subject area of
- Autologous, lentivirus‐modified, T‐rapa cell “micropharmacies” for lysosomal storage disorders Journal Articles
- Behavioural abnormalities in a murine model of a human lysosomal storage disease Journal Articles
- Bone marrow involvement and obstructive jaundice in Farber lipogranulomatosis: clinical and autopsy report of a new case Journal Articles
- Cloning of the cDNA and Gene Encoding Mouse Lysosomal Sialidase and Correction of Sialidase Deficiency in Human Sialidosis and Mouse SM/J Fibroblasts Journal Articles
- Cloning, expression and chromosomal mapping of human lysosomal sialidase and characterization of mutations in sialidosis Journal Articles
- Encapsulation for Somatic Gene Therapy Conferences
- Limited responsiveness related to the minimal important difference of patient-reported outcomes in rare diseases Journal Articles
- Neuraminidase 4 (NEU4): new biological and physiological player Journal Articles
- Nonautologous gene therapy with implantable devices Journal Articles
- PATIENT-REPORTED OUTCOMES IN RARE LYSOSOMAL STORAGE DISEASES: KEY INFORMANT INTERVIEWS AND A SYSTEMATIC REVIEW PROTOCOL Journal Articles
- The in vivo delivery of heterologous proteins by microencapsulated recombinant cells Journal Articles