Researchers say they have successfully restored limited vision in a blind man using a new type of gene therapy, reports Tina Hesman Saey of Science News.
Congenital blindness is a hereditary disease and can be cured by gene therapy. Visual loss in children or infant can occur either at the stage of prenatal (during the time of conception or intrauterine period) or postnatal stage (during birth).
Luxturna was developed by the US company Spark Therapeutics, which was acquired by Roche in 2019. The therapy is designed to treat patients with mutations on a gene called RPE65, which encodes a retinal protein necessary for the eye to respond to light.
Atsena Therapeutics, a new Durham-based biotechnology company, announced that it has raised $8.2 million and acquired the rights to develop a potential treatment for a common form of childhood blindness.
How to Improve the Health of the Retina
- Healthy and balanced diet.
- Avoiding unhealthy foods and drinks.
- Drinking plenty of water.
- Regular exercise.
- Wearing sunglass when out in the sun.
- Quitting smoking.
- Wearing eye protection.
- Regular eye check-up.
To estimate as realistic a market price of gene therapy as possible, we calibrate our assumed price per ΔQALY with the 4 data points currently available: Zolgensma, priced at $2.1 million per patient [132], Luxturna, priced at $0.425 million per eye treated [157], Kymriah, priced at $0.475 million for a one-time dose [
Gene therapy replaces a faulty gene or adds a new gene in an attempt to cure disease or improve your body's ability to fight disease. Gene therapy holds promise for treating a wide range of diseases, such as cancer, cystic fibrosis, heart disease, diabetes, hemophilia and AIDS.
There are two types of gene therapy treatment: Somatic cell gene therapy and germline therapy. Somatic cell gene therapy involves obtaining blood cells from a person with a genetic disease and then introducing a normal gene into the defective cell (Coutts, 1998).
Cancer types, which have been targeted with gene therapy, include brain, lung, breast, pancreatic, liver, colorectal, prostate, bladder, head and neck, skin, ovarian, and renal cancer. Currently, two cancer gene therapy products have received market approval, both of which are in China.
Gene therapy is currently available primarily in a research setting. The U.S. Food and Drug Administration (FDA) has approved only a limited number of gene therapy products for sale in the United States.
Gene therapy is the introduction of genes into existing cells to prevent or cure a wide range of diseases. For example, suppose a brain tumor is forming by rapidly dividing cancer cells. The reason this tumor is forming is due to some defective or mutated gene.
Although gene therapy is a promising treatment option for a number of diseases (including inherited disorders, some types of cancer, and certain viral infections), the technique remains risky and is still under study to make sure that it will be safe and effective.
How Gene Therapy Works. Gene therapy aims to be given one-time to target a faulty gene that causes disease. Gene therapy is the introduction, removal or change in genetic material— DNA or RNA—into the cells of a patient to treat a specific disease.
The distinction between the two is based on purpose. Gene therapy seeks to alter genes to correct genetic defects and thus prevent or cure genetic diseases. Genetic engineering aims to modify the genes to enhance the capabilities of the organism beyond what is normal.
Gene therapy achieves this by adding a correct copy of the gene into the genome of the cells in the target organ or tissue, while gene editing alters the genome at a specific location to correct or alter the genetic sequence.
Gene therapy replaces a faulty gene or adds a new gene in an attempt to cure disease or improve your body's ability to fight disease. Gene therapy holds promise for treating a wide range of diseases, such as cancer, cystic fibrosis, heart disease, diabetes, hemophilia and AIDS.
Luxturna is the first approved treatment for an IRD in the US, Europe and hopefully soon in Canada. Luxturna is a gene therapy for individuals with RP or LCA who have mutations in both copies of the RPE65 gene. The therapy is not a cure but can improve vision and may slow the progression of vision loss.
Leber congenital amaurosis is an eye disorder that primarily affects the retina, which is the specialized tissue at the back of the eye that detects light and color. People with this disorder typically have severe visual impairment beginning in infancy.
The RPE65 gene provides instructions for making a protein that is essential for normal vision. The RPE65 protein is produced in a thin layer of cells at the back of the eye called the retinal pigment epithelium (RPE).
Leber congenital amaurosis
Retinitis pigmentosa (RP) is the collective name for a range of diseases that damage the light sensitive cells of the retina and cause vision to fade. There is currently no cure, and no treatments are available to slow the progression of disease. Symptoms include night blindness and tunnel vision.
Currently approved gene therapiesKymriah and Yescarta are two ex vivo gene therapies that are approved by Health Canada; they are chimeric antigen receptor T-cell (CAR-T) therapies and both are used in the treatment of cancer.
10 Top Gene Therapy Companies in 2021
- Asklepios BioPharmaceutical. Asklepios BioPharmaceutical is a clinical-stage company that focuses on genetic medicines.
- CRISPR Therapeutics.
- Editas Medicine.
- Homology Medicines.
- Intellia Therapeutics.
- Passage Bio.
- Pfizer.
- Poseida Therapeutics.
In short, the answer is no. Gene therapy is still an experimental approach for treating AMD and is being tested in clinical trials. More research is needed to find out if it is a safe and effective treatment for AMD.
Researchers have investigated a recessive genetic disorder that destroys the eyes from developing and results in childhood blindness. After analyzing the genomes of each member of a consanguineous family with affected children, the geneticists pinpointed pathogenic mutations in a new gene, MARK3, as being the cause. Certain viruses are often used as vectors because they can deliver the new gene by infecting the cell. The viruses are modified so they can't cause disease when used in people. Some types of virus, such as retroviruses, integrate their genetic material (including the new gene) into a chromosome in the human cell.
Currently, retinal implants are the only approved and commercially available bionic eyes, though cornea transplants and cataract surgery can replace the cornea and lens if these structures are clouded or are incapable of focusing light for other reasons.
The first gene therapy in the U.S. now has a price tag: $850,000 for the one-time treatment, or, more specifically, $425,000 per eye for a retinal disorder.
The inventions expand upon the technology behind voretigene neparvovec-rzyl (Luxturna™), an AAV vector gene therapy that treats an inherited form of blindness. Luxturna was discovered by Jean Bennett and others and is sold by Spark Therapeutics for $850,000 ($425,000 per eye).
