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UCL Great Ormond Street Institute of Child Health

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Nervous System and Craniofacial Abnormalities

Principal Investigator, Professor Patrizia Ferretti

Human-spinal-cord

My research group has a long-standing interest in the basic mechanisms governing regeneration and repair of a variety of complex body structures in vertebrates, and on the relationship between regeneration and the normal and abnormal development of such structures with a particular focus on the nervous system and craniofacial abnormalities. Much focus is currently on better understanding plasticity and differentiation potential of human stem cells, the mechanisms modulating it, and how this knowledge can be used to devise strategies for repairing and restoring functionality in damaged or diseased human tissues. We use a broad range of cellular and molecular techniques and a multidisciplinary approach, supported by an extensive network of national and international collaborations, to address these important issues. 

3d

Results of animal studies, though of great value, cannot be simply extrapolated to humans.  Studying the cellular and molecular basis of human neural development and disease is hampered by the fact that the human central nervous cannot be experimentally investigated in-vivo. Hence, on the nervous system front, much effort in my group is currently devoted to the development of novel human in vitro models. The goal is to establish models relevant to neural development, congenital neural defects and neural damage, including hypoxic-ischaemic damage, that can be used for studying disease mechanisms as well as for toxicology studies and drug testing. Much focus is on 3-dimensional models as they can provide more relevant tissue-like systems for these studies.

Chondro

On the craniofacial abnormality front, our current laboratory-based projects aim at better understanding the plasticity of mesenchymal stem cells and skeletal progenitors, and define environments that can support their full maturation into cartilage (e.g. for ear reconstruction in children with microtia) and bone (e.g. for zygoma reconstruction in children with Treacher Collins Syndrome). The ultimate aim is to bioengineer craniofacial tissues using autologous stem cells to avoid the use of donor graft, and consequent morbidity, as well as immune-rejection. These studies involve close collaborations with biomaterial scientists and are closely integrated with the clinical research interests of clinical and surgical colleagues at Great Ormond Street Hospital, UCL Ear Institute and the Royal Free Hospital. 

Chart

Altogether, our central aim is to further develop both pharmacological and cell-based approaches to neuroprotection and craniofacial tissue repair by building on an increasing understanding of basic cellular and molecular responses to environmental changes and disease.

Current main areas of interest
  • Human somatic stem cell plasticity, with a particular focus on adipose-tissue-derived stem cells, and their potential for craniofacial tissue repair in combination with biocompatible scaffolds and morphometric analysis.
  • Human neural stem cell behavior and their use for establishing 3D models to study human neural development, response to injury (e.g. hypoxic- ischemic insult) and disease mechanisms.  This currently includes development of neural models from patients with Down's syndrome and Duchenne muscular dystrophy.
  • Role of the calcium-dependent enzymes, peptidylarginine deiminases, in health and disease.
  • Cellular and molecular basis underlying changes in the regenerative capability of the spinal cord
Group Members

Miss Reem Alkharji
PhD Student
reem.alkharji.19@ucl.ac.uk
UCL Profiles page: Reem Alkharji


Dr Oliver Gardner
Research Fellow
o.gardner@ucl.ac.uk
UCL Profiles page: Oliver Gardner


Mr Mohammed Hamdi Ibrahim Ali
PhD student
sejjm52@ucl.ac.uk