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

Erstveröffentlichung
2021
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Wissenschaftlicher Artikel


Authors
Moscatelli, Ilana
Almarza, Elena
Schambach, Axel
Ricks, David
Schulz, Ansgar
et al.
Institutions
UKU. Klinik für Kinder- und Jugendmedizin
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
Keywords
STEM-CELL TRANSPLANTATION; AUTOSOMAL RECESSIVE OSTEOPETROSIS; SEVERE COMBINED IMMUNODEFICIENCY; BONE-MARROW-TRANSPLANTATION; CHRONIC GRANULOMATOUS-DISEASE; LENTIVIRAL VECTOR; CHLORIDE CHANNEL; PROTON PUMP; OSTEOCLAST; RESORPTION
Dewey Decimal Group
DDC 610 / Medicine & health
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