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Discovering the potential of gene editing technology in treating rare disease

Around 200,000 people are thought to be affected by a rare disease. According to National Institutes of Health data, nearly 30 million Americans are thought to be affected by a rare disorder of some kind. Examples include neurodegenerative, familial, and blood-related conditions like Huntington's disease and muscular dystrophy.

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Discovering the potential of gene editing technology in treating rare disease

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  1. Discovering the potential of gene editing technology in treating rare disease Around 200,000 people are thought to be affected by a rare disease. According to National Institutes of Health data, nearly 30 million Americans are thought to be affected by a rare disorder of some kind. Examples include neurodegenerative, familial, and blood-related conditions like Huntington's disease and muscular dystrophy, as well as progressive degenerative diseases like cystic fibrosis and sickle cell disease.

  2. BioIntel360

  3. Though the precise underlying causes of all rare diseases are not fully understood. On the other hand, sporadic occurrences of some uncommon cancers or autoimmune diseases are possible. Lack of disease knowledge suggests that patients frequently go undiagnosed or receive the wrong diagnosis for a very long time. People with already limited treatment options are further delayed by the improper diagnosis, and many end up receiving therapy with drugs created to treat other diseases.

  4. Since over 90% of rare diseases lack any available approved therapies, individualized and targeted treatment options are necessary. For patients who are battling these life-threatening illnesses, this can be incredibly overwhelming and discouraging. Unprecedented improvements in sequencing technology in recent years have led to the identification of previously unknown rare disease mutations. On parallel lines, major developments in gene editing have enabled preclinical research and the advancement of clinical trials for rare diseases.

  5. Contact Us info@biointel360.com + 1 (813) 694-7124 http://biointel360.com/

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