Development and clinical translation of approved gene therapy products for genetic disorders

A Shahryari, M Saghaeian Jazi, S Mohammadi… - Frontiers in …, 2019 - frontiersin.org
The field of gene therapy is striving more than ever to define a path to the clinic and the
market. Twenty gene therapy products have already been approved and over two thousand …

Gene therapy on the move

KB Kaufmann, H Büning, A Galy… - EMBO molecular …, 2013 - embopress.org
The first gene therapy clinical trials were initiated more than two decades ago. In the early
days, gene therapy shared the fate of many experimental medicine approaches and was …

[HTML][HTML] Human IL2RA null mutation mediates immunodeficiency with lymphoproliferation and autoimmunity

K Goudy, D Aydin, F Barzaghi, E Gambineri… - Clinical …, 2013 - Elsevier
Cell-surface CD25 expression is critical for maintaining immune function and homeostasis.
As in few reported cases, CD25 deficiency manifests with severe autoimmune enteritis and …

Improving the safety of cell therapy products by suicide gene transfer

BS Jones, LS Lamb, F Goldman… - Frontiers in …, 2014 - frontiersin.org
Adoptive T-cell therapy can involve donor lymphocyte infusion after allogeneic
hematopoietic stem cell transplantation, the administration of tumor infiltrating lymphocyte …

Long-term outcome after haploidentical stem cell transplant and infusion of T cells expressing the inducible caspase 9 safety transgene

X Zhou, A Di Stasi, SK Tey, RA Krance… - Blood, The Journal …, 2014 - ashpublications.org
Adoptive transfer of donor-derived T lymphocytes expressing a safety switch may promote
immune reconstitution in patients undergoing haploidentical hematopoietic stem cell …

When the damage is done: injury and repair in thymus function

S Kinsella, JA Dudakov - Frontiers in immunology, 2020 - frontiersin.org
Even though the thymus is exquisitely sensitive to acute insults like infection, shock, or
common cancer therapies such as cytoreductive chemo-or radiation-therapy, it also has a …

Adoptive T‐cell therapy for cancer: The era of engineered T cells

C Bonini, A Mondino - European journal of immunology, 2015 - Wiley Online Library
Tumors originate from a number of genetic events that deregulate homeostatic mechanisms
controlling normal cell behavior. The immune system, devoted to patrol the organism against …

Improving the safety of cell therapy with the TK-suicide gene

R Greco, G Oliveira, MTL Stanghellini, L Vago… - Frontiers in …, 2015 - frontiersin.org
While opening new frontiers for the cure of malignant and non-malignant diseases, the
increasing use of cell therapy poses also several new challenges related to the safety of a …

Tracking genetically engineered lymphocytes long-term reveals the dynamics of T cell immunological memory

G Oliveira, E Ruggiero, MTL Stanghellini… - Science translational …, 2015 - science.org
Long-lasting immune protection from pathogens and cancer requires the generation of
memory T cells able to survive long-term. To unravel the immunological requirements for …

[HTML][HTML] Immune reconstitution after haploidentical hematopoietic stem cell transplantation

YJ Chang, XY Zhao, XJ Huang - Biology of Blood and Marrow …, 2014 - Elsevier
Haploidentical hematopoietic stem cell transplantation (HSCT) offers the benefits of rapid
and nearly universal donor availability and has been accepted worldwide as an alternative …