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Retinoschisis is an eye disease characterized by the abnormal splitting of the retina's neurosensory layers, usually in the outer plexiform layer.Retinoschisis can be divided into degenerative forms which are very common and almost exclusively involve the peripheral retina and hereditary forms which are rare and involve the central retina and sometimes the peripheral retina.
Retinoschisin also known as X-linked juvenile retinoschisis protein is a lectin [5] [6] that in humans is encoded by the RS1 gene. [7]It is a soluble, cell-surface protein that plays an important role in the maintenance of the retina where it is expressed and secreted by retinal bipolar cells and photoreceptors, [8] [9] as well as in the pineal gland. [10]
MeiraGTx's botaretigene sparoparvovec targets X-linked retinitis pigmentosa through RPGR gene restoration and Beacon Therapeutics’ laruparetigene zosaparvovec aims at similar conditions, showing retinal sensitivity improvements in trials. [8] Another notable effort is Atsena Therapeutics’ ATSN-201 for X-linked retinoschisis. [9]
This disease is an X-linked recessive degenerative disease of the central macula region, and it is caused by mutation in the RSI gene encoding the protein retinoschisin. Retinoschisin is produced in the photoreceptor and bipolar cells and it is critical in maintaining the synaptic integrity of the retina.
The company focuses on ophthalmologic genetic diseases. The company's technologies are aimed at products that might have the potential to treat achromatopsia, X-linked retinoschisis, [5] [6] X-linked retinitis pigmentosa, [7] and age-related macular degeneration. [8]
His lab studies potential treatments for a juvenile form of macular degeneration termed X-linked retinoschisis (XLRS). The lab developed a mouse model of XLRS and successfully treated the condition by gene therapy in mouse [ 3 ] [ 4 ] and rabbit models. [ 5 ]
DURHAM, N.C., Jan. 08, 2025 (GLOBE NEWSWIRE) -- Atsena Therapeutics, a clinical-stage gene therapy company focused on bringing the life-changing power of genetic medicine to reverse or prevent blindness, today announced the initiation of Part B of the LIGHTHOUSE study, a Phase I/II clinical trial evaluating subretinal injection of ATSN-201 for the treatment of X-linked retinoschisis (XLRS).
Choroideremia (/ k ɒ ˌ r ɔɪ d ɪ ˈ r iː m i ə /; CHM) is a rare, X-linked recessive form of hereditary retinal degeneration that affects roughly 1 in 50,000 males. The disease causes a gradual loss of vision, starting with childhood night blindness, followed by peripheral vision loss and progressing to loss of central vision later in life.