ArticleMolecular therapy : the journal of the American Society of Gene Therapy2019
Busulfan Combined with Immunosuppression Allows Efficient Engraftment of Gene-Modified Cells in a Rhesus Macaque Model.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 29 papers.
What it found
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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.
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Who cites it
29 citing papers in PubMed.
- Clonal dynamics, tolerance, and adverse events after CD45-ADC-conditioned autologous HSPC transplantation in macaques.Blood advances · 2026Article
- Article
- Engraftment of gene-edited hematopoietic stem cells after antibody-drug conjugate conditioning in nonhuman primates.Blood advances · 2026Article
- Hematopoietic stem cell therapy with gene modification to treat sickle cell disease.Stem cells translational medicine · 2025Review
- Engraftment and persistence ofScience translational medicine · 2025Article
- Current and future treatments for sickle cell disease: From hematopoietic stem cell transplantation to in vivo gene therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Review
- Stem cell therapies in the clinic.Bioengineering & translational medicine · 2025Review
- Lentiviral Vector-MediatedHuman gene therapy · 2024Article
- Fertility-preserving myeloablative conditioning using single-dose CD117 antibody-drug conjugate in a rhesus gene therapy model.Nature communications · 2023Article
- Efficient manufacturing and engraftment ofMolecular therapy. Methods & clinical development · 2023Article
- Enhancing mesenchymal stem cell survival and homing capability to improve cell engraftment efficacy for liver diseases.Stem cell research & therapy · 2023Review
- Pre-existing immunity does not impair the engraftment of CRISPR-Cas9-edited cells in rhesus macaques conditioned with busulfan or radiation.Molecular therapy. Methods & clinical development · 2023Article
- Comparison of busulfan and total body irradiation conditioning on hematopoietic clonal dynamics following lentiviral gene transfer in rhesus macaques.Molecular therapy. Methods & clinical development · 2023Article
- The unknown impact of conditioning on HSC engraftment and clonal dynamics.Molecular therapy. Methods & clinical development · 2023Article
- Models and Methods for Evaluating Regeneration of Spermatogenesis After Cytotoxic Treatments.Methods in molecular biology (Clifton, N.J.) · 2023Article
- IGF2-tagging of GAA promotes full correction of murine Pompe disease at a clinically relevant dosage of lentiviral gene therapy.Molecular therapy. Methods & clinical development · 2022Article
- Immunological barriers to haematopoietic stem cell gene therapy.Nature reviews. Immunology · 2022Review
- Improved engraftment and therapeutic efficacy by human genome-edited hematopoietic stem cells with Busulfan-based myeloablation.Molecular therapy. Methods & clinical development · 2022Article
- Lentiviral gene therapy prevents anti-human acid α-glucosidase antibody formation in murine Pompe disease.Molecular therapy. Methods & clinical development · 2022Article
- Review
Corrections and comments
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Authors and funding
13 authors.
Funding
Abstract
Busulfan conditioning is utilized for hematopoietic stem cell (HSC) depletion in the context of HSC gene-therapy conditioning but may result in insufficient immunosuppression. In this study, we evaluated whether additional immunosuppression is required for efficient engraftment of gene-modified cells using a rhesus HSC lentiviral gene-therapy model. We transduced half of rhesus CD34
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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.