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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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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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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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Lessons Learned from Two Decades of Clinical Trial Experience in Gene Therapy for Fanconi Anemia

Current gene therapy, 2016-10, Vol.16 (5), p.338-348

2016

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Make gene therapies more available by manufacturing them in lower-income nations

Nature (London), 2024-07, Vol.631 (8021), p.502-504

2024

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Hematopoietic Stem Cell Approaches to Cancer

Hematology/oncology clinics of North America, 2017-10, Vol.31 (5), p.897-912

2017

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Novel lineage depletion preserves autologous blood stem cells for gene therapy of Fanconi anemia complementation group A

Haematologica (Roma), 2018-11, Vol.103 (11), p.1806-1814

2018

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Purification of Human CD34+CD90+ HSCs Reduces Target Cell Population and Improves Lentiviral Transduction for Gene Therapy

Molecular therapy. Methods & clinical development, 2020-09, Vol.18, p.679-691

2020

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Efficient generation, purification, and expansion of CD34+ hematopoietic progenitor cells from nonhuman primate–induced pluripotent stem cells

Blood, 2012-09, Vol.120 (13), p.e35-e44

2012

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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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Role of high mobility group (HMG) chromatin proteins in DNA repair

DNA repair, 2005-07, Vol.4 (8), p.926-938

2005

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DNA Barcoding in Nonhuman Primates Reveals Important Limitations in Retrovirus Integration Site Analysis

Molecular therapy. Methods & clinical development, 2020-06, Vol.17, p.796-809

2020

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Envelope-Specific Adaptive Immunity following Transplantation of Hematopoietic Stem Cells Modified with VSV-G Lentivirus

Molecular therapy. Methods & clinical development, 2020-12, Vol.19, p.438-446

2020

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UM171 Enhances Lentiviral Gene Transfer and Recovery of Primitive Human Hematopoietic Cells

Molecular therapy. Methods & clinical development, 2018-09, Vol.10, p.156-164

2018

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Autologous, Gene-Modified Hematopoietic Stem and Progenitor Cells Repopulate the Central Nervous System with Distinct Clonal Variants

Stem cell reports, 2019-07, Vol.13 (1), p.91-104

2019

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Effective, safe, and sustained correction of murine XLA using a UCOE-BTK promoter-based lentiviral vector

Molecular therapy. Methods & clinical development, 2021-03, Vol.20, p.635-651

2021

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Decentralized manufacturing of anti CD19 CAR-T cells using CliniMACS Prodigy®: real-world experience and cost analysis in India

Bone marrow transplantation (Basingstoke), 2023-02, Vol.58 (2), p.160-167

2023

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Multilineage polyclonal engraftment of Cal-1 gene-modified cells and in vivo selection after SHIV infection in a nonhuman primate model of AIDS

Molecular therapy. Methods & clinical development, 2016-01, Vol.3 (C), p.16007-16007, Article 16007

2016

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Therapeutically relevant engraftment of a CRISPR-Cas9–edited HSC-enriched population with HbF reactivation in nonhuman primates

Science translational medicine, 2019-07, Vol.11 (503)

2019

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Women Lead Gene Therapy Science in 2021

Human gene therapy, 2021-04, Vol.32 (7-8), p.317-318

2021

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Rapid immune reconstitution of SCID-X1 canines after G-CSF/AMD3100 mobilization and in vivo gene therapy

Blood advances, 2018-05, Vol.2 (9), p.987-999

2018

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A distinct hematopoietic stem cell population for rapid multilineage engraftment in nonhuman primates

Science translational medicine, 2017-11, Vol.9 (414)

2017

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An Interview with Michel Sadelain, MD, PhD

Human gene therapy, 2018-05, Vol.29 (5), p.530-533

Adair, Jennifer E

2018

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In Vivo Murine-Matured Human CD3 + Cells as a Preclinical Model for T Cell-Based Immunotherapies

Molecular therapy. Methods & clinical development, 2017-09, Vol.6 (C), p.17-30

2017

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Black Lives Matter to Gene Therapy

Human gene therapy, 2020-07, Vol.31 (13-14), p.700-700

2020

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

Vascular niche promotes hematopoietic multipotent progenitor formation from pluripotent stem cells

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

Lessons Learned from Two Decades of Clinical Trial Experience in Gene Therapy for Fanconi Anemia

Make gene therapies more available by manufacturing them in lower-income nations

Hematopoietic Stem Cell Approaches to Cancer

Novel lineage depletion preserves autologous blood stem cells for gene therapy of Fanconi anemia complementation group A

Purification of Human CD34+CD90+ HSCs Reduces Target Cell Population and Improves Lentiviral Transduction for Gene Therapy

Efficient generation, purification, and expansion of CD34+ hematopoietic progenitor cells from nonhuman primate–induced pluripotent stem cells

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

Role of high mobility group (HMG) chromatin proteins in DNA repair

DNA Barcoding in Nonhuman Primates Reveals Important Limitations in Retrovirus Integration Site Analysis

Envelope-Specific Adaptive Immunity following Transplantation of Hematopoietic Stem Cells Modified with VSV-G Lentivirus

UM171 Enhances Lentiviral Gene Transfer and Recovery of Primitive Human Hematopoietic Cells

Autologous, Gene-Modified Hematopoietic Stem and Progenitor Cells Repopulate the Central Nervous System with Distinct Clonal Variants

Effective, safe, and sustained correction of murine XLA using a UCOE-BTK promoter-based lentiviral vector

Decentralized manufacturing of anti CD19 CAR-T cells using CliniMACS Prodigy®: real-world experience and cost analysis in India

Multilineage polyclonal engraftment of Cal-1 gene-modified cells and in vivo selection after SHIV infection in a nonhuman primate model of AIDS

Therapeutically relevant engraftment of a CRISPR-Cas9–edited HSC-enriched population with HbF reactivation in nonhuman primates

Women Lead Gene Therapy Science in 2021

Rapid immune reconstitution of SCID-X1 canines after G-CSF/AMD3100 mobilization and in vivo gene therapy

A distinct hematopoietic stem cell population for rapid multilineage engraftment in nonhuman primates

An Interview with Michel Sadelain, MD, PhD

In Vivo Murine-Matured Human CD3 + Cells as a Preclinical Model for T Cell-Based Immunotherapies

Black Lives Matter to Gene Therapy

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