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CRISPR/Cas9 for the treatment of haematological diseases: a journey from bacteria to the bedside

British journal of haematology, 2021-01, Vol.192 (1), p.33-49

2021

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Extending Gene Medicines to All in Need

The New England journal of medicine, 2024-05, Vol.390 (18), p.1721-1722

2024

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Clonal kinetics and single-cell transcriptional profiling of CAR-T cells in patients undergoing CD19 CAR-T immunotherapy

Nature communications, 2020-01, Vol.11 (1), p.219-13, Article 219

2020

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Targeted homology-directed repair in blood stem and progenitor cells with CRISPR nanoformulations

Nature materials, 2019-10, Vol.18 (10), p.1124-1132

2019

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Research priorities for an HIV cure: International AIDS Society Global Scientific Strategy 2021

Nature medicine, 2021-12, Vol.27 (12), p.2085-2098

2021

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Pyrimidoindole derivatives are agonists of human hematopoietic stem cell self-renewal

Science (American Association for the Advancement of Science), 2014-09, Vol.345 (6203), p.1509-1512

2014

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Engineering resistance to CD33-targeted immunotherapy in normal hematopoiesis by CRISPR/Cas9-deletion of CD33 exon 2

Leukemia, 2019-03, Vol.33 (3), p.762-808

2019

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Comparative analysis of risk factors for acute graft-versus-host disease and for chronic graft-versus-host disease according to National Institutes of Health consensus criteria

Blood, 2011-03, Vol.117 (11), p.3214-3219

2011

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Sorting Out the Best: Enriching Hematopoietic Stem Cells for Gene Therapy and Editing

Molecular therapy, 2018-10, Vol.26 (10), p.2328-2329

2018

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CD20 CAR T cells safely and reversibly ablate B cell follicles in a non-human primate model of HIV persistence

Molecular therapy, 2024-05, Vol.32 (5), p.1238-1251

2024

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In-Vivo Gene Therapy with Foamy Virus Vectors

Viruses, 2019-11, Vol.11 (12), p.1091

2019

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Long-term persistence and function of hematopoietic stem cell-derived chimeric antigen receptor T cells in a nonhuman primate model of HIV/AIDS

PLoS pathogens, 2017-12, Vol.13 (12), p.e1006753-e1006753

2017

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Next-Generation Conditioning for Bone Marrow Transplantation: Paving the Way for CAR-T Cell-Based Conditioning

Molecular therapy, 2018-05, Vol.26 (5), p.1167-1168

2018

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Stable HIV decoy receptor expression after in vivo HSC transduction in mice and NHPs: Safety and efficacy in protection from SHIV

Molecular therapy, 2023-04, Vol.31 (4), p.1059-1073

2023

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Vascular niche promotes hematopoietic multipotent progenitor formation from pluripotent stem cells

The Journal of clinical investigation, 2015-03, Vol.125 (3), p.1243-1254

2015

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Anti-PD-1 chimeric antigen receptor T cells efficiently target SIV-infected CD4+ T cells in germinal centers

The Journal of clinical investigation, 2024-04, Vol.134 (7), p.1-14

2024

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Long-Term Persistence of Anti-HIV Broadly Neutralizing Antibody-Secreting Hematopoietic Cells in Humanized Mice

Molecular therapy, 2019-01, Vol.27 (1), p.164-177

2019

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Rapamycin relieves lentiviral vector transduction resistance in human and mouse hematopoietic stem cells

Blood, 2014-08, Vol.124 (6), p.913-923

2014

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Long-term multilineage engraftment of autologous genome-edited hematopoietic stem cells in nonhuman primates

Blood, 2016-05, Vol.127 (20), p.2416-2426

2016

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Hepatic Cells Derived From Induced Pluripotent Stem Cells of Pigtail Macaques Support Hepatitis C Virus Infection

Gastroenterology (New York, N.Y. 1943), 2013-11, Vol.145 (5), p.966-969.e7

2013

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Thresholds for post-rebound SHIV control after CCR5 gene-edited autologous hematopoietic cell transplantation

eLife, 2021-01, Vol.10

2021

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The CD33 splice isoform lacking exon 2 as therapeutic target in human acute myeloid leukemia

Leukemia, 2020-09, Vol.34 (9), p.2479-2483

2020

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Differential impact of transplantation on peripheral and tissue-associated viral reservoirs: Implications for HIV gene therapy

PLoS pathogens, 2018-04, Vol.14 (4), p.e1006956

2018

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Targeting the membrane-proximal C2-set domain of CD33 for improved CD33-directed immunotherapy

Leukemia, 2021-09, Vol.35 (9), p.2496-2507

2021

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Semi-automated closed system manufacturing of lentivirus gene-modified haematopoietic stem cells for gene therapy

Nature communications, 2016-10, Vol.7 (1), p.13173-13173, Article 13173

2016

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CRISPR/Cas9 for the treatment of haematological diseases: a journey from bacteria to the bedside

Extending Gene Medicines to All in Need

Clonal kinetics and single-cell transcriptional profiling of CAR-T cells in patients undergoing CD19 CAR-T immunotherapy

Targeted homology-directed repair in blood stem and progenitor cells with CRISPR nanoformulations

Research priorities for an HIV cure: International AIDS Society Global Scientific Strategy 2021

Pyrimidoindole derivatives are agonists of human hematopoietic stem cell self-renewal

Engineering resistance to CD33-targeted immunotherapy in normal hematopoiesis by CRISPR/Cas9-deletion of CD33 exon 2

Comparative analysis of risk factors for acute graft-versus-host disease and for chronic graft-versus-host disease according to National Institutes of Health consensus criteria

Sorting Out the Best: Enriching Hematopoietic Stem Cells for Gene Therapy and Editing

CD20 CAR T cells safely and reversibly ablate B cell follicles in a non-human primate model of HIV persistence

In-Vivo Gene Therapy with Foamy Virus Vectors

Long-term persistence and function of hematopoietic stem cell-derived chimeric antigen receptor T cells in a nonhuman primate model of HIV/AIDS

Next-Generation Conditioning for Bone Marrow Transplantation: Paving the Way for CAR-T Cell-Based Conditioning

Stable HIV decoy receptor expression after in vivo HSC transduction in mice and NHPs: Safety and efficacy in protection from SHIV

Vascular niche promotes hematopoietic multipotent progenitor formation from pluripotent stem cells

Anti-PD-1 chimeric antigen receptor T cells efficiently target SIV-infected CD4+ T cells in germinal centers

Long-Term Persistence of Anti-HIV Broadly Neutralizing Antibody-Secreting Hematopoietic Cells in Humanized Mice

Rapamycin relieves lentiviral vector transduction resistance in human and mouse hematopoietic stem cells

Long-term multilineage engraftment of autologous genome-edited hematopoietic stem cells in nonhuman primates

Hepatic Cells Derived From Induced Pluripotent Stem Cells of Pigtail Macaques Support Hepatitis C Virus Infection

Thresholds for post-rebound SHIV control after CCR5 gene-edited autologous hematopoietic cell transplantation

The CD33 splice isoform lacking exon 2 as therapeutic target in human acute myeloid leukemia

Differential impact of transplantation on peripheral and tissue-associated viral reservoirs: Implications for HIV gene therapy

Targeting the membrane-proximal C2-set domain of CD33 for improved CD33-directed immunotherapy

Semi-automated closed system manufacturing of lentivirus gene-modified haematopoietic stem cells for gene therapy

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