Gene Therapy for Wiskott-Aldrich Syndrome--Long-Term Efficacy and Genotoxicity

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

(2013) A. Aiuti et al.   SCIENCE

The BET Family of Proteins Targets Moloney Murine Leukemia Virus Integration near Transcription Start Sites

(2013) Jan De Rijck et al.   Cell Reports

Late MRD response determines relapse risk overall and in subsets of childhood T-cell ALL: results of the AIEOP-BFM-ALL 2000 study

(2011) M. Schrappe et al.   BLOOD

Long-term outcome and lineage-specific chimerism in 194 patients with Wiskott-Aldrich syndrome treated by hematopoietic cell transplantation in the period 1980-2009: an international collaborative study

(2011) D. Moratto et al.   BLOOD

The role of plerixafor in optimizing peripheral blood stem cell mobilization for autologous stem cell transplantation

(2011) M Mohty et al.   LEUKEMIA

Insertion Sites in Engrafted Cells Cluster Within a Limited Repertoire of Genomic Areas After Gammaretroviral Vector Gene Therapy

(2011) Annette Deichmann et al.   MOLECULAR THERAPY

State-of-the-art gene-based therapies: the road ahead

(2011) Mark A. Kay   NATURE REVIEWS GENETICS

20 years of gene therapy for SCID

(2010) Alain Fischer et al.   NATURE IMMUNOLOGY

Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease

(2010) Stefan Stein et al.   NATURE MEDICINE

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

(2010) Anna Paruzynski et al.   Nature Protocols

Efficacy of Gene Therapy for X-Linked Severe Combined Immunodeficiency

(2010) Salima Hacein-Bey-Abina et al.   NEW ENGLAND JOURNAL OF MEDICINE

Stem-Cell Gene Therapy for the Wiskott–Aldrich Syndrome

(2010) Kaan Boztug et al.   NEW ENGLAND JOURNAL OF MEDICINE

Recent advances in understanding the pathophysiology of Wiskott-Aldrich syndrome

(2009) M. Bosticardo et al.   BLOOD

Multiple independent second-site mutations in two siblings with somatic mosaicism for Wiskott-Aldrich syndrome

(2009) K Boztug et al.   CLINICAL GENETICS

Wiskott-Aldrich syndrome

(2009) Luigi D Notarangelo et al.   CURRENT OPINION IN HEMATOLOGY

Wiskott–Aldrich Syndrome: Immunodeficiency resulting from defective cell migration and impaired immunostimulatory activation

(2009) Gerben Bouma et al.   IMMUNOBIOLOGY

Multiple mechanisms induce ectopic expression of LYL1 in subsets of T-ALL cell lines

(2009) Stefan Nagel et al.   LEUKEMIA RESEARCH

Gene Therapy for Immunodeficiency Due to Adenosine Deaminase Deficiency

(2009) Alessandro Aiuti et al.   NEW ENGLAND JOURNAL OF MEDICINE

Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients

(2008) Steven J. Howe et al.   JOURNAL OF CLINICAL INVESTIGATION

Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1

(2008) Salima Hacein-Bey-Abina et al.   JOURNAL OF CLINICAL INVESTIGATION