Successful engraftment of gene-corrected hematopoietic stem cells in non-conditioned patients with Fanconi anemia

Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia

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Cancer in the National Cancer Institute inherited bone marrow failure syndrome cohort after fifteen years of follow-up

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Biallelic mutations in the ubiquitin ligase RFWD3 cause Fanconi anemia

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Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy

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Cancer in the National Cancer Institute inherited bone marrow failure syndrome cohort after fifteen years of follow-up

(2017) Blanche P. Alter et al.   HAEMATOLOGICA

GENE-IS: Time-Efficient and Accurate Analysis of Viral Integration Events in Large-Scale Gene Therapy Data

(2017) Saira Afzal et al.   Molecular Therapy-Nucleic Acids

Lentiviral-Mediated Gene Therapy in Fanconi Anemia-A Mice Reveals Long-Term Engraftment and Continuous Turnover of Corrected HSCs

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UpSet: Visualization of Intersecting Sets

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Lentiviral Hematopoietic Stem Cell Gene Therapy in Patients with Wiskott-Aldrich Syndrome

(2013) A. Aiuti et al.   SCIENCE

Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy

(2013) A. Biffi et al.   SCIENCE

Bone Marrow Failure in Fanconi Anemia Is Triggered by an Exacerbated p53/p21 DNA Damage Response that Impairs Hematopoietic Stem and Progenitor Cells

(2012) Raphael Ceccaldi et al.   Cell Stem Cell

Myelodysplasia and leukemia of Fanconi anemia are associated with a specific pattern of genomic abnormalities that includes cryptic RUNX1/AML1 lesions

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Chromosome fragility in patients with Fanconi anaemia: diagnostic implications and clinical impact

(2011) M. Castella et al.   JOURNAL OF MEDICAL GENETICS

Quantification of lentiviral vector copy numbers in individual hematopoietic colony-forming cells shows vector dose-dependent effects on the frequency and level of transduction

(2010) S Charrier et al.   GENE THERAPY

Genome-wide high-throughput integrome analyses by nrLAM-PCR and next-generation sequencing

(2010) Anna Paruzynski et al.   Nature Protocols

Development of Lentiviral Vectors with Optimized Transcriptional Activity for the Gene Therapy of Patients with Fanconi Anemia

(2009) África González-Murillo et al.   HUMAN GENE THERAPY

Comprehensive genomic access to vector integration in clinical gene therapy

(2009) Richard Gabriel et al.   NATURE MEDICINE

Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy

(2009) N. Cartier et al.   SCIENCE

Head and neck squamous cell carcinoma in 13 patients with Fanconi anemia after hematopoietic stem cell transplantation

(2008) Caroline Masserot et al.   CANCER

Rapid Lentiviral Transduction Preserves the Engraftment Potential of Fanca−/− Hematopoietic Stem Cells

(2008) Lars UW Müller et al.   MOLECULAR THERAPY