In two new papers, researchers from The Jackson Laboratory (JAX) report the successful use of two approaches—gene therapy and bone marrow transplantation—to alleviate symptoms of multiple sulfatase ...
Cell culturing of patient skin fibroblasts and HT-1080 fibrosarcoma cells as well as stable transfection with pSB-FGE-His plasmids were described earlier. 6, 7 The FGE-E130D mutation was created by ...
Carmine Settembre, Ida Annunziata, Carmine Spampanato, Daniela Zarcone, Gilda Cobellis, Edoardo Nusco, Ester Zito, Carlo Tacchetti, Maria Pia Cosma, Andrea Ballabio Proceedings of the National Academy ...
Philadelphia, September 9, 2024 – Researchers from Children’s Hospital of Philadelphia (CHOP) tested a preclinical model for an experimental gene therapy designed to treat multiple sulfatase ...
An Ocean Springs mother whose young child is suffering from a rare and incurable disease is stopping at nothing to find a treatment for her child and others like her. However, there are obstacles - ...
Willow Cannan, now 4, of Ocean Springs plays outside while at home with her family on Friday, July 22, 2016. Willow is living with an extremely rare genetic disease called multiple sulfatase ...
Heart tissue in healthy mice (left). Heart tissue in MSD mice (middle). When MSD mice received a bone transplant from healthy mice, the unhealthy heart tissue became healthy. In two new papers, ...
Kylee Cannan threw a very important first pitch Tuesday night as the Shuckers met Pensacola on Independence Day. She threw the pitch, which was sponsored by the Sun Herald, hoping it would help strike ...
WEBVTT COMMUTE OUT. RANDI: HAPPENING TONIGHT, A FUNDRAISER FOR A LITTLE MISSISSIPPI GIRL, BATTLING A RARE, GENETIC DISORDER. >> HER LITTLE BODY IS SLOWLY DYING BEFORE OUR EYES. RANDI: 5-YEAR-OLD ...
Amber Olsen's mission is to help other families faced with the same disease. There is not enough time in the day for Amber Olsen. An average day for this mother means working as a business owner of ...
PHILADELPHIA, Sept. 9, 2024 /PRNewswire/ -- Researchers from Children's Hospital of Philadelphia (CHOP) tested a preclinical model for an experimental gene therapy designed to treat multiple sulfatase ...
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