ReviewMolecular therapy. Methods & clinical development2017
Genetic Engineering and Manufacturing of Hematopoietic Stem Cells.
Review in Molecular therapy. Methods & clinical development, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 14 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
14 citing papers in PubMed.
- Stem Cell for Cancer Immunotherapy: Current Approaches and Challenges.Stem cell reviews and reports · 2025Review
- Design and validation of a GMP stem cell manufacturing protocol for MPSII hematopoietic stem cell gene therapy.Molecular therapy. Methods & clinical development · 2024Article
- TGFCells · 2023Article
- Living Biomaterials to Engineer Hematopoietic Stem Cell Niches.Advanced healthcare materials · 2022Article
- Engineered Tissue Models to Replicate Dynamic Interactions within the Hematopoietic Stem Cell Niche.Advanced healthcare materials · 2022Review
- Implications of hematopoietic stem cells heterogeneity for gene therapies.Gene therapy · 2021Review
- Basic and Preclinical Research for Personalized Medicine.Journal of personalized medicine · 2021Review
- Effective, safe, and sustained correction of murine XLA using a UCOE-BTK promoter-based lentiviral vector.Molecular therapy. Methods & clinical development · 2021Article
- Gene Therapy for Primary Immunodeficiency.HemaSphere · 2021Review
- Vectofusin-1 Improves Transduction of Primary Human Cells with Diverse Retroviral and Lentiviral Pseudotypes, Enabling Robust, Automated Closed-System Manufacturing.Human gene therapy · 2019Article
- Re-educating immunity in respiratory allergies: the potential for hematopoietic stem cell-mediated gene therapy.Journal of molecular medicine (Berlin, Germany) · 2018Review
- TALEN-mediated functional correction of human iPSC-derived macrophages in context of hereditary pulmonary alveolar proteinosis.Scientific reports · 2017Article
- Perspectives on Manufacturing of High-Quality Cell Therapies.Molecular therapy : the journal of the American Society of Gene Therapy · 2017Article
- Showcasing Clinical Development and Production of Cellular Therapies.Molecular therapy : the journal of the American Society of Gene Therapy · 2017Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
2 authors.
Funding
Abstract
The marketing approval of genetically engineered hematopoietic stem cells (HSCs) as the first-line therapy for the treatment of severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID) is a tribute to the substantial progress that has been made regarding HSC engineering in the past decade. Reproducible manufacturing of high-quality, clinical-grade, genetically engineered HSCs is the foundation for broadening the application of this technology. Herein, the current state-of-the-art manufacturing platforms to genetically engineer HSCs as well as the challenges pertaining to production standardization and product characterization are addressed in the context of primary immunodeficiency diseases (PIDs) and other monogenic disorders.
Indexed as
Identifiers
What OpenQuestion holds
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.