ArticleHuman gene therapy methods2012
Transduction of human CD34+ repopulating cells with a self-inactivating lentiviral vector for SCID-X1 produced at clinical scale by a stable cell line.
Article in Human gene therapy methods, 2012. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03538899 (Phase I/II Safety and Efficacy Study of Gene Transfer for Artemis-Deficient Severe Combined Immunodeficiency), which is not on this map. Cited by 25 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Phase I/II Safety and Efficacy Study of Gene Transfer for Artemis-Deficient Severe Combined Immunodeficiency (ART-SCID) in Newly Diagnosed Patients Using Self-Inactivating Lentiviral Vector (AProArt) to Transduce Autologous CD34 Hematopoietic Cells
Who cites it
25 citing papers in PubMed, 45 citations in OpenAlex.
- Lentiviral hematopoietic stem cell gene therapy for X-linked severe combined immunodeficiency.Science translational medicine · 2016Trial
- Challenges in lentiviral vector production: retro-transduction of producer cell lines.Frontiers in bioengineering and biotechnology · 2025Article
- Quasi-perfusion studies for intensified lentiviral vector production using a continuous stable producer cell line.Molecular therapy. Methods & clinical development · 2024Article
- Development and clinical translation ofComputational and structural biotechnology journal · 2022Review
- Lentiviral Vector Bioprocessing.Viruses · 2021Review
- Lentiviral Vector Production from a Stable Packaging Cell Line Using a Packed Bed Bioreactor.Molecular therapy. Methods & clinical development · 2020Article
- Optimizing lentiviral vector transduction of hematopoietic stem cells for gene therapy.Gene therapy · 2020Article
- Production of Lentiviral Vectors Using Suspension Cells Grown in Serum-free Media.Molecular therapy. Methods & clinical development · 2020Article
- Benchmarking of Scale-X Bioreactor System in Lentiviral and Adenoviral Vector Production.Human gene therapy · 2020Article
- Immune Reconstitution After Gene Therapy Approaches in Patients With X-Linked Severe Combined Immunodeficiency Disease.Frontiers in immunology · 2020Review
- Preclinical Proof-of-Concept, Analytical Development, and Commercial Scale Production of Lentiviral Vector in Adherent Cells.Molecular therapy. Methods & clinical development · 2019Article
- Gene therapy targeting haematopoietic stem cells for inherited diseases: progress and challenges.Nature reviews. Drug discovery · 2019Review
- LentiPro26: novel stable cell lines for constitutive lentiviral vector production.Scientific reports · 2018Article
- Function and Safety of Lentivirus-Mediated Gene Transfer for CSF2RA-Deficiency.Human gene therapy methods · 2017Article
- Scalable Lentiviral Vector Production Using Stable HEK293SF Producer Cell Lines.Human gene therapy methods · 2017Article
- Development of Third-generation Cocal Envelope Producer Cell Lines for Robust Lentiviral Gene Transfer into Hematopoietic Stem Cells and T-cells.Molecular therapy : the journal of the American Society of Gene Therapy · 2016Article
- Progress and challenges in viral vector manufacturing.Human molecular genetics · 2016Review
- Production of lentiviral vectors.Molecular therapy. Methods & clinical development · 2016Review
- RD-MolPack technology for the constitutive production of self-inactivating lentiviral vectors pseudotyped with the nontoxic RD114-TR envelope.Molecular therapy. Methods & clinical development · 2016Article
- Construction of stable packaging cell lines for clinical lentiviral vector production.Scientific reports · 2015Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
7 authors at 1 institution in 1 country.
Funding
Abstract
Self-inactivating (SIN)-lentiviral vectors have safety and efficacy features that are well suited for transduction of hematopoietic stem cells (HSCs), but generation of vector at clinical scale has been challenging. Approximately 280 liters of an X-Linked Severe Combined Immunodeficiency Disorder (SCID-X1) SIN-lentiviral vector in two productions from a stable cell line were concentrated to final titers of 4.5 and 7.2×10(8) tu/ml. These two clinical preparations and three additional development-scale preparations were evaluated in human CD34(+) hematopoietic cells in vitro using colony forming cell (CFU-C) assay and in vivo using the NOD/Lt-scid/IL2Rγ(null) (NSG) mouse xenotransplant model. A 40-hour transduction protocol using a single vector exposure conferred a mean NSG repopulating cell transduction of 0.23 vector genomes/human genome with a mean myeloid vector copy number of 3.2 vector genomes/human genome. No adverse effects on engraftment were observed from vector treatment. Direct comparison between our SIN-lentiviral vector using a 40-hour protocol and an MFGγ(c) γ-retroviral vector using a five-day protocol demonstrated equivalent NSG repopulating cell transduction efficiency. Clonality survey by linear amplification-mediated polymerase chain reaction (LAM-PCR) with Illumina sequencing revealed common clones in sorted myeloid and lymphoid populations from engrafted mice demonstrating multipotent cell transduction. These vector preparations will be used in two clinical trials for SCID-X1.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.