Researchers have developed two induced pluripotent stem cell (iPSC) lines with NAGLU mutations — the underlying cause of Sanfilippo syndrome type B — using CRISPR-Cas9 gene editing technology. Since iPSCs have the potential to generate virtually any other cell, including nerve cells, they can be used…
Researchers at the National Institutes of Health have developed an induced pluripotent stem cell (iPSC) line from a 1-year-old child with a mutated NAGLU gene, the gene responsible for Sanfilippo syndrome type B. These cells may now be used to generate other mature cells, such as neurons, to investigate…
Two new experimental stem cell lines derived from a patient with mucopolysaccharydosis IIIB (MPSIIIB), also known as Sanfilippo syndrome type B, were developed by Spanish researchers. These stem cells will complement existing mouse models of the disease and may allow the development of a more reliable human model of…
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