EP
0000-0003-0554-0295 Employment
Employment history is unavailable.
Education
Education history is unavailable.
Projects & Funding
Projects & funding information is unavailable.
Publications (34)
- Liposomal CRISPR/Cas9-Mediated Local Genome Editing for Joint Disease in Mucopolysaccharidosis Type I Save
- Molecular Profile of Mucopolysaccharidosis Type I Patients in Brazil Save
- Newborn Intravenous Injection of Liposomal CRISPR/Cas9 Complex Has No Incidence of Off-Targets or Tumors in Mice Save
- Genome-edited autologous stem cell transplantation with enhanced brain conditioning to correct progranulin deficiency Save
- Hematopoietic stem cell transplantation for lysosomal disorders: Unraveling the mechanisms for CNS repopulation Save
- Basic and translational research in rare diseases in low- and middle-income countries: challenges and solutions. Save
- Laronidase-loaded liposomes reach the brain and other hard-to-treat organs after noninvasive nasal administration. Save
- Effect of genistein and coenzyme Q10 in oxidative damage and mitochondrial membrane potential in an attenuated type II mucopolysaccharidosis cellular model Save
- Clinical development of autologous genome-edited hematopoietic stem cells to treat mucopolysaccharidosis type I Save
- Genome-edited hematopoietic stem cells as a curative approach for Gaucher disease type 1 Save
- Editorial: Genome editing in stem cells. Save
- Pilot study of newborn screening for six lysosomal diseases in Brazil Save
- Engineering Human Hematopoietic Stem and Progenitor Cells for Lineage-Specific Expression of Galactocerebrosidase Using Genome Editing Save
- Microglia Replacement with Bone Marrow-Derived Cells after Transient Inhibition of the Colony-Stimulating Factor 1 Receptor (CSF1R) is Superior to Standard Myeloablative Conditioning in Neuropathic Lysosomal Storage Diseases Save
- Ex vivo gene therapy for lysosomal storage disorders: future perspectives Save
- Diagnosis and Emerging Treatment Strategies for Mucopolysaccharidosis VII (Sly Syndrome). Save
- Biochemical diagnosis of aromatic-L-amino acid decarboxylase deficiency (AADCD) by assay of AADC activity in plasma using liquid chromatography/tandem mass spectrometry Save
- Improved engraftment and therapeutic efficacy by human genome-edited hematopoietic stem cells with Busulfan-based myeloablation. Save
- Experience of the NPC Brazil Network with a Comprehensive Program for the Screening and Diagnosis of Niemann-Pick Disease Type C Save
- Genome Editing for Mucopolysaccharidoses Save