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Emerging Paradigms in the Sickle Cell Disease Treatment Market

According to an estimate by the Centers for Disease Control and Prevention (CDC), SCD affects over 100,000 Americans and is a major public health concern as it resulted in 75,000 hospitalizations between 1989 through 1993, and cost over $475 million. The Medical expenditure of children with...

5 Companies Leveraging CRISPR to Herald the End of Genetic Diseases

CRISPR, which is an acronym for Clustered Regularly Interspaced Short Palindromic Repeats, is a revolutionary genome editing approach that helps change an individual’s DNA, by altering it at a specific location. The Crisper-Cas9, short for clustered regularly interspaced short palindromic repeats and CRISPR-associated protein 9,...

5 Companies unraveling the potential of Gene Based Therapies for the treatment of Sanfilippo syndrome (Mucopolysaccharidosis type III (MPS III)

Mucopolysaccharidosis type III (MPS III), also known as Sanfilippo syndrome, is caused by the mutations in the glucosamine (N-acetyl)-6-sulfatase (GNS), heparan-alpha-glucosaminide N-acetyltransferase (HGSNAT), N-acetyl-alpha-glucosaminidase (NAGLU) and N-sulfoglucosamine sulfohydrolase (SGSH) genes, which are responsible for the synthesis of large sugar molecules called glycosaminoglycans (GAGs). The dysfunction...

CAN GENE THERAPY AND RNA TECHNOLOGY COMBAT CYSTIC FIBROSIS?

Cystic Fibrosis is a chronic genetic disorder of the mucus glands that affects 2,500 newborns each year in the U.S.  30,000 people are currently affected by this disease and about 1 in 20 or roughly 12 million Americans are carriers of the “CF” gene and are unaware of...

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