Gene Therapy, cell free – Page 9

Harnessing developmental biology to achieve safe and efficient in vivo genome editing of HSCs

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Post published:August 16, 2026
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Research Objective We seek to develop a more accessible method of genome editing of blood stem cells directly in the body, so that the treatment can be safer and less…

Role of ataxin-3 polyadenylation site selection in ALS neuron toxicity and disease pathogenesis

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Post published:August 16, 2026
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Research Objective Here we will study the role of ataxin-3 alternative polyadenylation in the pathogenesis of ALS, and test if ASOs can reduce distal polyadenylation of ataxin-3 to rescue ALS…

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