Evaluating the state of the science for adeno-associated virus integration: An integrated perspective
On August 18, 2021, the American Society of Gene and Cell Therapy (ASGCT) hosted a
virtual roundtable on adeno-associated virus (AAV) integration, featuring leading experts in …
virtual roundtable on adeno-associated virus (AAV) integration, featuring leading experts in …
[HTML][HTML] Side-effect management of chimeric antigen receptor (CAR) T-cell therapy
Highlights•CAR T cells are associated with unique side-effects.•Cytokine release syndrome
(CRS), immune effector cell-associated neurotoxicity syndrome (ICANS) and cytopenias are …
(CRS), immune effector cell-associated neurotoxicity syndrome (ICANS) and cytopenias are …
Hematologic cancer after gene therapy for cerebral adrenoleukodystrophy
CN Duncan, JR Bledsoe, B Grzywacz… - … England Journal of …, 2024 - Mass Medical Soc
Background Gene therapy with elivaldogene autotemcel (eli-cel) consisting of autologous
CD34+ cells transduced with lentiviral vector containing ABCD1 complementary DNA (Lenti …
CD34+ cells transduced with lentiviral vector containing ABCD1 complementary DNA (Lenti …
A systematic review and meta-analysis of gene therapy with hematopoietic stem and progenitor cells for monogenic disorders
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 …
Gene therapy using haematopoietic stem and progenitor cells
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 …
Lentiviral gene therapy for X-linked chronic granulomatous disease
Chronic granulomatous disease (CGD) is a rare inherited disorder of phagocytic cells,. We
report the initial results of nine severely affected X-linked CGD (X-CGD) patients who …
report the initial results of nine severely affected X-linked CGD (X-CGD) patients who …
[HTML][HTML] Role of myeloperoxidase and oxidant formation in the extracellular environment in inflammation-induced tissue damage
The heme peroxidase family generates a battery of oxidants both for synthetic purposes, and
in the innate immune defence against pathogens. Myeloperoxidase (MPO) is the most …
in the innate immune defence against pathogens. Myeloperoxidase (MPO) is the most …
Gene editing and CRISPR in the clinic: current and future perspectives
Genome editing technologies, particularly those based on zinc-finger nucleases (ZFNs),
transcription activator-like effector nucleases (TALENs), and CRISPR (clustered regularly …
transcription activator-like effector nucleases (TALENs), and CRISPR (clustered regularly …
Role of recombinant DNA technology to improve life
In the past century, the recombinant DNA technology was just an imagination that desirable
characteristics can be improved in the living bodies by controlling the expressions of target …
characteristics can be improved in the living bodies by controlling the expressions of target …
A review of chronic granulomatous disease
DE Arnold, JR Heimall - Advances in therapy, 2017 - Springer
Chronic granulomatous disease (CGD) is a primary immunodeficiency caused by defects in
any of the five subunits of the NADPH oxidase complex responsible for the respiratory burst …
any of the five subunits of the NADPH oxidase complex responsible for the respiratory burst …