Stargardt disease is an inherited form of macular degeneration that results in blurry and distorted central vision without impacting peripheral vision.

Mutations in the ABCA4 gene are known to cause Stargardt disease. This gene produces a protein which normally clears away byproducts from vitamin A processing in retina. If this function fails, waste builds up and forms yellow-brown clumps of yellowish fatty material called lipofuscin that forms deposits on tissues throughout the retina.

1. Cloning

Over the past decade, researchers have conducted extensive studies into genetic treatments and cures for Stargardt disease. Utilizing techniques such as gene therapy and stem cell technology, they have explored whether it might be possible to effectively address its causes – potentially curing it altogether.

This eye disease is caused by mutations to specific genes that provide instructions for how your body processes vitamin A. When these instructions become corrupted, fatty deposits appear beneath retina in macula causing photoreceptor cells in center retina to break down and die out, leading to distortion and blurriness in central vision. When these photoreceptor cells die off completely central vision becomes fuzzy and disoriented.

Although Stargardt disease does not yet have a cure, patients can slow vision loss through laser treatment to seal leaking blood vessels in the retina and low vision aids that enhance peripheral vision use. Furthermore, regular visits to an ophthalmologist for preventive care and monitoring their progression of vision loss is beneficial as well.

Researchers have suggested that cloning may provide a potential treatment option for autism; however, this suggestion has raised many ethical concerns related to its possible application in therapy. Many feel cloning violates moral rights to unique identity; moreover there is little evidence proving its success as an approach.

Stargardt disease treatment research includes oral therapies shown to increase mitochondrial function. MitoChem Therapeutics has identified compounds which appear to enhance mitochondrial function while slowing retinal cell degeneration; clinical trials for these compounds will soon take place as potential treatment of Stargardt disease.

Cloning can also help treat this condition by producing stem cells with working ABCA4 genes that would produce retinal pigment epithelial (RPE) cells to insert into a patient’s eyes, providing more photoreceptor cells which convert light into electrical signals sent back to their brain for interpretation. This would restore vision by increasing photoreceptor cell count allowing more photoreceptor cells to convert light into electrical impulses which then reach your visual cortex for interpretation.

2. Genetic Therapy

Stargardt disease is a genetic eye condition characterized by a gradual loss of central vision. While no cure exists, vision aids may help manage symptoms and enhance quality of life for those living with the disease. Patients should also seek genetic counseling in order to understand inheritance patterns and risks related to passing gene mutations onto future generations, as well as making informed decisions regarding family planning or genetic testing.

Genetic therapy could be one effective method of treating Stargardt disease. Scientists can use cloning to create embryos free from recessive genes, then employ genetic engineering techniques to replace their defective ABCA4 gene with one that functions. This would potentially prevent symptoms from manifesting themselves during gestation or at birth; however, it would likely not work on those already suffering due to retinal cells being too old to regenerate themselves.

Another option is transplanting healthy retinal pigmented epithelial (RPE) cells into the retina. RPE cells play an essential supportive role for photoreceptors and may slow or halt vision loss, making RPE cell transplantation an effective treatment option for various retinal diseases – including Stargardt. However, its success will ultimately depend on having access to RPE cells with functional ABCA4 genes as well as being integrated successfully into the retina.

Stargardt disease treatments also include drug and gene therapies. A variety of companies are conducting trials for such treatments; Nightstar Therapeutics expanded its pipeline with gene therapy program specifically for Stargardt disease in November 2017 while Acucela Inc has an orphan drug designated called Emixustat for Stargardt treatment by Acucela Inc, Acucela’s C20-D3 Retinyl Acetate/Fenretinide is being evaluated; additionally ICR14967 acts as a complement inhibitor and XL-18 acts as an Ocular Stem cell transplantation therapy therapy treatment option.

Stargardt disease sufferers should hold out hope that there are treatment options in clinical trial that may become viable solutions in time. As these new treatments take shape, people living with Stargardt should speak to their doctor and gain support from friends, family members and professional counseling for Stargardt.

3. Stem Cell Therapy

Retinal degeneration conditions such as Stargardt disease, Age-Related Macular Degeneration (AMD), and Retinitis Pigmentosa are a leading cause of blindness among those aged 60 or over. While no cure exists yet for these diseases, several treatments are currently in development to slow their progress and enhance patients’ quality of life – one being stem cell therapy which involves replacing damaged retinal pigmented epithelial cells (RPEs) that support photoreceptors with healthy ones – in hopes of slowing further vision loss while possibly restoring some lost visual acuity acuity as scientists hope RPE replacement will prevent further loss or restore lost visual acuity acuity lost through RPE replacement therapy.

Stargardt disease is a genetic condition in which fatty material accumulates in the macula, responsible for sharp central vision. Over time, this accumulation can eventually lead to legal blindness; fortunately, various therapies are currently being tested in order to effectively treat and manage Stargardt disease; some include pharmaceutical drugs that reduce vitamin A dimers and lipofuscin accumulation while preventing complement system dysregulation in RPE cells.

Other studies are exploring gene therapy and stem cell therapies as potential treatments for Stargardt disease. One such research has demonstrated that inserting functional ABCA4 genes could cause new RPE cells to form to replace those lost to death, with working ABCA4 genes that allow phototransduction cycles to proceed normally and eliminate lipofuscin build-up.

embryonic stem cells may provide another promising treatment option for Stargardt disease: replacing damaged RPE cells in patients’ eyes with healthy ones and stopping or slowing its progression.

Embryonic stem cells can be extracted from the inner cell mass of a human blastocyst and have the capacity to differentiate into various specialized cells. Researchers and clinicians can also extract adult stem cells from patients, including hematopoietic or mesenchymal ones which produce blood cells or form bone marrow.

4. Gene Therapy

Doctors are currently exploring gene therapy as an approach to curing Stargardt disease. By injecting healthy genes into the retina, doctors hope they can restore its ability to detect light and transmit visual signals to the brain. Unfortunately, this treatment isn’t available widely but may provide hope to those hoping to avoid blindness.

One way they are doing this is by producing new photoreceptor cells – these are responsible for turning light into electrical signals that the brain can interpret – which they are then adding back into a person’s retina to restore vision, although this does not cure its cause; vision will eventually worsen again as new cells die off and too much A2E builds up again.

Dilute echothiophate iodide medication has also proven successful at treating stargardt disease, by blocking an enzyme responsible for producing A2E and thus helping it leave the eye without increasing risk for retinal damage. It’s relatively straightforward and already approved by other diseases such as glaucoma.

Trials for this treatment option are currently taking place, including one taking place in Europe where 12 patients with Stargardt Disease will be given this drug, and its results will be published later this year.

There are other trials being carried out, one of which involves screening embryos for gene mutations that cause stargardt disease and then implanting any found with this genetic mutation into mothers wombs – these children would resemble their mothers but would not suffer from stargardt.

Gene therapy is a complex process with numerous risks involved. Inserted genes may alter other functions within cells and potentially cause cancer or other conditions, so scientists take great precautions to make sure no unwanted side effects arise from inserting genes at specific times within cells to ensure maximum effectiveness.