MT2015-20: Biochemical Correction of Severe EB by Allo HSCT and Serial Donor MSCs

Inclusion Criteria

• •Diagnosis of severe form of EB characterized by collagen, laminin, integrin, keratin or plakin deficiency (by immunofluorescence staining with protein specific antibodies or Western blotting and by mutation analysis).
• •Adequate organ function within 4 weeks of study registration defined as:
• •* Renal: glomerular filtration rate within normal range for age
• •* Hepatic: Hepatic: bilirubin, AST/ALT, ALP \< 5 x upper limit of normal
• •* Pulmonary: adequate pulmonary function in the opinion of the enrolling investigator
• •* Cardiac: left ventricular ejection fraction ≥ 45%, normal EKG or approved by Cardiology for transplant
• •Sexually active participants must agree to use adequate birth control for the during the study period (from before the start of the preparative chemotherapy through 1 year post-transplant)
• •Available donor per section 5: targeted MFI \< 1,000 (MFI exceeding 1000 must be approved by the PI and treatment team.)
• •Voluntary written consent - adult or parent (with information sheet for minors, if applicable) prior to any research related procedures or treatment