The Greenwood Genetic Center has designed a functional platform to assess the pathogenicity of variants of uncertain significance in IDUA identified by newborn screening for MPS I. With further ...

which is also known as Hurler or Hurler-Scheie syndrome. All three trials use ZFNs designed to edit liver cells at the same location in the albumin gene, but differ in delivering the corrective ...

There are currently seven known different types of MPS disorders: MPS I (Hurler-Scheie syndrome), MPS II (Hunter syndrome), MPS III (Sanfilippo syndrome), MPS IV (Morquio syndrome), MPS VI ...