Article
Mucopolysaccharidosis Type I Phenotypically Corrected with Edited Hematopoietic Stem Cells: Instead of altering the IDUA gene, a protein was inserted in a repurposable place in the genome known as a "safe harbor locus".
American journal of medical genetics. Part A - 1 Feb 2020
Abstract excerpt
No abstract is available from the source.
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.