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

Modified sound-evoked brainstem potentials in Foxp2 mutant mice

(2009) Simone Kurt et al.   BRAIN RESEARCH

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

Improved Metabolic Correction in Patients with Lysosomal Storage Disease Treated with Hematopoietic Stem Cell Transplant Compared with Enzyme Replacement Therapy

(2009) Robert F. Wynn et al.   JOURNAL OF PEDIATRICS

Evaluation of heparin cofactor II–thrombin complex as a biomarker on blood spots from mucopolysaccharidosis I, IIIA and IIIB mice

(2009) Kia Langford-Smith et al.   MOLECULAR GENETICS AND METABOLISM

Reprogramming erythroid cells for lysosomal enzyme production leads to visceral and CNS cross-correction in mice with Hurler syndrome

(2009) D. Wang et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

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

(2009) N. Cartier et al.   SCIENCE

Laronidase (Aldurazyme®): enzyme replacement therapy for mucopolysaccharidosis type I

(2008) Gregory M Pastores   EXPERT OPINION ON BIOLOGICAL THERAPY

Neonatal Intravenous Injection of a Gammaretroviral Vector Has a Low Incidence of Tumor Induction in Mice

(2008) Mindy Tittiger et al.   HUMAN GENE THERAPY

Improved retroviral vector design results in sustained expression after adult gene therapy in mucopolysaccharidosis I mice

(2008) Ramin Sedaghat Herati et al.   JOURNAL OF GENE MEDICINE

Heparin cofactor II–thrombin complex: A biomarker of MPS disease

(2008) Derrick R. Randall et al.   MOLECULAR GENETICS AND METABOLISM

Early Neurodegeneration Progresses Independently of Microglial Activation by Heparan Sulfate in the Brain of Mucopolysaccharidosis IIIB Mice

(2008) Jérôme Ausseil et al.   PLoS One