Concepedia

Intrabone hematopoietic stem cell gene therapy for adult and pediatric patients affected by transfusion-dependent ß-thalassemia

Sarah Marktel, Samantha Scaramuzza, Maria Pia Cicalese, Fabio Giglio, Stefania Galimberti, Maria Rosa Lidonnici, Valeria Calbi, Andrea Assanelli, Maria Ester Bernardo, Claudia Rossi,

Nature Medicine · 2019 · 225 citations · 32 references

DOI

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32

Global epidemiology of haemoglobin disorders and derived service indicators

Bernadette Modell · Bulletin of the World Health Organization · 2008 · 1.9K citations · Full text

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Hematopoietic Stem Cell Gene Therapy with a Lentiviral Vector in X-Linked Adrenoleukodystrophy

Nathalie Cartier, Salima Hacein‐Bey‐Abina, Cynthia C. Bartholomae et al. · Science · 2009 · 1.5K citations · Full text

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Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia

Marina Cavazzana, Emmanuel Payen, Olivier Nègre et al. · Nature · 2010 · 1.3K citations

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Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy

Alessandra Biffi, Eugenio Montini, Laura Lorioli et al. · Science · 2013 · 1.2K citations · Full text

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MRI R2 and R2* mapping accurately estimates hepatic iron concentration in transfusion-dependent thalassemia and sickle cell disease patients

John C. Wood, Cathleen Enriquez, Nilesh R. Ghugre et al. · Blood · 2005 · 1K citations · Full text

, , +17