Stem cell transplantation for osteopetrosis in patients beyond the age of 5 years.
Stepensky P, Grisariu S, Avni B, Zaidman I, Shadur B, Elpeleg O, Sirin M, Hoenig M, Schuetz C, Furlan I, Beer M, von Harsdorf S, Bunjes D, Debatin KM, Schulz AS.
Stepensky P, et al.
Blood Adv. 2019 Mar 26;3(6):862-868. doi: 10.1182/bloodadvances.2018025890.
Blood Adv. 2019.
PMID: 30885997
Free PMC article.
Here we present data from 7 patients with intermediate OP caused by mutations in TCIRG1 (n = 2), CLCN7 (n = 2), RANK (n = 1), SNX10 (n = 1), and CA2 (n = 1), who were transplanted between the ages of 5 to 30 years (mean, 15; median, 12). ...
Here we present data from 7 patients with intermediate OP caused by mutations in TCIRG1 (n = 2), CLCN7 (n = 2), RANK (n = 1), SNX10 ( …