher(s):
Panteleimon RompolasKeyword(s):
Gene Therapy, Healthcare IT, OphthalmologyTreatment for the Prevention of Permanent Vision Loss in Glaucoma Patients
Problem: Glaucoma is the number one cause of irreversible blindness worldwide. High pressure in the eye can lead to optic nerve damage, which can progress to permanent vision loss. For this reason, reduction of intraocular pressure is the main therapeutic mechanism available. Unfortunately, glaucoma can continue to progress even in patients with normalized...Researcher(s):
Qi Cui, Joshua Dunaief, Jacob SterlingKeyword(s):
Ophthalmology, Small MoleculeVersatile virtual reality system for functional vision evaluation
Virtual reality-based system and software for quick, accurate, and reproducible evaluation of functional vision capacity following clinical treatment Problem: In 2013, the total economic burden of eye disorders and vision loss in the United States was $139 billion with projections to increase by ~20% over the following 10 years. Developing treatments...Differentiation of induced pluripotent stem cells into retinal progenitor cells and mature, functional human retinal ganglion cells
Simplified and reproducible two-step differentiation method optimized for efficient differentiation of human-induced pluripotent stem cells into retinal progenitor cells and functional retinal ganglion cells Problem: More than three million Americans live with glaucoma – a disease that can cause blindness resulting from optic nerve...Researcher(s):
Venkata Chavali, Jason Mills, Joan O'BrienKeyword(s):
Cell Therapy, Ophthalmology, Regenerative MedicineAnti-Pulfrich monovision ophthalmic corrections that reduce depth misperception
Technology Overview: Presbyopia is the age-related loss of your eyes’ ability to focus on objects that are up close. A normal part of the aging process, the effects of presbyopia typically begin in humans sometime around 45 years of age. Researchers have estimated that there are close to 2 billion worldwide that have presbyopia. Monovision...Researcher(s):
Johannes Burge, Carlos Dorronsoro, Victor RodriguezKeyword(s):
OphthalmologyHigh Resolution Functional Imaging of Individual Cone Photoreceptors in the Living Human Eye
A non-invasive in vivo imaging technique using enhanced ophthalmoscopy to assess the intrinsic function of individual photoreceptor cells while maintaining high spatial resolution. This technique could be applied for both diagnostic purposes as well as the precise measurement of treatment response to both existing and experimental therapies for retinal...Researcher(s):
David Brainard, Robert Cooper, Jessica Morgan, William TutenKeyword(s):
OphthalmologyAn Iron Chelator for Protection Against Retinal Degeneration
Problem: Degenerative diseases of the retina such as age-related macular degeneration (AMD) are the leading cause of vision loss and blindness. Approximately 120 million people worldwide suffer from AMD. There is a need to further understand the pathogenesis of retinal degeneration in order to develop new therapeutics to prevent and treat these diseases....Researcher(s):
Joshua DunaiefKeyword(s):
OphthalmologyGene Therapy for Macular Degeneration
Gene therapy targeting disposal of toxic intracellular lipid debris and lipofuscin in wet age related macular degeneration. Problem: Macular degeneration is a genetic eye disorder that affects the retina resulting in progressive vision loss. Macular degeneration has two main forms, Stargardt’s macular degeneration (STGD) which affects juveniles,...Researcher(s):
Kathleen Boesze-BattagliaKeyword(s):
Gene Therapy, OphthalmologySurgical device for conjunctival closure
Surgical device for wound closure with minimum invasion into the tissue Problem: Surgical procedures in the eye require wound closure techniques that are minimally invasive. Conventional suturing techniques and the use of metal staples are not well suited for use in these regions. Solution: Dr. Tushar Ranchod, who was formerly a resident...Researcher(s):
Tushar RanchodKeyword(s):
Ophthalmology
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