GENE THERAPY FOR MACULAR DEGENERATIONThe invention provides compositions and methods for treatment of age-related macular degeneration, including gene therapy employing vectors and transgenes expressing protective CFH polypeptide and CFHT polypeptide sequences.HAGEMAN Gregory ScottRICHARDS Burt Timothy...
At present, such proteins that block VEGF proteins are injected by physicians into the eyes of people suffering from macular degeneration. This treatment helps control the overgrowth of abnormal, leaky blood vessels. However, this process must be repeated often and is cumbersome for patients and the...
With aging populations in many countries, the number of macular degeneration sufferers is expected to reach 196 million worldwide by 2020 and increase to 288 million by 2040, according toThe Lancet. Mohanty is the principal investigator for the research detailed in the article, 'Optical delivery ...
This open-label, multicenter, Phase 1, dosing finding trial (an open-laBel, dose-escalation study for CRISPR/Cas13-RNA targetInGtHerapy for theTreatment of neovascular age-related macular degeneration,BRIGHT) is designed to evaluate the safety and tolerability of high-fidelity CRISPR-Cas13 RNA-edit...
acular Degeneration 189. Therapeutic Gene Transfer as a Treatment Option for Age-Related Macular Degeneration189. Therapeutic Gene Transfer as a Treatment Option for Age-Related Macular DegenerationSome of the most successful gene therapy results have been obtained using recombinant viral vectors to treat...
A Phase 2 trial can begin investigations on MCO-010 gene therapy as treatment for patients withStargardt disease. The multi-characteristic opsin ambient-light activatable optogenetic monotherapy may restore vision in patients with this rare macular degeneration. ...
Autosomal recessive and X-linked disorders were identified as being ripe for gene supplementation therapy and over the last two decades these have advanced to clinical trials with one now available as an approved treatment. Therapeutic approaches have since evolved to encompass more complex strategies ...
Gene therapy is showing promise for one of the most common causes of blindness. Data presented today shows that six patients with wet age-related macular degeneration (AMD) have, so far, gone at least six months without the need for continued injections
Characterization of a novel Pde6b-deficient rat model of retinal degeneration and treatment with adeno-associated virus (AAV) gene therapy Article 29 September 2022 References Bessant DA, Ali RR, Bhattacharya SS . Molecular genetics and prospects for therapy of the inherited retinal dystrophies. ...
Choroidal neovascularization (CNV) is responsible for most cases of severe visual loss in age-related macular degeneration. Recently, the possibility of gene therapy has been proposed for the treatment of CNV. The purpose of this study was to examine the feasibility of ex vivo and in situ gene...