Article

Engineering Memory T Cells as a platform for Long-Term Enzyme Replacement Therapy in Lysosomal Storage Disorders

2024-04-28

Abstract excerpt

Enzymopathy disorders are the result of missing or defective enzymes. Amongst these enzymopathies, mucopolysaccharidosis type I, is a rare genetic lysosomal storage disorder caused by mutations in the gene encoding alpha-L-iduronidase (IDUA), ultimately causes toxic build-up of glycosaminoglycans (GAGs). There is currently no cure and standard treatments provide insufficient relief to the skeletal structure and ce...

Topics

Open a Topic to create a Post that cites this publication.

Identifiers and source

Literature Corpus work: a4430309-af4b-5643-b15a-30239c1c886d
DOI: 10.1101/2024.04.23.590790

Open publication

Related research

Semantic proximity does not establish scientific evidence.

Click a neighbor to travelStep 1 · 12 closest

Engineering Memory T Cells as a platform for Long-Term Enzyme Replacement Therapy in Lysosomal Storage DisordersDOI 10.1101/2024.04.23.590790Read

Human genome-edited hematopoietic stem cells phenotypically correct Mucopolysaccharidosis type ISemantic proximity Read

Human genome-edited hematopoietic stem cells phenotypically correct Mucopolysaccharidosis type ISemantic proximity Read

Delivering gene therapy for mucopolysaccharide diseasesSemantic proximity Read

In vivo tracking of T cells in humans unveils decade-long survival and activity of genetically modified T memory stem cells.Semantic proximity Read

Current and Future Treatment of Mucopolysaccharidosis (MPS) Type II: Is Brain-Targeted Stem Cell Gene Therapy the Solution for This Devastating Disorder?Semantic proximity Read

Treatment of a lysosomal storage disease, mucopolysaccharidosis VII, with microencapsulated recombinant cells.Semantic proximity Read

Brain‐targeted stem cell gene therapy corrects mucopolysaccharidosis type II via multiple mechanismsSemantic proximity Read

TALEN-mediated intron editing of HSPCs enables transgene expression restricted to the myeloid lineageSemantic proximity Read

AAV delivered lysosome-targeting chimeras mediate sustained antibody depletion <i>in vivo</i>Semantic proximity Read

<i>In Vivo</i> Delivery of Therapeutic Molecules by Transplantation of Genome-Edited Induced Pluripotent Stem CellsSemantic proximity Read

Engineering monocyte/macrophage−specific glucocerebrosidase expression in human hematopoietic stem cells using genome editingSemantic proximity Read

Scalable GMP-compliant gene correction of CD4+ T cells with IDLV template functionally validated in vitro and in vivoSemantic proximity Read