FUTURE THERAPIES
RP is a rare inherited eye disease with no cure. Existing treatments only help a fraction of the estimated 100,000 Americans with this condition. Some future treatments for retinitis pigmentosa (RP) include Stem cell therapy, gene therapy, Optogenetics, neuroprotective agents, nanobodies, N-acetylcysteine-amide (NACA).
Stem Cell Therapy | Stem cells can differentiate into various cell types, including photoreceptor cells and RPE cells, to replace lost or damaged cells.
Gene Therapy | A therapeutic gene can be injected to replace a disease-causing gene.
Optogenetics | Photosensitive proteins can be introduced into the retina to restore cone function.
Neuroprotective Agents | These include antioxidants, anti-apoptotic agents, and neurotrophic factors. They can be used in the early stages of the disease or as an adjunctive treatment.
Nanobodies | These can recognize the target rhodopsin extracellularly and lock it in a non-signaling state.
N-acetylcysteine-amide (NACA) | This molecule is designed to slow vision loss by protecting retinal cells from oxidative stress.
Other approaches under investigation include small molecules that target enzymes and receptors in cell signaling pathways involved in sight and exosomes that can deliver biologically active molecules to specific cells and tissues.
Stem Cell Research
Stem cell research is the study of stem cells to learn more about human biology and to develop treatments for diseases. By observing stem cells mature into different cell types, researchers can learn how diseases develop and how healthy cells replace damaged cells. Stem cells can be used to create "organoids", which are small models of organs, to study diseased cells and test drugs. Stem cells can be used to test new drugs to see if they are safe and effective before they are used in clinical trials. Stem cell treatments are being developed for a variety of conditions, including neurodegenerative diseases, diabetes, and heart disease. Stem cell research has raised controversy due to the ability to isolate and culture embryonic stem cells, and the ability to create induced pluripotent stem cells. The Catholic Church supports ethically responsible stem cell research, but opposes research that exploits or destroys human embryos.
CRISPR TECHNOLOGY
CRISPR (clustered regularly interspaced short palindromic repeats) is a technology that allows researchers to modify an organism's DNA. It's based on a natural defense system found in bacteria that helps protect them from viruses. In 1993, Dr. Francisco Mojica discovered CRISPR in bacterial genomes. He proposed that the repetitive DNA sequences were part of the bacteria's immune system. It works when a virus infects a bacterium, the bacterium transcribes the CRISPR DNA sequences into RNA. The RNA guides a protein called a nuclease to the viral DNA, where it cuts the DNA. This protects the bacterium from the virus.
CRISPR is used in laboratories to modify the DNA of living organisms. The CRISPR Cas9 protein is added to a cell along with a guide RNA. The Cas9 protein binds to the guide RNA and moves along the DNA strands until it finds a matching sequence. The Cas9 protein then cuts the DNA at the target, which usually disables a gene. CRISPR has many applications, including gene therapy, RNA editing, base editing, and diagnostics. In 2024, the FDA approved CRISPR's Casgevy gene-editing therapy to treat beta thalassemia, a rare blood disorder.
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