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

**Type:** Papers  
**Canonical URL:** https://scholariq.org/papers/gene-therapy-for-infantile-malignant-osteopetrosis-review-of-pre-clinical/

## Facts

| Field | Value |
| --- | --- |
| Author Names | Ilana Moscatelli,Elena Almarza,Axel Schambach,David Ricks,Ansgar Schulz,Christopher D. Herzog,Kim Henriksen,Maria Askmyr,Jonathan D. Schwartz,Johan Richter |
| Citations | 13 |
| DOI | 10.1016/j.omtm.2020.12.009 |
| Fields | Biochemistry, Genetics and Molecular Biology |
| Open Access | true |
| OA Status | gold |
| OA URL | http://www.cell.com/article/S2329050120302576/pdf |
| OpenAlex ID | https://openalex.org/W3117474922 |
| PMID | 33575431 |
| Type | article |
| Year | 2020 |

## Paper authors

- [Axel Schambach](https://scholariq.org/researchers/axel-schambach/)
- [Ansgar Schulz](https://scholariq.org/researchers/ansgar-schulz/)
- [Kim Henriksen](https://scholariq.org/researchers/kim-henriksen/)
- [Jonathan D. Schwartz](https://scholariq.org/researchers/jonathan-d-schwartz/)
- [Johan Richter](https://scholariq.org/researchers/johan-richter/)

## Paper journal

- [Molecular Therapy — Methods & Clinical Development](https://scholariq.org/journals/molecular-therapy-methods-and-clinical-development/)

## Paper primary topic

- [Virus-based gene therapy research](https://scholariq.org/topics/virus-based-gene-therapy-research/)

## Paper topics

- [Virus-based gene therapy research](https://scholariq.org/topics/virus-based-gene-therapy-research/)
- [RNA Interference and Gene Delivery](https://scholariq.org/topics/rna-interference-and-gene-delivery/)
- [Bone Metabolism and Diseases](https://scholariq.org/topics/bone-metabolism-and-diseases/)

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Source: ScholarIQ — public research metadata, principally OpenAlex. See https://scholariq.org/sources/ for provenance and https://scholariq.org/methodology/ for what these figures mean.