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Questions tagged [gene-therapy]

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In vivo delivery of ssDNA

I am looking for a system with which I can deliver (large quantities of) ssDNA in vivo (say in mice). I do not know whether it is of importance but the 5' end of the ssDNA fragments are assumed to be ...
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Condition for a genetically modified group of cell to remain and to spread?

I was wondering: when a treatment modifies a group of gene (like in gene therapy), what makes a group of cell remains? I mean, when a cell divide itself to create new ones, if a cell was initially ...
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How does a CRISPR therapy work?

Afaik you can use CRISPR to edit genes of a cell. But how should therapy for a human work based on CRISPR? Can a drug perform it on vast numbers of ill cells?