Gene therapy for infantile malignant osteopetrosis: review of pre-clinical research and proof-of-concept for phenotypic reversal
| Title: | Gene therapy for infantile malignant osteopetrosis: review of pre-clinical research and proof-of-concept for phenotypic reversal |
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| Authors: | Moscatelli, Ilana, Almarza, Elena, Schambach, Axel, Ricks, David, Schulz, Ansgar, Herzog, Christopher D., Henriksen, Kim, Askmyr, Maria, Schwartz, Jonathan D., Richter, Johan |
| Source: | In Molecular Therapy - Methods & Clinical Development 12 March 2021 20:389-397 |
| Database: | ScienceDirect |
| ISSN: | 23290501 |
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| DOI: | 10.1016/j.omtm.2020.12.009 |
| Published in: | Molecular Therapy - Methods & Clinical Development |
| Language: | English |