[HTML][HTML] Side-effect management of chimeric antigen receptor (CAR) T-cell therapy

ML Schubert, M Schmitt, L Wang, CA Ramos… - Annals of oncology, 2021‏ - Elsevier
Highlights•CAR T cells are associated with unique side-effects.•Cytokine release syndrome
(CRS), immune effector cell-associated neurotoxicity syndrome (ICANS) and cytopenias are …

Lentiviral vectors: basic to translational

T Sakuma, MA Barry, Y Ikeda - Biochemical Journal, 2012‏ - portlandpress.com
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 …

Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy

A Biffi, E Montini, L Lorioli, M Cesani, F Fumagalli… - Science, 2013‏ - science.org
Introduction Metachromatic leukodystrophy (MLD) is a neurodegenerative lysosomal
storage disease caused by arylsulfatase A (ARSA) deficiency. The disease primarily affects …

Gene therapy for Wiskott-Aldrich syndrome—long-term efficacy and genotoxicity

CJ Braun, K Boztug, A Paruzynski, M Witzel… - Science translational …, 2014‏ - science.org
Wiskott-Aldrich syndrome (WAS) is characterized by microthrombocytopenia,
immunodeficiency, autoimmunity, and susceptibility to malignancies. In our hematopoietic …

A modified γ-retrovirus vector for X-linked severe combined immunodeficiency

S Hacein-Bey-Abina, SY Pai, HB Gaspar… - … England Journal of …, 2014‏ - Mass Medical Soc
Background In previous clinical trials involving children with X-linked severe combined
immunodeficiency (SCID-X1), a Moloney murine leukemia virus–based γ-retrovirus vector …

Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients

SJ Howe, MR Mansour, K Schwarzwaelder… - The Journal of clinical …, 2008‏ - jci.org
X-linked SCID (SCID-X1) is amenable to correction by gene therapy using conventional
gammaretroviral vectors. Here, we describe the occurrence of clonal T cell acute …

Entering the modern era of gene therapy

XM Anguela, KA High - Annual review of medicine, 2019‏ - annualreviews.org
Gene therapies are gaining momentum as promising early successes in clinical studies
accumulate and examples of regulatory approval for licensing increase. Investigators are …

Ex vivo gene transfer and correction for cell-based therapies

L Naldini - Nature Reviews Genetics, 2011‏ - nature.com
Cell-based therapies are fast-growing forms of personalized medicine that make use of the
steady advances in stem cell manipulation and gene transfer technologies. In this Review, I …

The genotoxic potential of retroviral vectors is strongly modulated by vector design and integration site selection in a mouse model of HSC gene therapy

E Montini, D Cesana, M Schmidt, F Sanvito… - The Journal of clinical …, 2009‏ - jci.org
γ-Retroviral vectors (γRVs), which are commonly used in gene therapy, can trigger
oncogenesis by insertional mutagenesis. Here, we have dissected the contribution of vector …

CD19-targeting CAR T cell immunotherapy outcomes correlate with genomic modification by vector integration

CL Nobles, S Sherrill-Mix, JK Everett, S Reddy… - The Journal of Clinical …, 2020‏ - jci.org
Chimeric antigen receptor–engineered T cells targeting CD19 (CART19) provide an
effective treatment for pediatric acute lymphoblastic leukemia but are less effective for …