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[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 …
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 …
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 …
storage disease caused by arylsulfatase A (ARSA) deficiency. The disease primarily affects …
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 …
A modified γ-retrovirus vector for X-linked severe combined immunodeficiency
Background In previous clinical trials involving children with X-linked severe combined
immunodeficiency (SCID-X1), a Moloney murine leukemia virus–based γ-retrovirus vector …
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
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 …
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 …
accumulate and examples of regulatory approval for licensing increase. Investigators are …
Ex vivo gene transfer and correction for cell-based therapies
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 …
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 …
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
Chimeric antigen receptor–engineered T cells targeting CD19 (CART19) provide an
effective treatment for pediatric acute lymphoblastic leukemia but are less effective for …
effective treatment for pediatric acute lymphoblastic leukemia but are less effective for …