gene therapy for eye disease

Lukason M., DuFresne E., Rubin H., Pechan P., Li Q., Kim I., Kiss S., Flaxel C., Collins M., Miller J. Inhibition of choroidal neovascularization in a nonhuman primate model by intravitreal administration of an AAV2 vector expressing a novel anti-VEGF molecule. Anders C., Niewoehner O., Duerst A., Jinek M. Structural basis of PAM-dependent target DNA recognition by the Cas9 endonuclease. Samuel G. Jacobson and Artur V. Cideciyan from the University of Pennsylvania, and Dr. William W. Hauswirth from the University of Florida, Gainesville. The complement-inhibitory activity of CD59 resides in its capacity to block incorporation of C9 into membrane C5b-9. The researchers determined that the RPE65 gene therapy boosted the photoreceptor cell recharge cycle, but didnt delay cell death. Careers. WebThe team found that gene therapy improved patients eyesight and the sensitivity of the retina within a month of treatment. Chen J.S., Dagdas Y.S., Kleinstiver B.P., Welch M.M., Sousa A.A., Harrington L.B., Sternberg S.H., Joung J.K., Yildiz A., Doudna J.A. -, Moiseyev G, Chen Y, Takahashi Y, et al. Off-label drug use: the bevacizumab story. Diabetic macular edema (DME) is a frequent cause of vision loss, resulting from the breakdown of the outer blood-retinal barrier due to damaged endothelial tight junctions that allow salts, proteins, and water to accumulate within the retina.20,21 DME can occur at any stage of DR. Proliferative DR (PDR), or advanced DR, is characterized by neovascularization with or without pre-retinal or vitreal hemorrhages. Tolentino M.J., Brucker A.J., Fosnot J., Ying G.S., Wu I.H., Malik G., Wan S., Reich S.J. Dunn E.N., Hariprasad S.M., Sheth V.S. AAVs are currently the most commonly used vector for retinal gene transfer in both preclinical studies and clinical trials.114 Although AAV has an excellent safety profile, mild and temporary inflammatory responses have been reported with higher ocular AAV vector doses.115, 116, 117 Recently, Timmers etal.117 studied the contribution of the AAV vector genome and capsid in triggering ocular inflammatory responses. In CRISPR-edited OIR mice retina, there was approximately a 30% reduction in VEGFR2 production with significant inhibition of retinal neovascularization. Gene silencing with RNA interference to target ocular angiogenesis has been investigated in clinical trials. High-frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells. Effective delivery of large genes to the retina by dual AAV vectors. It is predicted that the number of individuals affected globally by AMD will reach 288 million by 2040.7 AMD can be classified as non-neovascular (dry form AMD [dAMD]) or neovascular forms (nAMD). Article plus Supplemental Information, GUID:F862C912-053E-413E-8734-8944B58F84BA, neovascularization, eye, gene therapy, VEGF, age-related macular degeneration, diabetic retinopathy, {"type":"clinical-trial","attrs":{"text":"NCT03577899","term_id":"NCT03577899"}}, {"type":"clinical-trial","attrs":{"text":"NCT03630952","term_id":"NCT03630952"}}, {"type":"clinical-trial","attrs":{"text":"NCT02699450","term_id":"NCT02699450"}}, {"type":"clinical-trial","attrs":{"text":"NCT03038880","term_id":"NCT03038880"}}, {"type":"clinical-trial","attrs":{"text":"NCT01944839","term_id":"NCT01944839"}}, {"type":"clinical-trial","attrs":{"text":"NCT01940900","term_id":"NCT01940900"}}, {"type":"clinical-trial","attrs":{"text":"NCT03999801","term_id":"NCT03999801"}}, {"type":"clinical-trial","attrs":{"text":"NCT03585556","term_id":"NCT03585556"}}, {"type":"clinical-trial","attrs":{"text":"NCT03748784","term_id":"NCT03748784"}}, {"type":"clinical-trial","attrs":{"text":"NCT03066258","term_id":"NCT03066258"}}, {"type":"clinical-trial","attrs":{"text":"NCT01678872","term_id":"NCT01678872"}}, {"type":"clinical-trial","attrs":{"text":"NCT01494805","term_id":"NCT01494805"}}, {"type":"clinical-trial","attrs":{"text":"NCT01301443","term_id":"NCT01301443"}}, {"type":"clinical-trial","attrs":{"text":"NCT01024998","term_id":"NCT01024998"}}, {"type":"clinical-trial","attrs":{"text":"NCT00109499","term_id":"NCT00109499"}}, {"type":"clinical-trial","attrs":{"text":"NCT01445899","term_id":"NCT01445899"}}, {"type":"clinical-trial","attrs":{"text":"NCT00713518","term_id":"NCT00713518"}}, {"type":"clinical-trial","attrs":{"text":"NCT00557791","term_id":"NCT00557791"}}, {"type":"clinical-trial","attrs":{"text":"NCT00701181","term_id":"NCT00701181"}}, {"type":"clinical-trial","attrs":{"text":"NCT00499590","term_id":"NCT00499590"}}, {"type":"clinical-trial","attrs":{"text":"NCT00725686","term_id":"NCT00725686"}}, {"type":"clinical-trial","attrs":{"text":"NCT00395057","term_id":"NCT00395057"}}, {"type":"clinical-trial","attrs":{"text":"NCT00306904","term_id":"NCT00306904"}}, {"type":"clinical-trial","attrs":{"text":"NCT00259753","term_id":"NCT00259753"}}, {"type":"clinical-trial","attrs":{"text":"NCT00363714","term_id":"NCT00363714"}}, {"type":"clinical-trial","attrs":{"text":"NCT00722384","term_id":"NCT00722384"}}, {"type":"clinical-trial","attrs":{"text":"NCT03144999","term_id":"NCT03144999"}}, {"type":"clinical-trial","attrs":{"text":"NCT03872479","term_id":"NCT03872479"}}. Identification of a natural soluble form of the vascular endothelial growth factor receptor, FLT-1, and its heterodimerization with KDR. Emerging treatments for wet age-related macular degeneration. Innovative therapies for neovascular age-related macular degeneration. Disease . Hit and go CAS9 delivered through a lentiviral based self-limiting circuit. Yang S., Zhao J., Sun X. Gene therapy requires the introduction of exogenous genetic material, such as DNA, RNA, small interfering (siRNA), microRNA (miRNA), and antisense oligonucleotides (synthesized nucleic acid sequence complementary to mRNA), into cells via viral or non-viral vectors to regulate, replace, or modulate specific gene functions.113 Gene therapy approaches are generally categorized into gene augmentation, gene-specific targeting, or genome editing.104 Gene augmentation therapy refers to the introduction of correct copies of genes into the host genome to compensate for the faulty gene, as is the case with Luxturna. Yau J.W., Rogers S.L., Kawasaki R., Lamoureux E.L., Kowalski J.W., Bek T., Chen S.J., Dekker J.M., Fletcher A., Grauslund J., Meta-Analysis for Eye Disease (META-EYE) Study Group Global prevalence and major risk factors of diabetic retinopathy. This was followed by a slow expansion of the area of improvement for up to 3 years after treatment. Subconjunctival injection of recombinant AAV-angiostatin ameliorates alkali burn induced corneal angiogenesis. Would you like email updates of new search results? Rubio R.G., Adamis A.P. Genetic deletion of S-opsin prevents rapid cone degeneration in a mouse model of Leber congenital amaurosis. Eye and transmitted securely. Sharma A., Kumar N., Kuppermann B.D., Bandello F., Loewenstein A. Faricimab: expanding horizon beyond VEGF. Expressions of angiopoietins and Tie2 in human choroidal neovascular membranes. National Library of Medicine Before WebPMCID: PMC8531184 DOI: 10.2174/1566523221666210423084233 Abstract This review provides comprehensive information about the advances in gene therapy in the anterior segment of the eye, including cornea, conjunctiva, lacrimal gland, and trabecular meshwork. Similarly, CRISPR-SpCas9-mediated disruption of VEGFR2 reduced CNV size compared with vehicle control laser-induced CNV mice. Non-genetic risk factors for AMDinclude smoking and deficiency in dietary intake of antioxidants such as carotenoids and zinc.11,12 During the last two decades,therapeutic intervention for nAMD has shifted from laser photocoagulation to block leaky vessels to pharmacotherapeutic intervention, particularly anti-vascular endothelial growth factor (VEGF) therapy.13 Anti-VEGF therapy has also become a mainstay for the treatment of neovascularization and vascular hyperpermeability in DR. DR is a leading cause of legal blindness in those of working age (2065 years).14 According to the International Diabetes Federation (IDF), in 2017 there were an estimated 451 million people with diabetes mellitus (DM), a number projected to increase to 693 million by 2045.15 The prevalence of any form of retinal pathology (retinopathy) in DM patients is approximately 35%, and the number of people with DR is estimated to increase to 191 million in 2030.16,17 Around 7%10% patients with DM will develop vision-threatening (proliferative) retinopathy, an advanced stage of DR.17,18 Diabetes-associated hyperglycemia damages the retinal vasculature vascular, including endothelial cells, basement membrane, and supporting pericytes.19 Non-proliferative DR (NPDR) is the initial stage of DR characterized by mild changes such as microaneurysms, microhemorrhages, hard exudates, and cotton wool spots, which can be detected clinically using ophthalmoscopy or fundus imaging. Campochiaro P.A., Lauer A.K., Sohn E.H., Mir T.A., Naylor S., Anderton M.C., Kelleher M., Harrop R., Ellis S., Mitrophanous K.A. A helper-dependent adenovirus vector system: removal of helper virus by Cre-mediated excision of the viral packaging signal. The VEGF-VEGFR2 system engages activation of PI3K, p38MAPK, FAK, and PLC. Brar V.S., Sharma R.K., Murthy R.K., Chalam K.V. Gene therapy is one of the ways Janssen aims to significantly improve health outcomes for Forty-two patients were enrolled and divided into five groups with different viral doses (3E9, 1E10, 6E10, 1.6E11, and 2.5E11 gene copies [GC]). Studies using a range of preclinical ocular neovascularization models have demonstrated that simultaneous inhibition of VEGF and PDGF, especially PDGF-B, results in an enhanced anti-angiogenic effect.89,90 However, clinical studies report that combining pegpleranib (Fovista) or rinucumab (a neutralizing monoclonal antibody to human PDGF-B) with anti-VEGF therapy showed no further benefits in treating nAMD.91 Results from two phase III clinical trials, OPH1002 (ClinicalTrials.gov: {"type":"clinical-trial","attrs":{"text":"NCT01944839","term_id":"NCT01944839"}}NCT01944839) and OPH1003 (ClinicalTrials.gov: {"type":"clinical-trial","attrs":{"text":"NCT01940900","term_id":"NCT01940900"}}NCT01940900), revealed that combined pegpleranib and ranibizumab therapy failed to improve vision at 12months when compared with ranibizumab monotherapy.92,93 Further studies and subgroup analyses of these data may reveal specific groups of retinal or CNV patients who may benefit from anti-PDGF and anti-VEGF combined therapy.50,91, Although intravitreal injections of anti-VEGF agents are effective and safe,25 they do not cure the fundamental problem, and most patients will require repeated intravitreal injections to sustain therapeutic efficacy due to the relatively short half-life of these agents.94,95 Following intravitreal injections (0.5mg), the half-life of ranibizumab is approximately 9 and 7days in the vitreous humor and aqueous humor, respectively.41,45 Clinically, a meaningful improvement in visual acuity was only seen in patients receiving monthly ranibizumab injections, rather than those who had received injections every 3months.96,97 The vitreous half-life of bevacizumab is between 4.9 and 6.7days after the intravitreal administration (1.25mg), as reported by two studies in human subjects.40,44 Aflibercept has a longer half-life of 11days, suggesting that intravitreal aflibercept administration could have a longer dosing interval than either ranibizumab or bevacizumab.42,98 The need for injections every 12months can be a cause of discomfort and anxiety, and this together with the need for repeated office visits with time and cost implications all impact patient compliance with treatment regimens.99 Additionally, frequent intravitreal injections can increase the risk of complications, including submacular hemorrhage, intraocular hypertension, endophthalmitis, and retinal detachment.100, 101, 102 Given this, researchers have sought to develop long-acting anti-VEGF modalities, sustained-release formulation, and device, and they have looked to gene therapies to address the abovementioned issues.73. It is offered by the Division of Ophthalmology at Childrens Hospital of Philadelphia (CHOP) to children who meet certain criteria. Indeed, brolucizumab-treated patients showed greater fluid reduction, as evidenced by larger decreases in retinal thickness compared with aflibercept.38 A timeline of major advances in understanding ocular angiogenesis and the developments of antiangiogenesis therapy is shown in Figure2 (reviewed in Yang etal.,65 Ferrara,66 and Shah and Gardner67). The phase I/IIa clinical trial was completed in 2017 with 3-year follow-up results reported in 2019. The ocular gene therapy, LUXTURNA, is the worlds first approved gene replacement therapy for an inherited blinding eye condition and one of the first gene Importantly, after 14months, there were no indels at potential off-target sites, and the treatment did not affect retinal function or histology. Researchers have developed a new gene therapy that could eventually provide an alternative treatment for Fuchs endothelial corneal dystrophy, a genetic eye disease Dismuke D.J., Tenenbaum L., Samulski R.J. Biosafety of recombinant adeno-associated virus vectors. Current clinical results suggest that viral vector-mediated gene augmentation holds the potential to provide long-lasting therapeutic benefits for patients with nAMD or DME. It works by using a neutralised virus to carry a functional gene into the affected eye tissue. (AsCpf1) or Lachnospiraceae bacterium (LbCpf1)134,216 have been developed. Massachusetts Eye and Ear made medical history on Tuesday by performing the first post-FDA approval gene therapy for patients with a form of inherited blindness. Kvanta A. Ocular angiogenesis: the role of growth factors. PMC When you watch a sunset, an intricate series of events takes place in your eyes. The term gene therapy commonly refers to gene replacement, where a normal, functional copy of a gene is introduced by way of a delivery vector to replace a mutated gene in a targeted population of cells. As reported previously, participants who received the gene therapy injections were able to see dim lights that they werent able to see before treatment. A phase I/IIa clinical trial (ClinicalTrials.gov: {"type":"clinical-trial","attrs":{"text":"NCT03066258","term_id":"NCT03066258"}}NCT03066258, REGENXBIO) was started to evaluate the safety and tolerability of RGX-314 gene therapy in nAMD patients previously treated with VEGF injections in 2017. Sugita Y., Ito K., Shiozuka K., Suzuki H., Gushima H., Tomita M., Masuho Y. Recombinant soluble CD59 inhibits reactive haemolysis with complement. Mutation of key residues of RPE65 abolishes its enzymatic role as isomerohydrolase in the visual cycle. Gene therapy strategies for inherited retinal diseases. This functional barrier also prevents the leakage of genetic material into the systemic circulation and thus localizes the expression of the therapeutic gene to the eye.108,109 Given that genetic material can be largely compartmentalized to the eye, smaller doses of genetic material can be delivered.110 Moreover, target cells in the retina such as photoreceptors and RPE do not divide, and as such gene therapy produces prolonged effects.111 Thus, gene therapy provides the possibility for targeted, localized, and sustained delivery of therapeutic genetic material into specific intraocular sites.112. Kim D., Kim J., Hur J.K., Been K.W., Yoon S.H., Kim J.S. Stewart M.W. WebIn retinal gene therapy, the most widely used vectors for ocular gene delivery are based on adeno-associated virus. Additional imaging results for 3 of the treated participants were published on May 14, 2015, in New England Journal of Medicine. Improvement and Decline in Vision with Gene Therapy in Childhood Blindness. Int J Mol Sci. Scientists from Trinity College Dublin have developed a new gene therapy approach that offers promise for one day treating an eye disease that leads to a Heier J.S., Brown D.M., Chong V., Korobelnik J.F., Kaiser P.K., Nguyen Q.D., Kirchhof B., Ho A., Ogura Y., Yancopoulos G.D., VIEW 1 and VIEW 2 Study Groups Intravitreal aflibercept (VEGF trap-eye) in wet age-related macular degeneration. Gene Therapy Intervention in Neovascular Eye Disease: Shin J., Jiang F., Liu J.J., Bray N.L., Rauch B.J., Baik S.H., Nogales E., Bondy-Denomy J., Corn J.E., Doudna J.A. Holekamp N.M., Bouck N., Volpert O. Pigment epithelium-derived factor is deficient in the vitreous of patients with choroidal neovascularization due to age-related macular degeneration. Kubo T., Yanagihara K., Takei Y., Mihara K., Sato Y., Seyama T. siRNAs conjugated with aromatic compounds induce RISC-mediated antisense strand selection and strong gene-silencing activity. Cross M.J., Dixelius J., Matsumoto T., Claesson-Welsh L. VEGF-receptor signal transduction. government site. Bookshelf New gene therapy for eye disease - techexplorist.com CRISPR-Cas-based gene editing may be on the cusp of clinical application for ocular neovascularization. Despite successful gene knockout using dual-vector AAV systems, the need for co-transduction could be a drawback for their application.202 For example, the co-delivery of two AAV vectors has been reported to be less efficient than delivery by a single AAV vector invivo.203,217 Alternatively, a split SpCas9 approach is less active compared with the delivery of intact SpCas9.212,218 Other Cas endonucleases, such as NmCas9 and StCas9, have received less attention, as their longer protospacer-adjacent motif (PAM) regions (5-NNNNGATT-3 and 5-NNAGAAW-3, respectively) limit sequences available for targeting.208. The eye is an immunoprivileged organ and the retinal photoreceptors and retinal pigment epithelium are key target cells for gene therapy to treat several inherited retinal diseases. Hangai M., Murata T., Miyawaki N., Spee C., Lim J.I., He S., Hinton D.R., Ryan S.J. An 80% decrease in VEGFR2 production in CRISPR-SpCas9-edited MVECs was detected by western blot. The area of improvement, however, then underwent a contraction. 8600 Rockville Pike RGX-314 protein levels increased in aqueous humor in a dose-dependent manner across the five cohorts. Approved by the Therapeutic Goods Administration, LUXTURNA is used to treat children and adults with biallelic pathological mutations in The activation of Tie-2 receptors in endothelial cells reinforces junctional proteins and stabilizes the vasculature to limit permeability.75,76 While VEGF promotes sprouting and tube formation from primitive vessels in the early stage of angiogenesis, the angiopoietins/Tie-2 system impacts the late stages of angiogenesis by recruiting mural cells (mainly pericytes), mediating interactions between endothelium cells and pericytes, to stabilize new vessels through the formation of endothelial tight junctions.77 Angiopoietins are ligands that bind to Tie-2, regulating vascular development, maintenance, and permeability. As research progresses, we may also This work was supported by grants from the National Health and Medical Research Council of Australia (GNT1185600), the Ophthalmic Research Institute of Australia, and the Shenzhen Key Laboratory of Biomimetic Materials and Cellular Immunomodulation (ZDSYS20190902093409851). Watanabe D., Suzuma K., Suzuma I., Ohashi H., Ojima T., Kurimoto M., Murakami T., Kimura T., Takagi H. Vitreous levels of angiopoietin 2 and vascular endothelial growth factor in patients with proliferative diabetic retinopathy. The current treatment strategies in neovascular AMD involve mainly anti-vascular endothelial growth factor (VEGF) injections, which help to stabilize the disease and also improve vision. Petris G., Casini A., Montagna C., Lorenzin F., Prandi D., Romanel A., Zasso J., Conti L., Demichelis F., Cereseto A. Ashikari M., Tokoro M., Itaya M., Nozaki M., Ogura Y. Suppression of laser-induced choroidal neovascularization by nontargeted siRNA. Inhibition of vascular endothelial cell growth factor activity by an endogenously encoded soluble receptor. March 20, 2018 Boston, Mass. New hope for blinding eye disease gene therapies - Medical Zhong H., Chen Y., Li Y., Chen R., Mardon G. CRISPR-engineered mosaicism rapidly reveals that loss of. Ranibizumab is 5- to 30-fold more potent at neutralizing VEGF-A than is bevacizumab.51 Ranibizumab was shown to improve vision and was approved at a dose of 0.5mg every 4weeks for nAMD treatment in 2006,52,53 and 0.3mg every 4weeks for DR and DME in 2012.37 Bevacizumab was originally approved for treatment of metastatic colorectal cancer but has been used since 2005 as an off-label treatment for nAMD at a dose of 1.25mg every 4weeks.54 Because of its lower cost and greater availability, bevacizumab has become the first option for many patients.55,56 Aflibercept (Eylea) is a recombinant fusion protein that forms a VEGF trap, which targets VEGF-A, VEGF-B, and placental growth factor (PlGF).57 PlGF is another member of the VEGF family that activates VEGFR1.58 PlGF was reported to potentiate angiogenic signaling by forming heterodimers with VEGF-A, or by displacing VEGF-A from VEGFR1, and contributes to the development of neovascularization.59 Aflibercept is 100-fold higher than both bevacizumab and ranibizumab,60 and as such aflibercept provides comparable effectivity but requires less frequent injection compared with ranibizumab and bevacizumab. This strategy, in which genes are used to treat or prevent a disease, is known as gene therapy. Gene therapy requires the introduction of exogenous genetic material, such as DNA, RNA, small interfering (siRNA), microRNA (miRNA), and antisense oligonucleotides (synthesized nucleic acid sequence complementary to mRNA), into cells via viral or non-viral vectors to regulate, replace, or Do D.V., Rhoades W., Nguyen Q.D. They suggested that lowering the total capsid dose by removing empty AAV capsids could decrease inflammation and improve viral transduction. Diabetic retinopathy: research to clinical practice. Researchers have developed a new gene therapy that could eventually provide an alternative treatment for Fuchs endothelial corneal dystrophy, a genetic eye disease affecting roughly one in 2,000 people globally. WebThe team found that gene therapy improved patients eyesight and the sensitivity of the retina within a month of treatment. Proof-of-concept gene therapy studies using gene-editing tools such as CRISPR-Cas have already been shown to be effective in suppressing neovascularization in animal models, highlighting the therapeutic potential of the system for treatment of aberrant ocular angiogenesis. In these studies, the non-replicating bicistronic EIAV vector encoding both endostatin and angiostatin was subretinally injected to encode both endostatin and angiostatin in 21 participants with advanced nAMD (three cohorts of 2.4E4, 2.4E5, and 8E5 transduction units [TU]). Vavvas D.G., Small K.W., Awh C.C., Zanke B.W., Tibshirani R.J., Kustra R. Awh C.C., Lane A.M., Hawken S., Zanke B., Kim I.K. Retinal neovascularization can also occur on the iris and fluid drainage angle at the anterior eye, promoting the risk of neovascular glaucoma (reviewed in Rodrguez etal.22). Pattanayak V., Lin S., Guilinger J.P., Ma E., Doudna J.A., Liu D.R. Kendall R.L., Thomas K.A. Long-term effects of invivo genome editing in the mouse retina using. This stands to diminish the a need for repeated intraocular injections. Armulik A., Abramsson A., Betsholtz C. Endothelial/pericyte interactions. With continued development in both preclinical and clinical settings, gene therapy may be a viable alternative approach for the treatment of ocular neovascularization. Emerson M.V., Lauer A.K. Brolucizumab (Beovu), a humanized single-chain antibody fragment inhibitor of VEGF-A, is the most recent (October 2019) FDA-approved agent for nAMD at a recommended dosage of 6mg.64 Its small molecular mass (26kDa) allows for high solubility, extended duration of action, and improved ocular tissue penetration.55 Brolucizumab is the first drug to offer less frequent dosing (3-month dosing interval) in the first year of therapy while maintaining comparable effectiveness. Retinal and choroidal angiogenesis: a review of new targets. Approximately 6months of data from cohorts 4 (1.6E11 GC) and 5 (2.5E11 GC) showed on average a stable improvement in BCVA and central retinal thickness, with 42% and 75% of patients remaining anti-VEGF rescue injection-free, respectively.135 A longer-term follow-up study for RGX-314 (ClinicalTrials.gov: {"type":"clinical-trial","attrs":{"text":"NCT03999801","term_id":"NCT03999801"}}NCT03999801, REGENXBIO) has recently started. Gene therapy restores sight in people with eye disease Exp Eye Res. Generation of a recombinant, membrane-targeted form of the complement regulator CD59: activity invitro and invivo. Yannuzzi N.A., Freund K.B. New gene therapy saves the sight of children with eye disease In this review article, we will discuss the evolution of preclinical and clinical strategies that have improved gene therapy in the eye, showing that treatment of vision loss has a bright future. WebAbstract. Endpoints including the number of intravitreal anti-VEGF retreatment injections, BCVA, and central point thickness (CPT) were followed during 36months. Moreover, the aberrant growth of new vessels interferes with normal tissue structure and corneal transparency.2,3 Neovascularization is associated with a range of ocular disorders, including neovascular age-related macular degeneration (nAMD), diabetic retinopathy (DR), retinopathy of prematurity (ROP), corneal neovascularization, retinal vessel occlusion, and neovascular glaucoma (reviewed in Campochiaro4 and Usui etal.5). Clinical success has been realized for Lebers congenital amaurosis, with voretigene neparvovec-rzyl (Luxturna) becoming the first-ever FDA-approved gene therapy in 2017. But migraine is a genetic neurologic disease. Learn about Gene Therapy for Retinitis Pigmentosa and
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