Peripheral blood stem and progenitor cell collection in pediatric candidates for ex vivo gene therapy: a 10-year series

Modeling, optimization, and comparable efficacy of T cell and hematopoietic stem cell gene editing for treating hyper‐IgM syndrome

(2021) Valentina Vavassori et al.   EMBO Molecular Medicine

Extensive Metabolic Correction of Hurler Disease By Hematopoietic Stem Cell-Based Gene Therapy: Preliminary Results from a Phase I/II Trial

(2020) Bernhard Gentner et al.   BLOOD

Plerixafor combined with standard regimens for hematopoietic stem cell mobilization in pediatric patients with solid tumors eligible for autologous transplants: two-arm phase I/II study (MOZAIC)

(2020) Bruce Morland et al.   BONE MARROW TRANSPLANTATION

Safety and Feasibility of Hematopoietic Progenitor Stem Cell Collection by Mobilization with Plerixafor Followed by Apheresis vs Bone Marrow Harvest in Patients with Sickle Cell Disease in the Multi‐center HGB ‐206 Trial

(2020) John F. Tisdale et al.   AMERICAN JOURNAL OF HEMATOLOGY

EMA Recommendation for the Pediatric Indications of Plerixafor (Mozobil) to Enhance Mobilization of Hematopoietic Stem Cells for Collection and Subsequent Autologous Transplantation in Children with Lymphoma or Malignant Solid Tumors

(2020) Dominik Karres et al.   ONCOLOGIST

Variable CD34+ recovery of cryopreserved allogeneic HPC products: transplant implications during the COVID-19 pandemic

(2020) Duncan Purtill et al.   Blood Advances

Gene therapy using haematopoietic stem and progenitor cells

(2020) Giuliana Ferrari et al.   NATURE REVIEWS GENETICS

Post-Transcriptional Genetic Silencing of BCL11A to Treat Sickle Cell Disease

(2020) Erica B. Esrick et al.   NEW ENGLAND JOURNAL OF MEDICINE

Intrabone hematopoietic stem cell gene therapy for adult and pediatric patients affected by transfusion-dependent ß-thalassemia

(2019) Sarah Marktel et al.   NATURE MEDICINE

Mobilized peripheral blood stem cell apheresis via Hickman catheter in pediatric patients

(2019) Nora Doberschuetz et al.   TRANSFUSION

Lentiviral haemopoietic stem/progenitor cell gene therapy for treatment of Wiskott-Aldrich syndrome: interim results of a non-randomised, open-label, phase 1/2 clinical study

(2019) Francesca Ferrua et al.   Lancet Haematology

Bone marrow harvesting from paediatric patients undergoing haematopoietic stem cell gene therapy

(2019) Francesca Tucci et al.   BONE MARROW TRANSPLANTATION

Lentiviral Hematopoietic Stem and Progenitor Cell Gene Therapy for Wiskott-Aldrich Syndrome (WAS): Up to 8 Years of Follow up in 17 Subjects Treated Since 2010

(2019) Francesca Ferrua et al.   BLOOD

Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia

(2018) Alexis A. Thompson et al.   NEW ENGLAND JOURNAL OF MEDICINE

Young child as a donor of cells for transplantation and lymphocyte based therapies

(2018) Jan Styczynski   TRANSFUSION AND APHERESIS SCIENCE

Peripheral blood progenitor cell collection in pediatric patients optimized by high pre-apheresis count of circulating CD34+ cells and high blood flow

(2018) Axel Karow et al.   BONE MARROW TRANSPLANTATION

Marker-free coselection for CRISPR-driven genome editing in human cells

(2017) Daniel Agudelo et al.   NATURE METHODS

Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy

(2017) Florian Eichler et al.   NEW ENGLAND JOURNAL OF MEDICINE

Efficient Ex Vivo Engineering and Expansion of Highly Purified Human Hematopoietic Stem and Progenitor Cell Populations for Gene Therapy

(2017) Erika Zonari et al.   Stem Cell Reports

Update on the safety and efficacy of retroviral gene therapy for immunodeficiency due to adenosine deaminase deficiency

(2016) Maria Pia Cicalese et al.   BLOOD

Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: an ad-hoc analysis of a non-randomised, open-label, phase 1/2 trial

(2016) Maria Sessa et al.   LANCET

CRISPR/Cas9 β-globin gene targeting in human haematopoietic stem cells

(2016) Daniel P. Dever et al.   NATURE

Abnormalities of acid–base balance and predisposition to metabolic acidosis in Metachromatic Leukodystrophy patients

(2015) L. Lorioli et al.   MOLECULAR GENETICS AND METABOLISM

Peripheral Blood Progenitor Cell Mobilization for Autologous and Allogeneic Hematopoietic Cell Transplantation: Guidelines from the American Society for Blood and Marrow Transplantation

(2014) Hien K. Duong et al.   BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION

Lower risk for serious adverse events and no increased risk for cancer after PBSC vs BM donation

(2014) M. A. Pulsipher et al.   BLOOD

Defining “poor mobilizer” in pediatric patients who need an autologous peripheral blood progenitor cell transplantation

(2013) Julián Sevilla et al.   CYTOTHERAPY

Anticoagulation techniques in apheresis: From heparin to citrate and beyond

(2012) Grace Lee et al.   JOURNAL OF CLINICAL APHERESIS

Risk of complications during hematopoietic stem cell collection in pediatric sibling donors: a prospective European Group for Blood and Marrow Transplantation Pediatric Diseases Working Party study

(2011) J. Styczynski et al.   BLOOD

Mobilization of hematopoietic stem and progenitor cells using inhibitors of CXCR4 and VLA-4

(2011) M P Rettig et al.   LEUKEMIA

Hematopoietic Stem Cell Gene Therapy for Adenosine Deaminase-Deficient Severe Combined Immunodeficiency Leads to Long-Term Immunological Recovery and Metabolic Correction

(2011) H. B. Gaspar et al.   Science Translational Medicine

Infusion of suicide-gene-engineered donor lymphocytes after family haploidentical haemopoietic stem-cell transplantation for leukaemia (the TK007 trial): a non-randomised phase I–II study

(2009) Fabio Ciceri et al.   LANCET ONCOLOGY