Stargardt disease affects the macula of your retina (light processing tissue lining the inner eye) which specializes in central vision and fine details. This area provides you with sharpest central vision.

Optic neuritis can cause blurry or wavy vision as well as blind spots, often first manifesting itself during childhood or adolescence.

Gene Therapy

Gene therapy has shown promise as an approach for treating some retinal diseases, including Stargardt disease. This technique involves replacing defective genes with functional ones; particularly promising is that eye is an immune-privileged organ; therefore introducing therapeutic proteins directly into eye can potentially avoid some of the side effects associated with intravenous delivery methods.

Recent publications of case reports document early and sustained improvements in visual acuity and color vision for patients diagnosed with Stargardt disease after receiving topical administration of echothiophate iodide drug treatment. These developments are encouraging, especially as this condition typically leads to legal blindness over time without treatment.

This case study chronicled a patient who had an alteration of PROM1 gene that causes its protein product, PROM1, to play an essential role in photoreceptor formation and light detection by responding cellular mechanisms and producing electrochemical signals for vision.

In the case of this particular patient, a mutation to PROM1 causes proteins to remain trapped in a region known as the myoid zone rather than migrating to OS sites where disks form; this leads to abnormal deposits accumulating within their macula which could ultimately result in central macular degeneration.

Studies have demonstrated that transfection of RPE cells with genetically engineered human PEDF protein slows or stops degeneration and restores vision in animal models of Stargardt’s Macular Degeneration; however, this approach is currently unavailable for human use. Anti-VEGF agents (antivascularization Growth Factor Agents) have proven highly successful at decreasing age-related macular degeneration neovascularization; these treatments may also be combined with various forms of therapy like anti-inflammatory medication and nutritional supplements for greater effect.

OCU410, a gene therapy product specifically targeting AMD by reversing genetic mutations, is currently under clinical trials as part of a Phase 1 clinical trial that is set to conclude enrollment by fall of 2020. This therapy can be delivered locally into the eye through injection or used alongside anti-VEGF agents in order to decrease neovascular AMD risk.

Stem Cell Therapy

Stem cells are special cells with unique properties. They possess the capacity to divide into multiple, specialized cells with various functions and self-renew, making them vital components in medical procedures that utilize regenerative medicine for treating disease and injury. Two main types of stem cells used for regenerative medicine treatments are embryonic stem cells and adult stem cells; embryonic ones possess multipotency so can become any cell type while adult ones have limited differentiation potential; in regenerative medicine procedures these cells may come from either their own body or from sources like bone marrow, umbilical cord blood, liver dental pulp and skin cells from either embryonic stem cells or adult ones obtained through transplant.

Regenerative medicine is rapidly progressing. Scientists are discovering innovative methods to encourage the proliferation of specific cells that can then be utilized in surgical procedures for injuries or diseases, including spinal cord injuries. For instance, stem cell therapies used to repair spinal cord injuries have produced remarkable results; patients undergoing such therapies experience improved motor function while some even regain their ability to walk again!

Regenerative medicine offers another avenue of relief through stem cell use to promote the formation of cartilage. Articular cartilage protects joints from degeneration and pain, so damage to it may lead to degeneration of joint tissue and pain. Researchers have discovered that placing progenitor or stem cells at fracture or nonunion sites may help speed up formation of primary hyaline cartilage formation.

Stem cell therapies are being explored as treatments for various musculoskeletal conditions in orthopedics. Currently, stem cells are being utilized to reduce scarring/fibrosis, repair structures damaged by trauma and alleviate pain; healing bone fractures/nonunions; regenerate articular cartilage in arthritic joints; repair ligaments and tendons are all possible through stem cell therapies.

One risk associated with adipose-derived stem cells is the possibility that bacteria, viruses or other pathogens could enter damaged tissues during harvesting and injection of the cells. An infection in damaged tissue could impede healing while also hindering their therapeutic effect.

Other Treatments

Stargardt disease, a hereditary retinal condition affecting light processing tissue lining the inside surface of the eye), cannot be cured; instead it gradually degrades over time from childhood or adolescence onward. Due to the loss of light-sensing rods and cones called rods and cones, vision gradually degrades with age; useful vision may still persist for some years, though eventually enough photoreceptors deteriorate that central vision disappears completely and reading and driving become impossible while peripheral vision still may remain.

Diseased deposits of fat beneath the retina cause cones and rods from receiving light, leading to gradual, sporadic declines in central visual acuity starting as early as childhood or adolescence. With time, reading becomes difficult or handwork become challenging; drivers may have trouble recognising faces or facial expressions and people may even struggle with driving safely or recognising facial features and expressions; eventually deterioration reaches 20/200 or worse which is considered legal blindness.

Juvenile macular degeneration has been linked to genetic mutations in multiple genes. Some mutations are autosomal dominant, meaning an affected individual passes their gene on to all their children without fail; other types may only pass down one chance in four of being passed onto subsequent generations.

Foundation Fighting Blindness is conducting a clinical trial testing a new experimental drug called ALK-001 as an experimental Stargardt disease treatment. ALK-001 is a synthetic compound which increases levels of an essential chemical found in eye tissue called acetylcholine for brain and retina cell transmission, slowing or stopping its progress of macular degeneration in Stargardt patients as well as other conditions causing similar degeneration. Researchers hope ALK-001 may also be useful against other causes of macular degeneration.

Stargardt’s macular degeneration can also be treated using protective eyewear such as sunglasses and wide-brimmed hats on bright days when outdoors, to block UV rays that cause new blood vessels to form beneath the macula, leaking fluids out and leading to bleeding similar to wet age-related macular degeneration. Doctors may then inject patients with drugs which block vascular endothelial growth factor which causes this leakage and bleeding in these new blood vessels.