Erythroid-Progenitor-Targeted Gene Therapy Using Bifunctional TFR1 Ligand-Peptides in Human Erythropoietic Protoporphyria

Paper ID: PAPER000128
Year: 2019
DOI: 10.1016/j.ajhg.2018.12.021
PMID: 30712775
Journal: The American Journal of Human Genetics
Source URL

Experiments

EXP000633 with P1-9R (antisense oligonucleotide (AON))