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Enhancement of Mouse Hematopoietic Stem/Progenitor Cell Function Via Transient Gene Delivery Using Integration-Deficient Lentiviral Vectors
(Elsevier Science Bv, 2018)
Integration-deficient lentiviruses (IdLVs) deliver genes effectively to tissues but are lost rapidly from dividing cells. This property can be harnessed to express transgenes transiently to manipulate cell biology. Here, ...
The Potential For Chemical Mixtures From The Environment To Enable The Cancer Hallmark Of Sustained Proliferative Signalling
(Oxford Univ Press, 2015)
The aim of this work is to review current knowledge relating the established cancer hallmark, sustained cell proliferation to the existence of chemicals present as low dose mixtures in the environment. Normal cell proliferation ...
Lentiviral Hematopoietic Stem Cell Gene Therapy in Inherited Immune and Lysosomal Enzyme Deficiencies
(CyberLeninka, 2016)
Rare diseases affect millions of people worldwide. Many of those are inherited disorders resulting in chronic disability and requiring cost-intensive care. Hematopoietic stem cell gene therapy has been developed over more ...
Preclinical Efficacy and Safety Evaluation of Hematopoietic Stem Cell Gene Therapy in a Mouse Model of Mngie
(Elsevier, 2018)
Mitochondrial neurogastrointestinal encephalomyopathy (MNGIE) is an autosomal recessive disorder caused by thymidine phosphorylase (TP) deficiency resulting in systemic accumulation of thymidine (d-Thd) and deoxyuridine ...
Lentiviral Hematopoietic Stem Cell Gene Therapy in Inherited Metabolic Disorders
(Mary Ann Liebert, Inc, 2014)
After more than 20 years of development, lentiviral hematopoietic stem cell gene therapy has entered the stage of initial clinical implementation for immune deficiencies and storage disorders. This brief review summarizes ...