Tolerance induction and microglial engraftment after fetal therapy without conditioning in mice with Mucopolysaccharidosis type VII

Association between mucopolysaccharidosis Type VII and hydrops fetalis

(2019) Max Holtz et al.   ULTRASOUND IN OBSTETRICS & GYNECOLOGY

Mapping the Mouse Cell Atlas by Microwell-Seq

(2018) Xiaoping Han et al.   CELL

Two distinct ontogenies confer heterogeneity to mouse brain microglia

(2018) Shrutokirti De et al.   DEVELOPMENT

A novel Blind Start study design to investigate vestronidase alfa for mucopolysaccharidosis VII, an ultra-rare genetic disease

(2018) Paul Harmatz et al.   MOLECULAR GENETICS AND METABOLISM

Microglial Ramification, Surveillance, and Interleukin-1β Release Are Regulated by the Two-Pore Domain K + Channel THIK-1

(2018) Christian Madry et al.   NEURON

Embryonic Microglia Derive from Primitive Macrophages and Are Replaced by cmyb -Dependent Definitive Microglia in Zebrafish

(2018) Giuliano Ferrero et al.   Cell Reports

Pharmacokinetic and Pharmacodynamic Modeling to Optimize the Dose of Vestronidase Alfa, an Enzyme Replacement Therapy for Treatment of Patients with Mucopolysaccharidosis Type VII: Results from Three Trials

(2018) Yulan Qi et al.   CLINICAL PHARMACOKINETICS

Engrafted parenchymal brain macrophages differ from microglia in transcriptome, chromatin landscape and response to challenge

(2018) Anat Shemer et al.   Nature Communications

Siglec-H is a microglia-specific marker that discriminates microglia from CNS-associated macrophages and CNS-infiltrating monocytes

(2017) Hiroyuki Konishi et al.   GLIA

Hematopoietic Stem Cell Gene Therapy for Storage Disease: Current and New Indications

(2017) Alessandra Biffi   MOLECULAR THERAPY

Emptying the stores: lysosomal diseases and therapeutic strategies

(2017) Frances M. Platt   NATURE REVIEWS DRUG DISCOVERY

Characterizing microglia activation: a spatial statistics approach to maximize information extraction

(2017) Benjamin M. Davis et al.   Scientific Reports

Haematopoietic stem cell transplantation in inborn errors of metabolism

(2016) Robert Chiesa et al.   CURRENT OPINION IN HEMATOLOGY

Clinical course of sly syndrome (mucopolysaccharidosis type VII)

(2016) Adriana M Montaño et al.   JOURNAL OF MEDICAL GENETICS

Sall1 is a transcriptional regulator defining microglia identity and function

(2016) Anne Buttgereit et al.   NATURE IMMUNOLOGY

New tools for studying microglia in the mouse and human CNS

(2016) Mariko L. Bennett et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

In utero stem cell transplantation and gene therapy: rationale, history, and recent advances toward clinical application

(2016) Graça Almeida-Porada et al.   Molecular Therapy-Methods & Clinical Development

Lysosomal storage disease as an etiology of nonimmune hydrops

(2015) Alexis C. Gimovsky et al.   AMERICAN JOURNAL OF OBSTETRICS AND GYNECOLOGY

Correction of murine hemoglobinopathies by prenatal tolerance induction and postnatal nonmyeloablative allogeneic BM transplants

(2015) W. H. Peranteau et al.   BLOOD

C-Myb+ Erythro-Myeloid Progenitor-Derived Fetal Monocytes Give Rise to Adult Tissue-Resident Macrophages

(2015) Guillaume Hoeffel et al.   IMMUNITY

The many faces of hydrops

(2015) S. Christopher Derderian et al.   JOURNAL OF PEDIATRIC SURGERY

β2-microglobulin is a systemic pro-aging factor that impairs cognitive function and neurogenesis

(2015) Lucas K Smith et al.   NATURE MEDICINE

Stable long-term mixed chimerism achieved in a canine model of allogeneic in utero hematopoietic cell transplantation

(2014) J. D. Vrecenak et al.   BLOOD

Intrauterine Blood Transfusion: Current Indications and Associated Risks

(2014) Irene T.M. Lindenburg et al.   FETAL DIAGNOSIS AND THERAPY

An RNA-Sequencing Transcriptome and Splicing Database of Glia, Neurons, and Vascular Cells of the Cerebral Cortex

(2014) Y. Zhang et al.   JOURNAL OF NEUROSCIENCE

Neurodegeneration by Activation of the Microglial Complement-Phagosome Pathway

(2014) L.-G. Bodea et al.   JOURNAL OF NEUROSCIENCE

Mfsd2a is critical for the formation and function of the blood–brain barrier

(2014) Ayal Ben-Zvi et al.   NATURE

IgG Subclasses and Allotypes: From Structure to Effector Functions

(2014) Gestur Vidarsson et al.   Frontiers in Immunology

Direct and indirect antigen presentation lead to deletion of donor-specific T cells after in utero hematopoietic cell transplantation in mice

(2013) A. Nijagal et al.   BLOOD

Busulfan Conditioning Enhances Engraftment of Hematopoietic Donor-derived Cells in the Brain Compared With Irradiation

(2013) Fiona L Wilkinson et al.   MOLECULAR THERAPY

Immune response to intrathecal enzyme replacement therapy in mucopolysaccharidosis I patients

(2013) Moin Vera et al.   PEDIATRIC RESEARCH

STAR: ultrafast universal RNA-seq aligner

(2012) Alexander Dobin et al.   BIOINFORMATICS

Rodent motor and neuropsychological behaviour measured in home cages using the integrated modular platform SmartCage™

(2012) Taline V Khroyan et al.   CLINICAL AND EXPERIMENTAL PHARMACOLOGY AND PHYSIOLOGY

Lysosomal storage disorder in non-immunological hydrops fetalis (NIHF) - more common than assumed? Report of four cases with transient NIHF and a review of the literature

(2012) Catharina Whybra et al.   Orphanet Journal of Rare Diseases

Maternal T cells limit engraftment after in utero hematopoietic cell transplantation in mice

(2011) Amar Nijagal et al.   JOURNAL OF CLINICAL INVESTIGATION

Fate Mapping Analysis Reveals That Adult Microglia Derive from Primitive Macrophages

(2010) F. Ginhoux et al.   SCIENCE

Automated home-cage behavioural phenotyping of mice

(2010) Hueihan Jhuang et al.   Nature Communications

Systematic and integrative analysis of large gene lists using DAVID bioinformatics resources

(2009) Da Wei Huang et al.   Nature Protocols

Involvement of the Toll-like receptor 4 pathway and use of TNF-  antagonists for treatment of the mucopolysaccharidoses

(2009) C. M. Simonaro et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Identification of new markers for neurodegeneration process in the mouse model of Sly disease as revealed by expression profiling of selected genes

(2008) Magali Richard et al.   JOURNAL OF NEUROSCIENCE RESEARCH

Neutralizing antibodies to therapeutic enzymes: considerations for testing, prevention and treatment

(2008) Jinhai Wang et al.   NATURE BIOTECHNOLOGY

Maternal Alloantigens Promote the Development of Tolerogenic Fetal Regulatory T Cells in Utero

(2008) J. E. Mold et al.   SCIENCE