Gene therapy returns to centre stage

By L. Naldini. Nature, 2015

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How researchers see this paper

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Rheumatoid arthritis: pathological mechanisms and modern pharmacologic therapies (2018, background): “Gene therapy means that treating RA by inserting a gene into a patient’s cells instead of using drugs.(147) Targeting gene therapy in RA is a treatment strategy that is still in very early stages of development but could lead to new possibilities because of treating a disease at its root.”
Transcriptome engineering with RNA-targeting Type VI-D CRISPR effectors (2018, positive): “This allows CasRx effector domain fusions to be paired with a CRISPR array encoding multiple guide RNAs while remaining under the packaging size limit of the versatile AAV delivery vehicle (Naldini, 2015) for primary cell and in vivo delivery.”
Gene therapy comes of age (2018, positive): “Long-term cell replacement afforded by genetically engineered hematopoietic stem cells (HSCs) may durably alleviate a range of conditions, obviating, for example, the need for lifelong enzyme administration or transfusion therapy (3, 4).”