Gene therapy for infantile malignant osteopetrosis : review of pre-clinical research and proof-of-concept for phenotypic reversal
(2021) In Molecular Therapy - Methods and Clinical Development 20. p.389-397- Abstract
Infantile malignant osteopetrosis is a devastating disorder of early childhood that is frequently fatal and for which there are only limited therapeutic options. Gene therapy utilizing autologous hematopoietic stem and progenitor cells represents a potentially advantageous therapeutic alternative for this multisystemic disease. Gene therapy can be performed relatively rapidly following diagnosis, will not result in graft versus host disease, and may also have potential for reduced incidences of other transplant-related complications. In this review, we have summarized the past sixteen years of research aimed at developing a gene therapy for infantile malignant osteopetrosis; these efforts have culminated in the first clinical trial... (More)
Infantile malignant osteopetrosis is a devastating disorder of early childhood that is frequently fatal and for which there are only limited therapeutic options. Gene therapy utilizing autologous hematopoietic stem and progenitor cells represents a potentially advantageous therapeutic alternative for this multisystemic disease. Gene therapy can be performed relatively rapidly following diagnosis, will not result in graft versus host disease, and may also have potential for reduced incidences of other transplant-related complications. In this review, we have summarized the past sixteen years of research aimed at developing a gene therapy for infantile malignant osteopetrosis; these efforts have culminated in the first clinical trial employing lentiviral-mediated delivery of TCIRG1 in autologous hematopoietic stem and progenitor cells. Infantile malignant osteopetrosis (IMO) presents a highly unmet medical need with limited therapeutic options. Gene therapy utilizing autologous hematopoietic stem and progenitor cells represents a potentially advantageous therapeutic alternative for this disease. Here, we have summarized all nonclinical studies supporting the initiation of a clinical gene therapy trial for IMO.
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- author
- Moscatelli, Ilana LU ; Almarza, Elena ; Schambach, Axel ; Ricks, David ; Schulz, Ansgar ; Herzog, Christopher D. ; Henriksen, Kim ; Askmyr, Maria LU ; Schwartz, Jonathan D. and Richter, Johan LU
- organization
- publishing date
- 2021
- type
- Contribution to journal
- publication status
- published
- subject
- keywords
- autosomal recessive osteopetrosis, gene therapy, hematopoietic stem and progenitor cells, infantile malignant osteopetrosis, lentivirus, osteoclast disorders
- in
- Molecular Therapy - Methods and Clinical Development
- volume
- 20
- pages
- 9 pages
- publisher
- Cell Press
- external identifiers
-
- pmid:33575431
- scopus:85100293648
- ISSN
- 2329-0501
- DOI
- 10.1016/j.omtm.2020.12.009
- language
- English
- LU publication?
- yes
- id
- 7b644f83-be9b-4765-a493-0c6d60adb72b
- date added to LUP
- 2021-02-11 09:13:02
- date last changed
- 2025-04-04 14:20:21
@article{7b644f83-be9b-4765-a493-0c6d60adb72b,
abstract = {{<p>Infantile malignant osteopetrosis is a devastating disorder of early childhood that is frequently fatal and for which there are only limited therapeutic options. Gene therapy utilizing autologous hematopoietic stem and progenitor cells represents a potentially advantageous therapeutic alternative for this multisystemic disease. Gene therapy can be performed relatively rapidly following diagnosis, will not result in graft versus host disease, and may also have potential for reduced incidences of other transplant-related complications. In this review, we have summarized the past sixteen years of research aimed at developing a gene therapy for infantile malignant osteopetrosis; these efforts have culminated in the first clinical trial employing lentiviral-mediated delivery of TCIRG1 in autologous hematopoietic stem and progenitor cells. Infantile malignant osteopetrosis (IMO) presents a highly unmet medical need with limited therapeutic options. Gene therapy utilizing autologous hematopoietic stem and progenitor cells represents a potentially advantageous therapeutic alternative for this disease. Here, we have summarized all nonclinical studies supporting the initiation of a clinical gene therapy trial for IMO.</p>}},
author = {{Moscatelli, Ilana and Almarza, Elena and Schambach, Axel and Ricks, David and Schulz, Ansgar and Herzog, Christopher D. and Henriksen, Kim and Askmyr, Maria and Schwartz, Jonathan D. and Richter, Johan}},
issn = {{2329-0501}},
keywords = {{autosomal recessive osteopetrosis; gene therapy; hematopoietic stem and progenitor cells; infantile malignant osteopetrosis; lentivirus; osteoclast disorders}},
language = {{eng}},
pages = {{389--397}},
publisher = {{Cell Press}},
series = {{Molecular Therapy - Methods and Clinical Development}},
title = {{Gene therapy for infantile malignant osteopetrosis : review of pre-clinical research and proof-of-concept for phenotypic reversal}},
url = {{http://dx.doi.org/10.1016/j.omtm.2020.12.009}},
doi = {{10.1016/j.omtm.2020.12.009}},
volume = {{20}},
year = {{2021}},
}