Gene therapy helps treat rare diseases by correcting genetic defects that cause the disease rather than addressing symptoms, offering the potential for long-term or curative solutions. By targeting the root cause, gene therapy aims to reduce symptom severity and frequency, decrease reliance on medications, and improve the ability to live independently.
While initial costs are high, the therapy could lower long-term healthcare expenses by reducing the need for continuous treatments and hospitalizations. Gene therapy also supports personalized medicine by tailoring treatments to specific genetic mutations. It has potential applications across many rare diseases, including cystic fibrosis, muscular dystrophy, and certain inherited forms of blindness.
Advancements in research and clinical trials continue to explore and expand the possibilities of gene therapy. Researchers must understand what happens with the body’s 25,000 genes, especially the 22,00 protein-encoding genes that express around 100,000 proteins and what the genes and proteins do and how, when, where, and in what sequence could hold the key to helping patients with rare diseases without lethal side effects. With ongoing progress, gene therapy could reshape the treatment landscape for rare diseases, bringing new options to patients.
While initial costs are high, the therapy could lower long-term healthcare expenses by reducing the need for continuous treatments and hospitalizations. Gene therapy also supports personalized medicine by tailoring treatments to specific genetic mutations. It has potential applications across many rare diseases, including cystic fibrosis, muscular dystrophy, and certain inherited forms of blindness.
Advancements in research and clinical trials continue to explore and expand the possibilities of gene therapy. Researchers must understand what happens with the body’s 25,000 genes, especially the 22,00 protein-encoding genes that express around 100,000 proteins and what the genes and proteins do and how, when, where, and in what sequence could hold the key to helping patients with rare diseases without lethal side effects. With ongoing progress, gene therapy could reshape the treatment landscape for rare diseases, bringing new options to patients.
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