Gene therapy comes of age
BACKGROUND Nearly five decades ago, visionary scientists hypothesized that genetic
modification by exogenous DNA might be an effective treatment for inherited human …
modification by exogenous DNA might be an effective treatment for inherited human …
Hematopoietic stem cell gene therapy: progress and lessons learned
RA Morgan, D Gray, A Lomova, DB Kohn - Cell stem cell, 2017 - cell.com
The use of allogeneic hematopoietic stem cells (HSCs) to treat genetic blood cell diseases
has become a clinical standard but is limited by the availability of suitable matched donors …
has become a clinical standard but is limited by the availability of suitable matched donors …
Gene therapy in patients with transfusion-dependent β-thalassemia
AA Thompson, MC Walters, J Kwiatkowski… - … England Journal of …, 2018 - Mass Medical Soc
Background Donor availability and transplantation-related risks limit the broad use of
allogeneic hematopoietic-cell transplantation in patients with transfusion-dependent β …
allogeneic hematopoietic-cell transplantation in patients with transfusion-dependent β …
In situ programming of leukaemia-specific T cells using synthetic DNA nanocarriers
TT Smith, SB Stephan, HF Moffett, LE McKnight… - Nature …, 2017 - nature.com
An emerging approach for treating cancer involves programming patient-derived T cells with
genes encoding disease-specific chimeric antigen receptors (CARs), so that they can …
genes encoding disease-specific chimeric antigen receptors (CARs), so that they can …
Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia
M Cavazzana-Calvo, E Payen, O Negre, G Wang… - Nature, 2010 - nature.com
The β-haemoglobinopathies are the most prevalent inherited disorders worldwide. Gene
therapy of β-thalassaemia is particularly challenging given the requirement for massive …
therapy of β-thalassaemia is particularly challenging given the requirement for massive …
How I treat thalassemia
EA Rachmilewitz, PJ Giardina - Blood, The Journal of the …, 2011 - ashpublications.org
The purpose of this article is to set forth our approach to diagnosing and managing the
thalassemias, including β-thalassemia intermedia and β-thalassemia major. The article …
thalassemias, including β-thalassemia intermedia and β-thalassemia major. The article …
β-Thalassemia
D Rund, E Rachmilewitz - New England Journal of Medicine, 2005 - Mass Medical Soc
β-Thalassemia, which is caused by a decrease in the production of β-globin chains, affects
multiple organs and is associated with considerable morbidity and mortality. Lifelong care is …
multiple organs and is associated with considerable morbidity and mortality. Lifelong care is …
Viral vectors for gene therapy: the art of turning infectious agents into vehicles of therapeutics
Considered by some to be among the simpler forms of life, viruses represent highly evolved
natural vectors for the transfer of foreign genetic information into cells. This attribute has led …
natural vectors for the transfer of foreign genetic information into cells. This attribute has led …
Lentiviral vectors: basic to translational
More than two decades have passed since genetically modified HIV was used for gene
delivery. Through continuous improvements these early marker gene-carrying HIVs have …
delivery. Through continuous improvements these early marker gene-carrying HIVs have …