Ronald Klein on Scientists Eager to Test ALS Gene Therapy

COMMENT In our paper (Jackson et al., 2015), we measured endogenous levels of TDP-43 mRNA to address auto-regulation. We also carefully studied whether co-expressing two transgenes with AAV resulted in non-specific gene competition. The data in the paper support ...

Ronald Klein on First C9ORF72 Mice Mimic Key Pathology, Behavior

COMMENT This is an exciting and potentially very important new model system for C9 forms of ALS and FTLD. The paper underscores the utility of somatic cell gene transfer with AAV vectors for disease modeling. The vector approach is rapid and therefore expedites ...

Ronald Klein on Stem Cells: New Ways to Make Them Branch Out

COMMENT The nanoparticle gene transfer described by Bharali et al. is an exciting new avenue for genetic modification in the brain. The study suggests the organically modified nanoparticles are easy to make, are safe, and provide efficient and widespread ...

Ronald Klein on Viral Transgene Models Parkinson's in Primate

COMMENT The findings by Kirik et al. are a breakthrough because they provide a human-like model of Parkinson's disease and make the earlier findings in rats (Kirik et al., 2002; Lo Bianco et al., 2002; Klein et al., 2002) even more relevant for Parkinson& ...

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