Generation of induced pluripotent stem cell lines TRNDi045-A-40 and TRNDi045-A-41 modeling mucopolysaccharidosis type IIIA and type I from parental KOLF2.1J.

Mucopolysaccharidoses (MPS) are lysosomal storage disorders caused by enzyme deficiencies that impair glycosaminoglycan degradation and cause multisystem disease. We generated two human induced pluripotent stem cell (iPSC) lines from the KOLF2.1J background using CRISPR/Cas9-mediated knock-in of pathogenic variants: TRNDi045-A-40 carries a homozygous SGSH c.734G > A (p.Arg245His) variant associated with severe Sanfilippo syndrome type A (MPS IIIA), and TRNDi045-A-41 harbors a homozygous IDUA c.1205-1206 GG > AA (p.Trp402Ter) variant causing Hurler syndrome (MPS I). These lines model MPS diseases with different enzyme mutations. The resulting iPSCs are pluripotent, genetically validated, and support studies of disease mechanisms and therapies for MPS IIIA and MPS I.