Michaela S.
- Country
- France
- Department
- Centre d’Investigations Cliniques CIC-1419 Mère- Enfants
Research Overview
- Phase I/II Open‑Label Study Assessing Safety and Efficacy of LV‑FOXP3‑T4 Induced CD4⁺ Treg Cell Therapy ± Low‑Dose Aldesleukin in Patients with IPEX Syndrome
- 48‑Week Verapamil Hydrochloride to Preserve Beta‑Cell Function in Ages 7–45 With Stage 1 or 2 Type 1 Diabetes – Randomized Placebo‑Controlled Trial
- A Phase II double-blind multi-center, placebo-controlled trial, to assess the efficacy and safety of alpelisib (BYL719) in pediatric and adult patients with Megalencephaly-CApillary malformation Polymicrogyria syndrome (MCAP)
Data & Insights
Has run more than one trial at 2 of 21 partner sites.
Specializations
Genetic Immunodeficiencies
Specializes in treating rare genetic disorders affecting the immune system, including Familial Hemophagocytic Lymphohistiocytosis (FHL) and X-Linked Chronic Granulomatous Disease. Focuses on innovative gene therapy approaches using modified autologous cells.
- Hematopoietic stem cell therapy
- Lentiviral vector treatments
- Immunological reconstitution
Particularly interested in cellular modification techniques for immune system disorders.
Developmental Disorders
Investigates therapeutic solutions for complex developmental conditions, with expertise in treating Megalencephaly-Capillary Malformation Polymicrogyria Syndrome (MCAP). Studies targeted molecular therapies for rare genetic syndromes.
- Neurological development disorders
- Targeted molecular treatments
- Pediatric genetic conditions
Focuses on long-term therapeutic interventions for developmental disorders.
Innovative Therapies
Explores cutting-edge treatments including Fecal Microbiota Transplantation for inflammatory bowel conditions and advanced gene therapy for Severe Combined Immunodeficiency (SCID). Specializes in novel therapeutic approaches for complex medical conditions.
- Microbiome-based treatments
- Gene therapy applications
- Autoimmune disorders
Develops pioneering treatment strategies for previously untreatable conditions.
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