Research Highlights

UCL Stem Cell Research

Research Highlights

Stem-cell-based, tissue engineered tracheal replacement in a child: a 2-year follow-up study The first child stem cell-supported trachea transplant is functioning well two years on. The follow-up of the procedure, published in The Lancet on 26 July 2012, shows that the new organ has strengthened and does not appear to have induced any signs of rejection. Read "Stem-cell-based, tissue engineered tracheal replacement in a child: a 2-year follow-up study" In a video interview, UCL Prof Martin Birchall describes the case, and explains how stem-cell based, tissue engineered transplants might offer new therapeutic options for patients, including children, with failing organs.
Transplant of patient-derived stem cells into mice with muscular dystrophy Scientists led by Dr Francesco Saverio Tedesco and Professor Giulio Cossu have reprogrammed muscular dystrophy patients' fibroblast cells into stem cells and then differentiated them into muscle precursor cells. The muscle cells were then genetically modified and transplanted into mice. Their work is published in *Science translational Medicine* on 27 June 2012. Read "Transplantation of Genetically Corrected Human iPSC-Derived Progenitors in Mice with Limb-Girdle Muscular Dystrophy"
Restoration of vision after transplantation of photoreceptors UCL Professor Robin Ali and Dr Rachel Pearson have shown for the first time that transplanting light-sensitive photoreceptors into the eyes of visually impaired mice can restore their vision. The research is published in *Nature*, 18 April 2012. Read "Restoration of vision after transplantation of photoreceptors"
Step towards creating intestine transplant using patient’s own cells UCL Dr Paolo de Coppi and colleagues have reported a new protocol that creates a natural intestinal scaffold, as a base for developing functional intestinal tissue. This work, published online in Biomaterials on 3 February 2012, represents a step towards creating intestine transplant using patient’s own cells. Read "A rat decellularized small bowel scaffold that preserves villus-crypt architecture for intestinal regeneration"
Haemophilia gene therapy shows early success Study of gene therapy developed at UCL and St. Jude Children’s Research Hospital (USA) and led by UCL Dr Amit Nathwani offers first proof adults with haemophilia B benefit from treatment The findings are published in New England Journal of Medicine, 10 December 2011 Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B
Tracheobronchial transplantation with a stem-cell-seeded bioartificial nanocomposite UCL Professor Alex Seifalian developed an artificial windpipe using a novel nanocomposite polymer; it was then coated with the patient's stem cells and implanted in a 36 year-old cancer patient. The surgery was performed by Prof. Paolo Macchiarini (who holds a UCL Honorary appointment) at the Karolinska University Hospital in Stockholm, Sweden. The results of this proof-of-concept study were published online in The Lancet on 24 November 2011. Tracheobronchial transplantation with a stem-cell-seeded bioartificial nanocomposite: a proof-of-concept study
New gene identified for familial motor neuron disease UCL Professor John Hardy is part of an international team that has identified a new gene for motor neurone disease. Their findings are published in Neuron, 21 September 2011. "A Hexanucleotide Repeat Expansion in C9ORF72 Is the Cause of Chromosome 9p21-Linked ALS-FTD"
Gene therapy success for children born without functioning immune system Two papers published on 24 August 2011 in Science Translational Medicine report success in two established gene therapy programmes to cure children born unable to fight infection. The programmes, which looked at X-SCID and ADA-SCID, were led by Professors Adrian Thrasher and Bobby Gaspar of the UCL Institute of Child Health. 14 out of 16 patients across the two programmes were successfully treated. "Long-Term Persistence of a Polyclonal T Cell Repertoire After Gene Therapy for X-Linked Severe Combined Immunodeficiency" "Hematopoietic Stem Cell Gene Therapy for Adenosine Deaminase–Deficient Severe Combined Immunodeficiency Leads to Long-Term Immunological Recovery and Metabolic Correction"
First targeted treatment success for Duchenne Muscular Dystrophy A team of UCL scientists together with the MDEX Consortium chaired by UCL Professor Francesco Muntoni show that a gene-based drug treatment was effective in restoring the dystrophin protein that is missing in sufferers of Duchenne Muscular Dystrophy , in 7 out of 19 trial participants. Their findings are published in The Lancet, 25 July 2011. "Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study"
Turning back the cellular clock could repair damage after a heart attack UCL Professor Paul Riley explains how his team restored the ability of mouse heart cells to differentiate and repair damage caused by a heart attack. Their findings are published in Nature, 8 June 2011. "De novo cardiomyocytes from within the activated adult heart after injury"

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