Gene therapy using haematopoietic stem and progenitor cells
G Ferrari, AJ Thrasher, A Aiuti - Nature Reviews Genetics, 2021 - nature.com
Haematopoietic stem and progenitor cell (HSPC) gene therapy has emerged as an effective
treatment modality for monogenic disorders of the blood system such as primary …
treatment modality for monogenic disorders of the blood system such as primary …
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 …
A systematic review and meta-analysis of gene therapy with hematopoietic stem and progenitor cells for monogenic disorders
F Tucci, S Galimberti, L Naldini, MG Valsecchi… - Nature …, 2022 - nature.com
Ex-vivo gene therapy (GT) with hematopoietic stem and progenitor cells (HSPCs)
engineered with integrating vectors is a promising treatment for monogenic diseases, but …
engineered with integrating vectors is a promising treatment for monogenic diseases, but …
Genetic engineering meets hematopoietic stem cell biology for next-generation gene therapy
The growing clinical success of hematopoietic stem/progenitor cell (HSPC) gene therapy
(GT) relies on the development of viral vectors as portable" Trojan horses" for safe and …
(GT) relies on the development of viral vectors as portable" Trojan horses" for safe and …
The meaning, the sense and the significance: translating the science of mesenchymal stem cells into medicine
Mesenchymal stem cells (MSCs) are the focus of intensive efforts worldwide directed not
only at elucidating their nature and unique properties but also developing cell-based …
only at elucidating their nature and unique properties but also developing cell-based …
Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome
A Aiuti, L Biasco, S Scaramuzza, F Ferrua, MP Cicalese… - Science, 2013 - science.org
Introduction Wiskott-Aldrich syndrome (WAS) is a primary immunodeficiency characterized
by eczema, thrombocytopenia, infections, and a high risk of developing autoimmunity and …
by eczema, thrombocytopenia, infections, and a high risk of developing autoimmunity and …
Advances in stem cell research and therapeutic development
Despite many reports of putative stem-cell-based treatments in genetic and degenerative
disorders or severe injuries, the number of proven stem cell therapies has remained small …
disorders or severe injuries, the number of proven stem cell therapies has remained small …
Chimeric antigen receptor expressing natural killer cells for the immunotherapy of cancer
RS Mehta, K Rezvani - Frontiers in immunology, 2018 - frontiersin.org
Adoptive cell therapy has emerged as a powerful treatment for advanced cancers resistant
to conventional agents. Most notable are the remarkable responses seen in patients …
to conventional agents. Most notable are the remarkable responses seen in patients …
Gene therapy for ADA‐SCID, the first marketing approval of an ex vivo gene therapy in Europe: paving the road for the next generation of advanced therapy medicinal …
Gene and cell therapy research recently reached a fundamental milestone toward the goal
to deliver new medicines for orphan diseases. In 2016, the European Commission granted …
to deliver new medicines for orphan diseases. In 2016, the European Commission granted …
Gene therapy for Wiskott-Aldrich syndrome—long-term efficacy and genotoxicity
Wiskott-Aldrich syndrome (WAS) is characterized by microthrombocytopenia,
immunodeficiency, autoimmunity, and susceptibility to malignancies. In our hematopoietic …
immunodeficiency, autoimmunity, and susceptibility to malignancies. In our hematopoietic …