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Clinica Chimica Acta; International Journal of Clinical Chemistry|May 26, 2019
Inherited monogenic defects of ceramide metabolism: Molecular bases and diagnosesPatricia Dubot, Frédérique Sabourdy, Jitka Rybova, et al.
Journal of Inherited Metabolic Disease|September 9, 2022
Skin inflammation and impaired adipogenesis in a mouse model of acid ceramidase deficiencyJitka Rybova, Ladislav Kuchar, Jakub Sikora, et al.
Biomolecules|February 25, 2023
Acid Ceramidase Deficiency: Bridging Gaps between Clinical Presentation, Mouse Models, and Future Therapeutic InterventionsAnnie Kleynerman, Jitka Rybova, Mary L Faber, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|August 7, 2024
Hematopoietic stem cell transplantation leads to biochemical and functional correction in two mouse models of acid ceramidase deficiencyJitka Rybova, Teresa Sundararajan, Ladislav Kuchar, et al.
Cytotherapy|April 3, 2026
Improved lentivector-modified hematopoietic stem cell transplantation in the Townes mouse model of sickle cell diseaseTheresa A Dlugi, Juliana Alvarez-Argote, Teresa Sundararajan, et al.
American Journal of Physiology. Heart and Circulatory Physiology|December 12, 2024
Cardiac dysfunction and altered gene expression in acid ceramidase-deficient miceAnnie Kleynerman, Jitka Rybova, William M McKillop, et al.
EMBO Molecular Medicine|March 17, 2022
Autologous, lentivirus-modified, T-rapa cell "micropharmacies" for lysosomal storage disordersMurtaza S Nagree, Tania C Felizardo, Mary L Faber, et al.
Laboratory Investigation; a Journal of Technical Methods and Pathology|June 13, 2019
Hepatic pathology and altered gene transcription in a murine model of acid ceramidase deficiencyFabian P S Yu, Salvatore Molino, Jakub Sikora, et al.
Communications Biology|May 25, 2023
Spinal muscular atrophy-like phenotype in a mouse model of acid ceramidase deficiencyMurtaza S Nagree, Jitka Rybova, Annie Kleynerman, et al.
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