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These studies suggest gene therapy shows promise for treating various rare diseases, including respiratory, neurological, metabolic, and central nervous system disorders, though more research is needed to ensure safety and efficacy.
20 papers analyzed
Gene therapy has emerged as a promising treatment for rare diseases, many of which are genetic in origin and lack effective treatments. This approach involves modifying or replacing defective genes to treat or prevent disease. Recent advancements in gene therapy have led to significant progress, particularly for rare neurological, metabolic, and respiratory diseases.
Gene Therapy for Neurological Diseases:
Gene Therapy for Metabolic Diseases:
Gene Therapy for Respiratory Diseases:
Gene Editing and Stem Cell Therapies:
Challenges and Future Prospects:
Gene therapy holds great promise for treating rare diseases, particularly those with a genetic basis. Significant strides have been made in developing therapies for neurological, metabolic, and respiratory conditions. While challenges remain, the continued advancement of gene editing technologies and stem cell therapies offers hope for more effective and widespread treatments in the future.
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