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University of North Carolina at Chapel Hill: Breakthrough gene therapies in sight

Share Patients with genetic lysosomal storage diseases – particularly children – are living longer because of better treatments. But with promising advances and longer lives comes complications, the loss of eyesight as these rare diseases take their toll over time. Lysosomal storage diseases are inherited metabolic diseases that are characterized by an abnormal build-up of various toxic materials in the body’s cells as a result of enzyme deficiencies, according to the National Organization for Rare Disorders. With these diseases, patients are missing the mucopolysaccharidosis type 1 (MPS1) gene. Current therapies that allow patients to live longer don’t address corneal clouding caused by these diseases. However, one Carolina-affiliated startup, RainBio, is developing a novel gene therapy for MPSI corneal blindness, giving patients a chance not only for longer lives but better ones.

Breakthrough gene therapies in sight | UNC-Chapel Hill

Patients with genetic lysosomal storage diseases – particularly children – are living longer because of better treatments. But with promising advances and longer lives comes complications, the loss of eyesight as these rare diseases take their toll over time. Lysosomal storage diseases are inherited metabolic diseases that are characterized by an abnormal build-up of various toxic materials in the body’s cells as a result of enzyme deficiencies, according to the National Organization for Rare Disorders. With these diseases, patients are missing the mucopolysaccharidosis type 1 (MPS1) gene. Current therapies that allow patients to live longer don’t address corneal clouding caused by these diseases. However, one Carolina-affiliated startup, RainBio, is developing a novel gene therapy for MPSI corneal blindness, giving patients a chance not only for longer lives but better ones.

Lowcountry couple to use $300K lottery win to buy new home

Lowcountry couple to use $300K lottery win to buy new home A North Charleston couple said their house hunt just received a big boost thanks to a lottery win. (Source: Live 5/File) By Patrick Phillips | April 23, 2021 at 9:12 AM EDT - Updated April 23 at 9:12 AM NORTH CHARLESTON, S.C. (WCSC) - A North Charleston couple said their house hunt just received a big boost thanks to a lottery win. The couple, who did not want to be named, purchased a $10 Carolina Bonus Cash scratch-off ticket at the Carolina Express on Ashley Phosphate Road. “It happened at the right time,” the man said. “We’ve been looking at houses for a while.”