Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy

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

(2013) A. Aiuti et al.   SCIENCE

Brain conditioning is instrumental for successful microglia reconstitution following hematopoietic stem cell transplantation

(2012) A. Capotondo et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Lentiviral vector common integration sites in preclinical models and a clinical trial reflect a benign integration bias and not oncogenic selection

(2011) A. Biffi et al.   BLOOD

Gene therapy for leukodystrophies

(2011) A. Biffi et al.   HUMAN MOLECULAR GENETICS

Metachromatic leukodystrophy: natural course of cerebral MRI changes in relation to clinical course

(2011) Samuel Groeschel et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Ex vivo gene transfer and correction for cell-based therapies

(2011) Luigi Naldini   NATURE REVIEWS GENETICS

Dynamics of gene-modified progenitor cells analyzed by tracking retroviral integration sites in a human SCID-X1 gene therapy trial

(2010) G. P. Wang et al.   BLOOD

Evidence for the cure of HIV infection by CCR5 32/ 32 stem cell transplantation

(2010) K. Allers et al.   BLOOD

Comparison of human cord blood engraftment between immunocompromised mouse strains

(2010) S. P. McDermott et al.   BLOOD

Gene therapy augments the efficacy of hematopoietic cell transplantation and fully corrects mucopolysaccharidosis type I phenotype in the mouse model

(2010) I. Visigalli et al.   BLOOD

An unusual form of metachromatic leukodystrophy in three siblings

(2010) S. Yatziv et al.   CLINICAL GENETICS

THE GROSS MOTOR FUNCTION MEASURE: A MEANS TO EVALUATE THE EFFECTS OF PHYSICAL THERAPY

(2010) Dianne J. Russell et al.   DEVELOPMENTAL MEDICINE AND CHILD NEUROLOGY

Development and reliability of a classification system for gross motor function in children with metachromatic leucodystrophy

(2010) CHRISTIANE KEHRER et al.   DEVELOPMENTAL MEDICINE AND CHILD NEUROLOGY

Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia

(2010) Marina Cavazzana-Calvo et al.   NATURE

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

Stem-Cell Gene Therapy for the Wiskott–Aldrich Syndrome

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

Current International Perspectives on Hematopoietic Stem Cell Transplantation for Inherited Metabolic Disorders

(2010) Jaap J. Boelens et al.   PEDIATRIC CLINICS OF NORTH AMERICA

Fate Mapping Analysis Reveals That Adult Microglia Derive from Primitive Macrophages

(2010) F. Ginhoux et al.   SCIENCE

Identification of Hematopoietic Stem Cell-Specific miRNAs Enables Gene Therapy of Globoid Cell Leukodystrophy

(2010) B. Gentner et al.   Science Translational Medicine

Metachromatic leukodystrophy - mutation analysis provides further evidence of genotype-phenotype correlation

(2009) A Biffi et al.   CLINICAL GENETICS

The genotoxic potential of retroviral vectors is strongly modulated by vector design and integration site selection in a mouse model of HSC gene therapy

(2009) Eugenio Montini et al.   JOURNAL OF CLINICAL INVESTIGATION

Localized donor cells in brain of a Hunter disease patient after cord blood stem cell transplantation

(2009) Ken Araya et al.   MOLECULAR GENETICS AND METABOLISM

Gene Therapy for Immunodeficiency Due to Adenosine Deaminase Deficiency

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

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

(2009) N. Cartier et al.   SCIENCE

Metachromatic leukodystrophy: an overview of current and prospective treatments

(2008) A Biffi et al.   BONE MARROW TRANSPLANTATION

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

Physiological Promoters Reduce the Genotoxic Risk of Integrating Gene Vectors

(2008) Daniela Zychlinski et al.   MOLECULAR THERAPY