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Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy
Open accessAbstract
Slowing Brain Disease with Gene Therapy: X-linked adrenoleukodystrophy (ALD), the hereditary brain demyelinating disorder that was featured in the movie “ Lorenzo's Oil ,” is typically treated by transplantation of bone marrow from matched donors. This treatment slows progression of the disease by introducing cells that differentiate into myelin-producing cells. Cartier et al. (p. 818 ; see Perspective by Naldini ) tested an alternative gene therapy–based approach in two young patients without matched donors. A lentiviral vector was used to introduce a wild-type copy of the ALD gene into the patients' hematopoietic stem cells ex vivo. The modified cells were then infused back into the patients. Expression of the transferred gene was still detectable in the patients' blood cells 2 years later, and both patients showed neurological improvement and a delay in disease progression comparable to that seen with bone marrow transplants.
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“Expansions of cells with a common integration site were observed in a gene therapy trial using lentiviral vectors for adrenoleukodystrophy [51].”
From Clinical use of lentiviral vectors (2018) · cites this paper as [51]
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In the citing paper
“Gene therapy is a cutting-edge approach that has shown potential in various genetic disorders, including X-ALD.”
From X-linked adrenoleukodystrophy and primary adrenal insufficiency (2023) · cites this paper as [48]
Experimental — accuracy not yet measuredThe cited source says what the claim attributes to it.
In the citing paper
“Lentiviral vector-based gene transfer into CD34 + HSCs has subsequently been applied in the treatment of several genetic diseases, including β-thalassemia [67], X-linked adrenoleukodystrophy [51], metachromatic leukodystrophy [68, 69], and Wiskott-Aldrich Syndrome [70].”
From Clinical use of lentiviral vectors (2018) · cites this paper as [51]
The cited source says what the claim attributes to it.
In the citing paper
“Early-stage treatment of childhood cerebral ALD with hematopoietic stem cell transplantation and, more recently, gene therapy can arrest disease progression if performed in the window of opportunity when the MRI is abnormal but neurological symptoms are not yet apparent [7,38,43,44,45,46,47,48,49,50].”
From Structure and Function of the ABCD1 Variant Database: 20 Years, 940 Pathogenic Variants, and 3400 Cases of Adrenoleukodystrophy (2022) · cites this paper as [50]
The cited source says what the claim attributes to it.
In the citing paper
“A lentiviral vector has been used to introduce a wild-type copy of the ABCD1 gene into the patients’ hematopoietic stem cells ex vivo. Subsequently, the genetically modified cells have been reintroduced into the patients (48).”
From X-linked adrenoleukodystrophy and primary adrenal insufficiency (2023) · cites this paper as [48]
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In the citing paper
“The positive results of HSC transplantation into cALD patients [24,356] indicated a role for brain microglia because microglia were thought to originate in bone marrow at that time [357].”
From Revisiting the Pathogenesis of X-Linked Adrenoleukodystrophy (2025) · cites this paper as [356]
In the citing paper
“In ALD, phagocytes derived from autologous bone marrow transplants likely elicit their beneficial effects by replacing mutant microglia, as they stall terminal neuro-inflammation and demyelination 42.”
From Efficient derivation of microglia-like cells from human pluripotent stem cells (2016) · cites this paper as [42]
In the citing paper
“Allogenic hematopoietic stem cell transplantation [145,146] and autologous stem cell-based gene therapy [147] can arrest the inflammatory demyelinating process with a typical delay of 12–18 months, which has been attributed to the slow replacement of microglia with bone marrow-derived phagocytes [147,148].”
From Peroxisomes in brain development and function (2016) · cites this paper as [147]
In the citing paper
“This is strongly supported by the fact that both allogeneic hematopoietic stem cell (HSC) transplantation and autologous HSC gene therapy are very effective in arresting and even reversing neuroinflammatory cerebral ALD, provided that the procedure is performed at an early stage [101,102].”
From X-linked adrenoleukodystrophy: Clinical, metabolic, genetic and pathophysiological aspects (2012) · cites this paper as [102]
In the citing paper
“In 2009, autologous HSCT was demonstrated to be successful to halt cerebral demyelination in two boys with no compatible donors who received their own genetically corrected stem cells [ 82 ].”
From Peroxisomal ABC Transporters: An Update (2021) · cites this paper as [82]
In the citing paper
“These promising results suggest that such a therapeutic strategy may be as effective as allogeneic HSCT.”
From Peroxisomal ABC Transporters: An Update (2021) · cites this paper as [82]
In the citing paper
“the CD34+ PBMCs (lymphoid and myeloid progenitors) transduced with normal ABCD1 can efficiently correct the clinical phenotype of the X-ALD patients [ 82 ]”
From Peroxisomal ABC Transporters: An Update (2021) · cites this paper as [82]
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“Devices for direct host cell modification Genetic modification of a patient’s cells offers a direct means to treat chronic cell and tissue dysfunction, and efforts in viral gene therapy have enjoyed increasing success in establishing the feasibility and efficacy of this approach [6–14].”
BackgroundDighe, N., Khoury, M., Mattar, C. N. Z., Chong, M. S. K., Choolani, M. A., Chen, J., Antoniou, M. N., & Chan, J. K. Y. (2014) · PLoS ONE · doi:10.1371/journal.pone.0104805 · Cited by 23
“HSC-based gene therapy is gaining traction with a number of clinical trials targeting different conditions (X-SCID, ADA-SCID, ALD, CGD, Fanconi anemia, b-thalassemia) showing encouraging results [2,3,6,27-30] and which have implications for curative lifelong treatment.”
BackgroundRecent Developments in Therapeutic Approaches for Lysosomal Storage Diseases
Urbanelli, L., Magini, A., Polchi, A., Polidoro, M., & Emiliani, C. (2010) · Recent Patents on CNS Drug Discovery · doi:10.2174/157488911794079127 · Cited by 22
Baron, Y., Sens, J., Lange, L., Nassauer, L., Klatt, D., Hoffmann, D., Kleppa, M., Barbosa, P. V., Keisker, M., Steinberg, V., et al. (2022) · Molecular Therapy — Nucleic Acids · doi:10.1016/j.omtn.2021.12.033 · Cited by 23
Newborn Screening for X-Linked Adrenoleukodystrophy in Nebraska: Initial Experiences and Challenges
Baker, C. V., Keller, A. C., Lutz, R., Eveans, K., Baumert, K., DiPerna, J. C., & Rizzo, W. B. (2022) · International Journal of Neonatal Screening · doi:10.3390/ijns8020029 · Cited by 23
“Progressive neurologic disease in boys with ccALD can be halted if treated with allogeneic hematopoietic stem cell transplantation (HSCT) at the earliest onset of demyelination [11–13] and lentiviral-mediated ex vivo gene therapy is under investigation as an alternative option [14,15].”
BackgroundKim, Y.-S., Wielgosz, M. M., Hargrove, P. W., Kepes, S., Gray, J., Persons, D., & Nienhuis, A. (2010) · Molecular Therapy · doi:10.1038/mt.2010.48 · Cited by 25
Jiang, Y., Wei, N., Zhu, J., Zhai, D., Wu, L., Chen, M., Xu, G., & Liu, X. (2011) · Neuroscience · doi:10.1016/j.neuroscience.2011.10.043 · Cited by 22
Persons, D. A. (2010) · Nature · doi:10.1038/467277a · Cited by 22
Adeno‐Associated Virus Vector–Based Gene Therapy for Monogenetic Metabolic Diseases of the Liver
Junge, N., Mingozzi, F., Ott, M., & Baumann, U. (2015) · Journal of Pediatric Gastroenterology and Nutrition · doi:10.1097/mpg.0000000000000703 · Cited by 21
Stein, S., Scholz, S., Schwäble, J., Sadat, M., Modlich, U., Schultze‐Strasser, S., Diaz, M., Chen-Wichmann, L., Müller-Kuller, U., Brendel, C., et al. (2013) · Human Gene Therapy Clinical Development · doi:10.1089/humc.2013.019 · Cited by 23
“…Diez et al., 2011; Huston et al., 2011; Tolar et al., 2012; Scaramuzza et al., 2013) and ongoing clinical trials, although the follow-up time of treated patients is still too short to make conclusive statements at present (Cartier et al., 2009; Cavazzana-Calvo et al., 2010; Biffi et al., 2011).”
Self-citationGori, J. L., Beard, B., Ironside, C. M., Karponi, G., & Kiem, H. (2012) · Cancer Gene Therapy · doi:10.1038/cgt.2012.25 · Cited by 22
Structure–activity relationship studies of symmetrical cationic bolasomes as non-viral gene vectors
Huang, Z., Zhang, Y., Cheng, Q., Zhang, J., Liu, Y., Wang, B., & Yu, X. (2016) · Journal of Materials Chemistry B · doi:10.1039/c6tb00870d · Cited by 22
Chromosome therapy: Potential strategies for the correction of severe chromosome aberrations
Plona, K. L., Kim, T. H., Halloran, K., & Wynshaw-Boris, A. J. (2016) · American Journal of Medical Genetics Part C Seminars in Medical Genetics · doi:10.1002/ajmg.c.31530 · Cited by 22
“Other suitable candidates for clinical use are situations where the diseased tissue can be removed from patients, corrected ex vivo in lab conditions, and re-transplanted into the patient (i.e, bone marrow) [De Ravin et al.; Cartier et al., 2009].”
BackgroundGeric, I., Tyurina, Y. Y., Krysko, O., Krysko, D. V., de Schryver, E., Kagan, V. E., Veldhoven, P. P. V., Baes, M. I., & Verheijden, S. (2017) · Immunology · doi:10.1111/imm.12844 · Cited by 22
The Molecular Imaging of Natural Killer Cells
Shapovalova, M., Pyper, S. R., Moriarity, B. S., & LeBeau, A. M. (2018) · Molecular Imaging · doi:10.1177/1536012118794816 · Cited by 22
“The expression of iRFP682 in our system was driven by a strong, constitutive MND promoter.(58) Flow cytometery and a LiCor Odyssey Infrared Imager were used to confirm expression of iRFP682 in modified primary NK cells that had expanded ex vivo (Figure 1A and B).”
MethodPerfecting Pregnancy: Law, Disability, and the Future of Reproduction
Karpin, I., & Savell, K. (2012) · UTS ePRESS (University of Technology Sydney) · Cited by 22
Lentiviral vectors for induction of self-differentiation and conditional ablation of dendritic cells
Pincha, M., Salguero, G., Wedekind, D., Sundarasetty, B. S., Lin, A. H., Kasahara, N., Brugman, M. H., Jirmo, A. C., Modlich, U., Gutzmer, R., et al. (2011) · Gene Therapy · doi:10.1038/gt.2011.15 · Cited by 21
Zhou, S., Ma, Z., Lu, T., Janke, L. J., Gray, J. T., & Sorrentino, B. P. (2013) · PLoS ONE · doi:10.1371/journal.pone.0062333 · Cited by 22
“This approach has been studied for the treatment of a variety of immunodeficiency disorders such as X-linked severe combined immunodeficiency (XSCID) [1,2], ADA-deficiency [3–6], Wiskott Aldrich Syndrome (WAS) [7], bthalassemia [8] and adrenoleukodystrophy [9] and has resulted in significant clinical benefit in these…”
BackgroundPerinatal systemic gene delivery using adeno-associated viral vectors
Karda, R., Buckley, S. M. K., Mattar, C. N. Z., Ng, J., Massaro, G., Hughes, M. P., Kurian, M. A., Baruteau, J., Gissen, P., Chan, J. K. Y., et al. (2014) · Frontiers in Molecular Neuroscience · doi:10.3389/fnmol.2014.00089 · Cited by 22
“The ascendancy of gene therapy through a number of recent successful gene therapy trials (Gaspar et al., 2004; Bainbridge et al., 2008; Cartier et al., 2009; Nathwani et al., 2011; Aiuti et al., 2013; Biffi et al., 2013) and the approval of the first gene therapy product to be sold in Europe…”
BackgroundFetal gene therapy: recent advances and current challenges
Mattar, C. N. Z., Choolani, M. A., Biswas, A., Waddington, S. N., & Chan, J. K. Y. (2011) · Expert Opinion on Biological Therapy · doi:10.1517/14712598.2011.585153 · Cited by 21
Gene, Stem Cell, and Future Therapies for Orphan Diseases
Phillips, M. I. (2012) · Clinical Pharmacology & Therapeutics · doi:10.1038/clpt.2012.82 · Cited by 20
Hayward, C., Patel, H. C., & Lyon, A. R. (2014) · Circulation Journal · doi:10.1253/circj.cj-14-1053 · Cited by 21
Redox Imbalance in Neurological Disorders in Adults and Children
Rey, F., Berardo, C., Maghraby, E., Mauri, A., Messa, L., Esposito, L., Casili, G., Ottolenghi, S., Bonaventura, E., Cuzzocrea, S., et al. (2023) · Antioxidants · doi:10.3390/antiox12040965 · Cited by 23
Gene therapy for β-thalassaemia: the continuing challenge
Yannaki, E., Emery, D. W., & Stamatoyannopoulos, G. (2010) · Expert Reviews in Molecular Medicine · doi:10.1017/s1462399410001626 · Cited by 21
Gene therapy using retrovirus vectors: vector development and biosafety at clinical trials
Knayo, D. O. I., & Takeuchi, Y. (2015) · Uirusu · doi:10.2222/jsv.65.27 · Cited by 22
Optimizing autologous cell grafts to improve stem cell gene therapy
Psatha, N., Karponi, G., & Yannaki, E. (2016) · Experimental Hematology · doi:10.1016/j.exphem.2016.04.007 · Cited by 21
Biogenesis and Function of Peroxisomes in Human Disease with a Focus on the ABC Transporter
Imanaka, T. (2019) · Biological and Pharmaceutical Bulletin · doi:10.1248/bpb.b18-00723 · Cited by 22
Pluripotent Stem Cell-Based Therapeutics for Muscular Dystrophies
Selvaraj, S., Kyba, M., & Perlingeiro, R. C. R. (2019) · Trends in Molecular Medicine · doi:10.1016/j.molmed.2019.07.004 · Cited by 23
Wada, M., Shimada, Y., Iizuka, S., Ishii, N., Hiraki, H., Tachibana, T., Maeda, K., Saito, M., Arakawa, S., Ishimoto, T., et al. (2020) · Molecular Therapy — Methods & Clinical Development · doi:10.1016/j.omtm.2020.09.012 · Cited by 21
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Gene Therapy for Hemoglobinopathies: Tremendous Successes and Remaining Caveats
Malik, P. (2016) · Molecular Therapy · doi:10.1038/mt.2016.57 · Cited by 20
Stem Cell Therapy for the Central Nervous System in Lysosomal Storage Diseases
Siddiqi, F., & Wolfe, J. H. N. (2016) · Human Gene Therapy · doi:10.1089/hum.2016.088 · Cited by 20
“Similarly, lentiviral vector correction of HSCs in the peroxisomal disorder adrenoleukodystrophy also improved the CNS in human patients.(111) Given the extensive history of little or variable effect on the CNS after bone marrow transplantation in many LSDs, the success of HSC treatment may be limited to certain disea…”
POLR3-Related Leukodystrophy: Exploring Potential Therapeutic Approaches
Perrier, S., Michell‐Robinson, M. A., & Bernard, G. (2021) · Frontiers in Cellular Neuroscience · doi:10.3389/fncel.2020.631802 · Cited by 23
The intersection of vector biology, gene therapy, and hemophilia
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Allogenic microglia replacement: A novel therapeutic strategy for neurological disorders
Rao, Y., & Peng, B. (2023) · Fundamental Research · doi:10.1016/j.fmre.2023.02.025 · Cited by 18
“Another stunning example showing the efficacy of the Mr BMT strategy in the treatment of human diseases is X-linked adrenoleukodystrophy (ALD), a kind of peroxisomal storage disease characterized by devastating demyelination and progressive myelopathy [171–174] .”
BackgroundThe pathology of X-linked adrenoleukodystrophy: Tissue specific changes as a clue to pathophysiology
Yska, H. A. F., Engelen, M., & Bugiani, M. (2024) · Orphanet Journal of Rare Diseases · doi:10.1186/s13023-024-03105-0 · Cited by 20
“More recently, gene therapy with a lentiviral vector that targeted CD34 + cells had similar effects on cerebral ALD progression, thus strengthening this hypothesis [62].”
BackgroundImmune modulation by genetic modification of dendritic cells with lentiviral vectors
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Metadata
- Authors
- Cartier, N., Hacein-Bey-Abina, S., Bartholomae, C. C., Veres, G., Schmidt, M., Kutschera, I., Vidaud, M., Abel, U., Dal-Cortivo, L., Caccavelli, L., Mahlaoui, N., Kiermer, V., Mittelstaedt, D., Bellesme, C., Lahlou, N., Lefrère, F., Blanche, S., Audit, M., Payen, E., Leboulch, P., l’Homme, B., Bougnères, P., Von Kalle, C., Fischer, A., Cavazzana-Calvo, M., & Aubourg, P.
- Affiliations
- Inserm (FR)
- Department of Biotherapy, Hôpital Necker-Enfants Malades, 75743 Paris, France.
- German Cancer Research Center (DE)
- 117 Southwest 72nd Place, Gainesville, FL 32608, USA.
- Hôpital Necker-Enfants Malades (FR)
- Nature Publishing Group, New York, NY 10013–1917, USA.
- 13687 Quinton Road, San Diego, CA 92129, USA.
- Assistance Publique – Hôpitaux de Paris (FR)
- Hôpital Saint-Vincent-de-Paul (FR)
- GenoSafe (FR)
- Brigham and Women's Hospital (US)
- Department of Pediatric Endocrinology and Neurology, Hôpital Saint-Vincent de Paul, Assistance Publique–Hôpitaux de Paris, 82 Avenue Denfert-Rochereau, 75674 Paris, France.
- National Center for Tumor Diseases and German Cancer Research Center (Deutsches Krebsforschungszentrum), 69120 Heidelberg, Germany.
- Corresponding author
- Cartier, Nathalie
- Aubourg, Patrick
- ORCID
- Venue
- Science
- Published
- 2009-11-06
- Volume
- 326
- Issue
- 5954
- Pages
- 818-823
- ISSN
- 0036-8075 (ISSN-L)
- 1095-9203
- OpenAlex
- W2141570423
- Cited by
- 1,508
- References
- 40
- Citations by year
- 2026: 25 · 2025: 43 · 2024: 54 · 2023: 50 · 2022: 62 · 2021: 56 · 2020: 71 · 2019: 61 · 2018: 76 · 2017: 96 · 2016: 98 · 2015: 107 · 2014: 130 · 2013: 137 · 2012: 139
- FWCI
- 84.34
- Type
- journal article
- Language
- en
- Open access
- green
- cea.hal.science
- Licence
- other-oa
- Locations
- Science (publishedVersion)
- PubMed (publishedVersion)
- HAL (submittedVersion, other-oa)
- Funders
- Deutsche Forschungsgemeinschaft (SPP 1230)
- Institut National de la Santé et de la Recherche Médicale
- Fondation de France
- Association Européenne contre les Leucodystrophies
- Assistance publique-Hôpitaux de Paris
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Health Sciences › Medicine › Genetics (clinical) › Lysosomal Storage Disorders Research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Clinical Biochemistry › Metabolism and Genetic Disorders
- Keywords
- X-linked adrenoleukodystrophy, hematopoietic stem cell gene therapy, lentiviral vectors, ABCD1 gene, myeloablative conditioning, demyelination
- MeSH terms
- ATP Binding Cassette Transporter, Subfamily D, Member 1, Adrenoleukodystrophy, Animals, Brain, Cell Differentiation, Child, Fatty Acids, Female, Genetic Vectors, Hematopoiesis, Hematopoietic Stem Cells, Humans, Leukocytes, Mononuclear, Male, Transduction, Genetic, Transplantation, Autologous, Genetic Therapy, HIV-1, Gene Expression, Virus Integration, Microglia, Hematopoietic Stem Cell Transplantation, Disease Progression, ATP-Binding Cassette Transporters, Cell Lineage, Transplantation Conditioning, Myeloablative Agonists, Mice
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