Advanced search

Filter By:

Journal Check one or more journals to show results from those journals only.

Choose more journals

Article type Check one or more article types to show results from those article types only.
Subject Check one or more subjects to show results from those subjects only.
Date Choose a date option to show results from those dates only.

Custom date range

Clear all filters
Sort by:
Showing 1–3 of 3 results
Advanced filters: Author: Alejandra Zeballos Clear advanced filters

  • Exon skipping technologies remain hindered by aberrant splicing and low efficacy. Here, Miskalis et.al. developed SPLICER, a Cas9 base editor toolbox which enhances overall exon skipping efficiency and lowers cryptic splicing, demonstrating the use of SPLICER for skipping APP exon 17 in a mouse model of Alzheimer’s disease.

    • Angelo Miskalis
    • Shraddha Shirguppe
    • Pablo Perez-Pinera
    ResearchOpen Access
    Nature Communications
    Volume: 15, P: 1-16

  • TDP43 proteinopathies are a devastating group of neurodegenerative disorders. Here the authors show that RNA-targeting CRISPR effector proteins can be used to mitigate TDP-43 pathology when targeting ataxin-2, a modifier of TDP-43-associated toxicity, and apply this to a mouse model.

    • M. Alejandra Zeballos C.
    • Hayden J. Moore
    • Thomas Gaj
    ResearchOpen Access
    Nature Communications
    Volume: 14, P: 1-17