Dialog Box

Symposium 2021 - International Speakers

Keynote: Professor Neal Young 

Chief, Haematology  Branch, National Heart, Lung and Blood Institute, National Institutes of Health (NIH), Bethesda, Maryland 

Dr. Neal Young is a world expert in the study of bone marrow failure. His clinical and research interests include aplastic anaemia, telomeropathies and other inherited bone marrow failure syndromes, myelodysplastic syndromes, paroxysmal nocturnal haemoglobinuria, and parvovirus B19. 

His scientific investigations have spanned basic science and translational research, epidemiology and molecular biology, and especially pivotal clinical treatment protocols. His work has led to understanding the pathophysiology of aplastic anaemia as immune-mediated and the development of effective immunotherapy that has dramatically improved survival in this devastating disease.

Most recently, his group has discovered that eltrombopag can stimulate haematopoietic  stem cells in bone marrow failure and that danazol  is both effective in the treatment of telomeropathies and elongates telomeres. 

During the course of his career, he has trained over 100 postdoctoral and clinical fellows and mentored many junior faculty members.  He has published over 500 original research articles, reviews, book chapters, and monographs.

 

Keynote: Professor Alison Bertuch

Professor of Paediatric Haemotology/Oncology and Molecular & Human Genetics, Baylor College of Medicine

Director of the Bone Marrow Failure Program at Texas Children's Hospital  

Professor Bertuch is the director of the Bone Marrow Failure Program at Texas Children’s Hospital, the largest children’s hospital and among the top-ranked paediatric haematology/oncology centres in the United States. She is also Professor of Pediatric Hematology/Oncology and Molecular & Human Genetics at Baylor College of Medicine.

She has a long-standing interest in telomere biology and her research contributions have spanned from the basic science of telomeres to translational and clinical studies on the telomere biology disorders, such as the bone marrow failure predisposition syndrome dyskeratosis congenita. She is particularly interested in the complexity of the molecular genetics underlying these disorders.

Professor Bertuch has served in several leadership roles including as a Co-leader of the Clinical Care Consortium for Telomere Associated Ailments, as an inaugural Executive Committee member of the North American Pediatric Aplastic Anemia Consortium, and recent Chairperson of the American Society of Hematology Scientific Committee on Bone Marrow Failure. She is on the Medical Advisory Board of Team Telomere and the Scientific Advisory Board of the Shwachman-Diamond Syndrome Registry. She has received several honors including election to the American Society for Clinical Investigation and the American Pediatric Society.


Dr  Paula Río

Principal Investigator in the Hematopoietic Innovative Therapies Division at CIEMAT/CIBERER/IIS-FJD, Madrid Spain

Dr Río is a Principal Investigator in the Hematopoietic Innovative Therapies Division at CIEMAT/CIBERER/IIS-FJD, Madrid (Spain). She has been working in the field of gene therapy for Fanconi anemia for more than 20 years. Nowadays she is deeply involved as Associated investigator in two different clinical trials focused on the correction of HSCs from Fanconi anemia A patients (FANCOLEN, Eudra CT: 2011-006100-12 and FANCOLEN II EudraCT 2018-002502-31) (Rio et al., Nat. med, 2019). She is also in charge of Production department of the GMP facility Clinistem for the development of gene therapy trials for rare diseases.

Since 2010 she has been focused in gene editing strategies, first by targeting HSCs from FA patients by Homologous recombination (Rio*, Baños* et al.,EBMM, 2014; Diez et al.,EBMM 2017) and nowadays by Non-homologous end joining using the CRISPR/Cas9 system (Román-Rodríguez et al., Cell Stem Cell, 2019).

Professor Ketan Patel

Director MRC Weatherall Institute of Molecular Medicine and MRC Molecular Haematology Unit, University of Oxford

Professor KJ Patel is the Director of the Weatherall Institute for Molecular Medicine and also the MRC Molecular Hematology Unit at the University of Oxford. KJ trained in medicine in London and spent his research career until recently at the MRC Laboratory of Molecular Biology, Cambridge which is one of the premier research institutes in the world. He was also professor for molecular medicine and stem cell genomics at the University of Cambridge. His research focuses on the molecular basis of inherited genomic instability and the role it plays in the biology of stem cells particularly those that make blood. He is a Fellow of the Royal Society (FRS), Fellow of the Academy of Medical Sciences UK (FMedSci) and also a member of the European Molecular Biology Organisation (EMBO).


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