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Gene therapy for in...
Gene therapy for infantile malignant osteopetrosis : review of pre-clinical research and proof-of-concept for phenotypic reversal
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- Moscatelli, Ilana (author)
- Lund University,Lunds universitet,Avdelningen för molekylärmedicin och genterapi,Institutionen för laboratoriemedicin,Medicinska fakulteten,Division of Molecular Medicine and Gene Therapy,Department of Laboratory Medicine,Faculty of Medicine
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- Almarza, Elena (author)
- Rocket Pharmaceuticals Ltd
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- Schambach, Axel (author)
- Hannover Medical School,Harvard University
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- Ricks, David (author)
- Rocket Pharmaceuticals Ltd
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- Schulz, Ansgar (author)
- University Hospital of Ulm
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- Herzog, Christopher D. (author)
- Rocket Pharmaceuticals Ltd
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- Henriksen, Kim (author)
- Nordic Bioscience AS
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- Askmyr, Maria (author)
- Lund University,Lunds universitet,Avdelningen för molekylärmedicin och genterapi,Institutionen för laboratoriemedicin,Medicinska fakulteten,Division of Molecular Medicine and Gene Therapy,Department of Laboratory Medicine,Faculty of Medicine
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- Schwartz, Jonathan D. (author)
- Rocket Pharmaceuticals Ltd
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- Richter, Johan (author)
- Lund University,Lunds universitet,Avdelningen för molekylärmedicin och genterapi,Institutionen för laboratoriemedicin,Medicinska fakulteten,Division of Molecular Medicine and Gene Therapy,Department of Laboratory Medicine,Faculty of Medicine
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(creator_code:org_t)
- Elsevier BV, 2021
- 2021
- English 9 s.
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In: Molecular Therapy - Methods and Clinical Development. - : Elsevier BV. - 2329-0501. ; 20, s. 389-397
- Related links:
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http://dx.doi.org/10... (free)
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http://www.cell.com/...
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https://doi.org/10.1...
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Abstract
Subject headings
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- 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.
Subject headings
- MEDICIN OCH HÄLSOVETENSKAP -- Medicinska och farmaceutiska grundvetenskaper -- Medicinsk genetik (hsv//swe)
- MEDICAL AND HEALTH SCIENCES -- Basic Medicine -- Medical Genetics (hsv//eng)
- MEDICIN OCH HÄLSOVETENSKAP -- Klinisk medicin -- Ortopedi (hsv//swe)
- MEDICAL AND HEALTH SCIENCES -- Clinical Medicine -- Orthopaedics (hsv//eng)
Keyword
- autosomal recessive osteopetrosis
- gene therapy
- hematopoietic stem and progenitor cells
- infantile malignant osteopetrosis
- lentivirus
- osteoclast disorders
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- By the author/editor
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Moscatelli, Ilan ...
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Almarza, Elena
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Schambach, Axel
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Ricks, David
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Schulz, Ansgar
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Herzog, Christop ...
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Henriksen, Kim
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Askmyr, Maria
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Schwartz, Jonath ...
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Richter, Johan
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- About the subject
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- MEDICAL AND HEALTH SCIENCES
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MEDICAL AND HEAL ...
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and Basic Medicine
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and Medical Genetics
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- MEDICAL AND HEALTH SCIENCES
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MEDICAL AND HEAL ...
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and Clinical Medicin ...
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and Orthopaedics
- Articles in the publication
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Molecular Therap ...
- By the university
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Lund University