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  1. No Access

    Article

    Central and peripheral delivered AAV9-SMN are both efficient but target different pathomechanisms in a mouse model of spinal muscular atrophy

    Spinal muscular atrophy (SMA) is a neuromuscular disease caused by loss of the SMN1 gene and low SMN protein levels. Although lower motor neurons are a primary target, there is evidence that peripheral organ defe...

    Aoife Reilly, Marc-Olivier Deguise, Ariane Beauvais, Rebecca Yaworski… in Gene Therapy (2022)

  2. Article

    Open Access

    Digital immunoassay for biomarker concentration quantification using solid-state nanopores

    Single-molecule counting is the most accurate and precise method for determining the concentration of a biomarker in solution and is leading to the emergence of digital diagnostic platforms enabling precision ...

    Liqun He, Daniel R. Tessier, Kyle Briggs, Matthaios Tsangaris… in Nature Communications (2021)

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