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

Erstveröffentlichung

2021

Authors

Moscatelli, Ilana

Almarza, Elena

Schambach, Axel

Ricks, David

Schulz, Ansgar

et al.

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.2020.12.009

Institutions

UKU. Klinik für Kinder- und Jugendmedizin

Subject 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

Metadata

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