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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...

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