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16 Νοε 2020 · For example, the success of in vivo AAV gene transfer to the human retina and central nervous system by Luxturna and Zolgensma for Leber’s congenital amaurosis and spinal muscular atrophy,...
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8 Νοε 2023 · In this article, we highlight success and ongoing challenges in three areas of high activity in gene therapy: inherited blood cell diseases by targeting hematopoietic stem cells, malignant...
31 Μαΐ 2021 · Typical ex vivo gene therapies involve retrovirally transduced patient-derived hematopoietic stem cells or T cells, which are transplanted back into patients.
The review article presents a broad overview of the field of therapy by in vivo gene transfer, which is based on direct administration of a gene-therapy vector to the body rather than transplant of gene-corrected cells.
9 Νοε 2022 · Gene immunotherapy strategies targeting immune cells to generate cancer therapies in vivo are already seeking commercialization. New companies such as Kelonia Therapeutics and Interius BioTherapeutics are developing viral gene immunotherapy methods to transduce T cells in vivo.
20 Αυγ 2013 · The progression in the field of gene therapy have developed into two different strategies: ex vivo and in vivo gene therapy. 20 Ex vivo gene therapy includes the harvesting of cells from a patient followed subsequently by genetically modification ex vivo in a laboratory.
3 Φεβ 2021 · There are now five treatments approved for commercialization and currently available, i.e., Luxturna, Zolgensma, the two chimeric antigen receptor T cell (CAR-T) therapies (Yescarta and Kymriah), and Strimvelis (the gammaretrovirus approved for adenosine deaminase-severe combined immunodeficiency [ADA-SCID] in Europe).
