In vivo HSC gene therapy for SARS-CoV2 infection using a decoy receptor.
Sign Up to like & getrecommendations! Published in 2022 at "Human gene therapy"
DOI: 10.1089/hum.2021.295
Abstract: While SARS-CoV2 vaccines have shown an unprecedented success, the ongoing emergence of new variants and necessity to adjust vaccines justifies the development of alternative prophylaxis and therapy approaches. Hematopoietic stem cell (HSC) gene therapy using… read more here.
Keywords: sars cov2; vivo; vivo hsc; therapy ... See more keywords
In Vivo HSC Gene Therapy for Hemoglobinopathies: A Proof of Concept Evaluation in Rhesus Macaques
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DOI: 10.1182/blood-2020-141468
Abstract: Current gene therapy or genome editing studies for hemoglobinopathies require highly sophisticated medical facilities to perform hematopoietic stem cell collections / selections and genetic modifications. In addition, patients receive high-dose chemotherapy to facilitate engraftment of… read more here.
Keywords: bone marrow; vivo hsc; hsc gene; gene ... See more keywords
In vivo HSC prime editing rescues sickle cell disease in a mouse model
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DOI: 10.1182/blood.2022018252
Abstract: Key Points • Correction of the sickle-cell mutation and disease phenotypes is achieved by in vivo HSC transduction with vectorized prime editors.• Our approach for in vivo HSC prime editing that does not require HSC… read more here.
Keywords: disease; prime editing; vivo hsc; hsc prime ... See more keywords
In vivo HSC transduction in rhesus macaques with an HDAd5/3+ vector targeting desmoglein 2 and transiently overexpressing cxcr4
Sign Up to like & getrecommendations! Published in 2022 at "Blood Advances"
DOI: 10.1182/bloodadvances.2022007975
Abstract: Abstract We developed a new in vivo hematopoietic stem cell (HSC) gene therapy approach that involves only IV injections and does not require myeloablation/conditioning and HSC transplantation. In this approach, HSCs are mobilized from the… read more here.
Keywords: hsc transduction; bone marrow; transduction; vector ... See more keywords