Gene therapy for infantile malignant osteopetrosis: review of pre-clinical research and proof-of-concept for phenotypic reversal

Erstveröffentlichung
2021Authors
Moscatelli, Ilana
Almarza, Elena
Schambach, Axel
Ricks, David
Schulz, Ansgar
Wissenschaftlicher Artikel
Published in
Molecular Therapy - Methods and Clinical Development ; 20 (2021). - S. 389-397. - eISSN 2329-0501
Link to publication
https://dx.doi.org/10.1016/j.omtm.2020.12.009Institutions
UKU. Klinik für Kinder- und JugendmedizinSubject headings
[Free subject headings]: STEM-CELL TRANSPLANTATION | AUTOSOMAL RECESSIVE OSTEOPETROSIS | SEVERE COMBINED IMMUNODEFICIENCY | BONE-MARROW-TRANSPLANTATION | CHRONIC GRANULOMATOUS-DISEASE | LENTIVIRAL VECTOR | CHLORIDE CHANNEL | PROTON PUMP | OSTEOCLAST | RESORPTION[DDC subject group]: DDC 610 / Medicine & health