CRISPR gene editing to cure beta thalassaemia and sickle cell disease
New Scientist - 12-Jun-2020Didn't change the inherited faulty gene, but gene to switch on fetal haemoglobin production
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Co-Founder and Acting CMO at Smart Immune, hematopoietic stem cell-based biotherapies
MARINA CAVAZZANA is a pediatrician, Professor of Hematology since 2000, Director of the Department of Biotherapy at Necker Hospital, Paris Descartes University. She is the Director of the Inserm / Assistance Publique – Hôpitaux de Paris GHU Ouest Biotherapy Clinical Investigation Center and leads the research “Human lymphohematopoiesis” Laboratory at Imagine Institute.
Her main research and clinical interests are the development of the hematopoietic immune system, and cell and gene therapy for inherited and acquired disease of the hematopoietic system. Her group studies the means to improve the clinical results of hematopoietic stem cell transplantation, crossing HLA-barriers, and the differentiation of mouse and human stem cells towards lymphocyte lineages. She has initiated several clinical trials based on the use of ex vivo gene modified hematopoietic stem cells to treat patients with inherited disorders, the preliminary clinical results of which are encouraging. She is the author or co-author of one patent and of more than 270 publications in peer-reviewed journals and was awarded 2 ERC (2011 and 2016).
See also: Smart Immune - Biotechnology company focused on developing the next generation of hematopoietic stem cell-based biotherapies
Details last updated 20-Jun-2020
Didn't change the inherited faulty gene, but gene to switch on fetal haemoglobin production
People with sickle cell disease make abnormal versions of haemoglobin – distorting red blood cell...