Hematopoietic stem cell gene therapy leads to marked visceral organ improvements and a delayed onset of neurological abnormalities in the acid sphingomyelinase deficient mouse model of Niemann–Pick disease
- 1 October 2000
- journal article
- Published by Springer Nature in Gene Therapy
- Vol. 7 (20), 1768-1776
- https://doi.org/10.1038/sj.gt.3301300
Abstract
At the forefront of medicine, Gene Therapy brings you the latest research into genetic and cell-based technologies to treat disease. It also publishes Progress & Prospects reviews and News and Commentary articles, which highlight the cutting edge of the field.Keywords
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