Allogeneic haematopoietic stem cell transplantation for mitochondrial neurogastrointestinal encephalomyopathy

Outcomes of transplantation using various hematopoietic cell sources in children with Hurler syndrome after myeloablative conditioning

(2013) J. J. Boelens et al.   BLOOD

Clinical and biochemical improvements in a patient with MNGIE following enzyme replacement

(2013) B. E. Bax et al.   NEUROLOGY

Clinical and biochemical improvement following HSCT in a patient with MNGIE: 1-year follow-up

(2012) F. Sicurelli et al.   JOURNAL OF NEUROLOGY

Course and management of allogeneic stem cell transplantation in patients with mitochondrial neurogastrointestinal encephalomyopathy

(2012) Massimiliano Filosto et al.   JOURNAL OF NEUROLOGY

Clinical and genetic spectrum of mitochondrial neurogastrointestinal encephalomyopathy

(2011) Caterina Garone et al.   BRAIN

Hematopoietic gene therapy restores thymidine phosphorylase activity in a cell culture and a murine model of MNGIE

(2011) J Torres-Torronteras et al.   GENE THERAPY

Allogeneic hematopoietic SCT as treatment option for patients with mitochondrial neurogastrointestinal encephalomyopathy (MNGIE): a consensus conference proposal for a standardized approach

(2010) J Halter et al.   BONE MARROW TRANSPLANTATION

Outcomes after related and unrelated umbilical cord blood transplantation for hereditary bone marrow failure syndromes other than Fanconi anemia

(2010) R. Bizzetto et al.   HAEMATOLOGICA

Collated mutations in mitochondrial DNA (mtDNA) depletion syndrome (excluding the mitochondrial gamma polymerase, POLG1)

(2009) J. Poulton et al.   BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE

Defining the Intensity of Conditioning Regimens: Working Definitions

(2009) Andrea Bacigalupo et al.   BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION